Bill Would Improve Children’s Access to Life-Saving Medical Care, Simplify Out-of-State Medicaid Enrollment Processes for Pediatric Care Providers

Washington, D.C. – The Rare Disease Company Coalition (RDCC) is proud to endorse the bipartisan, bicameral Accelerating Kids’ Access to Care Act (S. 2372/H.R. 4758), introduced by Senators Chuck Grassley and Michael Bennet, and Representatives Lori Trahan and Mariannette Miller-Meeks. The bill is intended to improve access to healthcare services for children with medically complex conditions, including those living with rare diseases by removing barriers to out-of-state health care. By streamlining the complicated and lengthy Medicaid provider screening and enrollment process, this bill would enable children to receive more timely access to the medical expertise and care they require.

People living with a rare disease face many challenges in the journey from diagnosis to accessing appropriate treatment. With over 10,000 rare diseases, and for many rare conditions, there may only be a handful of clinical centers scattered across the country that have a specialist with the expertise to treat a specific rare disease. As a result, rare disease patients and their caregivers are often required to cross state lines to receive proper care.

Because state Medicaid programs often require out-of-state providers to be screened and enrolled into their state program, individuals living with a rare disease face complicated hurdles, lengthy delays, and burdensome processes. These barriers can divert precious time and resources, or even block access entirely. This issue is only heightened with pediatric patients.

“For children with rare diseases, accessing specialized care — no matter where their doctor is located — is absolutely crucial,” said Curt Oltmans, Chair of the Rare Disease Company Coalition. “Passing the Accelerating Kids’ Access to Care Act has the ability to change the lives of families by removing significant hurdles to life-saving care, ensuring no parent has to waste precious time to get their child to the specialist they need.”

The Accelerating Kids’ Access to Care Act provides a solution by simplifying and streamlining the process for out-of-state pediatric care providers to enroll in another state’s Medicaid program. By creating a new process that allows pediatric providers to enroll in multiple programs, the bill aims to ensure that state Medicaid programs can cover necessary care regardless of where that care occurs within the U.S.

The RDCC is proud to join the over 200 organizations nationwide that have signed on to support the Accelerating Kids’ Access to Care Act. We urge Congress to act now to ensure our children have access to the care they need.

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About the Rare Disease Company Coalition (RDCC): Founded in May 2021, the Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes so that critical innovation can continue and positive changes can be enacted for the rare disease community. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Program Provides Crucial Incentives to Spur the Research and Development of Rare Disease Treatments for Most Vulnerable Population

Washington, D.C. – The Rare Disease Company Coalition (RDCC) is proud to endorse H.R. 7384, The Creating Hope Reauthorization Act of 2024, which would reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program. H.R. 7384 was introduced Thursday by Representatives Michael McCaul (R-TX-10), Gus Bilirakis (R-FL-12), Michael Burgess (R-TX-26), Anna Eshoo (CA-D-16), Nanette Barragán (CA-D-44) and Lori Trahan (MA-D-3). 

Half of the 30 million individuals living with a rare disease in the United States are children – and 30 percent of those children won’t live to see their fifth birthday. Pediatric rare disease research is critical to provide hope for these children and their families. 

Over the past decade, the program has proven to be an innovative and cost-effective policy that spurs research & development (R&D) in rare pediatric populations. The PRV program enables RDCC members to provide hope to children living with a rare disease while reinvesting critical dollars into additional R&D for rare disease treatments. The program has zero cost to taxpayers, and remains a critical incentive to attract investment into rare disease drug development.  

The PRV program is set to expire on September 30, 2024, and without Congressional action, hope for millions of children may vanish.

“It is absolutely critical that Congress reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, which provides a crucial incentive for life science companies to direct research and resources toward drug development for our most vulnerable population – our children,” said Curt Oltmans, Chair of the Rare Disease Company Coalition. “Reauthorization of this program will allow our members to continue discovering, developing, and delivering treatments to children living with a rare disease. The Rare Disease Company Coalition is grateful to champions in the House who are leading the way to reauthorize this life-changing program.”

Rare disease drug development is uniquely challenging, and with those challenges come increased risk. Incentives like the PRV program are crucial to mitigating the financial risks associated with investment in the rare disease space. Without incentives like PRVs, investors may view rare disease drug development as too risky – and for pre commercial companies that rely on capital markets for funding, that could make the difference between bringing a product to market and having to shutter programs. Research shows that rare disease companies are struggling to survive in the current policy and funding ecosystems, and policies like the PRV program are necessary to attract investors that are fundamental to rare disease drug development. 

The RDCC applauds the introduction of the Creating Hope Reauthorization Act, and urges Congress to act quickly to reintroduce the Rare Pediatric Disease PRV Program. 

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About the Rare Disease Company Coalition (RDCC): Founded in May 2021, the Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes so that critical innovation can continue and positive changes can be enacted for the rare disease community. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

The RDCC sent a letter to Senator Cassidy and the Senate Health, Education, Labor, and Pensions (HELP) Committee in response to the recent request for information (RFI) on ways to improve and protect access to gene therapies for Americans with rare ultra-rare diseases. Coverage and reimbursement is a critical issue for all patients living with a rare disease and their families. As the RFI recognizes, if a patient cannot access innovative treatments, to them it is as if that innovation never occurred. 

RDCC members currently have over 200 programs in development, many of which are first-in-class gene and cell therapies. With the appropriate policy framework, these therapies could ultimately prove life changing or life saving to patients with no other treatments available.  

But current coverage and reimbursement policies have not kept pace with the rapidly-evolving science. We urge policymakers to consider solutions to ensure the ecosystem is better-equipped to accommodate tremendous leaps in innovation in the development of gene and cell therapies and ensure timely access for patients living with a rare disease. 

Interested in learning more about the RDCC’s recommendations? Click here.

The Rare Disease Company Coalition (RDCC) strongly supports the ORPHAN Cures Act introduced by Senators Barrasso (R-WY) and Carper (D-DE) and Representatives Joyce (R-PA-13) and Nickel (D-NC-13). The bipartisan and bicameral ORPHAN Cures Act works to encourage rare disease research and development (R&D) by addressing a critical disincentive in the Inflation Reduction Act (IRA) that impedes rare disease innovation and investment.

Currently, the IRA’s Orphan Drug Exclusion offers a price negotiation exemption for orphan drugs that treat only one orphan condition. Rare disease companies rely on this exemption as a critical incentive for R&D, but in limiting it to drugs that treat only one condition the IRA discourages rare disease companies from further exploring promising research that could lead to additional treatment options. The ORPHAN Cures Act would instead expand the Orphan Drug Exclusion to allow for the research and development of existing products that could help find treatments for the more than 95 percent of rare diseases without approved therapies.

For RDCC member companies that invest over half of their annual revenues back into research and development, improving the Orphan Drug Exclusion will have a significant impact in delivering desperately needed therapies and hope to underserved patient and caregiver communities.

Real-life impact: 1 in 5 orphan drugs are Food and Drug Administration (FDA)-approved for more than one use, and 60% of those second indications are for another rare disease. By limiting the Orphan Drug Exclusion to medicines that treat only one indication, the IRA discourages companies from pursuing development of promising therapies.

The unique needs of rare disease: The current IRA Orphan Drug Exclusion policy does not reflect the unmet need for treatments for rare diseases and the unique demands of rare disease drug development. The ORPHAN Cures Act ensures developers can continue to direct valuable resources towards rare disease research, building on the 40 years of progress enabled by the Orphan Drug Act (ODA).

The ORPHAN Cures Act strengthens the intent of the ODA: Since the enactment of the ODA in 1983, the number of orphan drugs approved by the FDA has increased from just 38 to more than 600 treatments for more than 1,000 rare diseases. While this has made a dent on the 95% of the 10,000 rare diseases that exist, continued progress requires both strengthening ODA policies and continuing to advance recognition of and support for the unique needs of rare disease R&D. The ORPHAN Cures Act achieves this by removing a disincentive for developing rare disease treatments.

The final word: The ORPHAN Cures Act addresses the concerning effects of the IRA’s Orphan Drug Exclusion and allows biopharmaceutical companies to meet the clear and urgent need for continued innovation in rare disease R&D. The RDCC thanks Senators Barrasso and Carper and Representatives Joyce and Nickel for supporting the rare disease community through this important legislation.

An Experienced DC Regulatory and Policy Counsel, Owens Brings Expertise on Navigating Rare Disease Policy Landscape

Washington, DC – November 15, 2023 – The Rare Disease Company Coalition (RDCC) recently named Graham Owens as Executive Director of the Coalition. Owens joins the RDCC at a pivotal time as the RDCC continues to advance a stronger and more stable policy environment for rare diseases.  The RDCC provides a unified voice for life science companies committed to discovering, developing, and delivering rare disease treatments

“I am inspired by the great progress the Coalition has made in advancing rare disease policy and I am excited to further address the needs of RDCC companies and the patients they serve,” said Graham Owens, Executive Director of the RDCC. “As the rare disease community faces policy and regulatory uncertainty, , the RDCC plays a crucial role in advocating for investment in and support of rare disease drug development. This support ensures rare disease companies can continue to develop critical treatments for the 95% of rare diseases without an FDA-approved treatment.”

“As Chair, I am immensely excited to welcome Graham as Executive Director of the RDCC,” said Deirdre Parsons, Board Chair of the RDCC. “Graham’s professional background and energy will further advance the Coalition as the leading voice for rare disease innovators in 2024 and beyond.” 

Owens most recently served as the Regulatory and Oversight Counsel for the Senate Committee on Small Business and Entrepreneurship. His experience drafting and moving legislation to ensure regulations are not “one-size-fits-all” aligns with the RDCC’s mission of promoting policies that consider the unique challenges and opportunities facing rare disease innovators today. Prior to serving in the Senate, Owens was President of Delta Agriculture where he led efforts to navigate and address key regulations and oversaw the company’s government relations portfolio, among other roles. He also previously served as the Director of Regulatory, Tax and Domestic Economic Policy for the National Association of Manufacturers (NAM). He is a graduate of the University of Virginia and the George Washington University Law School, and an active member of the District of Columbia Bar.

About the Rare Disease Company Coalition (RDCC)

Founded in May 2021, the Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes so that critical innovation can continue and positive changes can be enacted for the rare disease community. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Healthcare Capital Group Unveils White Paper Addressing Financial Risks and Policy Challenges Threatening the Future of Rare Disease Innovation

WASHINGTON, DC – (October 26, 2023) – On Wednesday, October 25, the Rare Disease Company Coalition (RDCC) hosted a congressional briefing on Capitol Hill. The briefing unveiled a new white paper, “Rare Disease Companies in the Public Markets: Challenging Performance Against a Backdrop of Policy Uncertainty.” The study analyzes the financial risks of rare disease drug development and examines the impact of policies and proposals on critical capital investment in rare disease research and development.

“Rare and non-rare are two different universes,” said Neal Masia, CEO of Health Capital Group and author of the new white paper.  “There are a lot of policy risks fueling investor concerns.”

The event brought together the policy, patient advocacy, investor, and industry communities for a panel discussion on the significance of the recent decline in rare disease funding and the urgency of acting to protect and build upon the progress made over the past 40 years since the adoption of the Orphan Drug Act (ODA).

“Even the smallest shift in public policy can leave our loved ones with no hope for treatment,” said Dorothea Lantz, Director of Community Engagement at the Prader-Willi Syndrome Association. “As a rare disease community, we understand the costs and the risk of rare disease research and development.”

Panelists agreed that policy plays an integral role in incentivizing investment in rare disease R&D and that recent fluctuations have made it difficult for companies to access critical capital. The study highlights this struggle, demonstrating that the market value of rare disease companies declined by nearly 7% per year over the past five years, versus an annual decline of 1.3% for non-rare disease companies. If a more stable policy environment does not prevail, rare disease companies will continue struggling to survive. 

“Investors are forward-looking. They are thinking about what the environment is going to look like 10 years from now, and they’re pretty good at connecting the dots,” said Ritu Baral, Managing Director, Senior Biotechnology Analyst at Cowen and Company. “And what can happen to turn this around? Incentives are needed to provide a buffer for current laws.”

Concerns about the policy environment have already impacted rare disease company valuations and access to capital. Rare disease companies saw nearly $10 billion less in investment available for research in 2022, stemming from decreases in venture capital investments, the IPO market, and partnership revenues. Venture capital investments in rare disease were down over 41% in 2022 and rare disease IPOs were down 81%, marking a substantial reduction of investment in rare disease companies. In a period of such financial uncertainty, seemingly small policies and proposals can have hugely consequential impacts on the ability for rare disease companies to discover, develop, and deliver potentially life-changing treatments to patients.

“Research and development is the only way that our families are going to have a chance at a normal life,” continued Lantz. “We are here to make our voices heard, not just for Prader-Willi Syndrome, but for the whole rare disease community. We hope policymakers are listening.”


Videos of Hill Briefing on October 25, 2023

A Setback in Investment for Continued Research & Development for the Rare Disease Community

Posted June 30, 2023

CMS today released revised guidance on the Inflation Reduction Act Medicare Drug Price Negotiation Program, clarifying that a drug with more than one designation for a rare disease or condition will not qualify for the Orphan Drug Exclusion, even if the drug has not been approved for any additional indications. Unfortunately, by making orphan products with a second designation eligible for drug price negotiation, this provision will disincentivize further investment in rare disease research and development. 

Why it’s important: This policy will impact the rare disease drug development pipeline for decades to come, increasing health disparities for the 30 million Americans living with rare, debilitating diseases, 50% of which affect children.

Undermining the bipartisan Orphan Drug Act (ODA): The ODA was enacted to incentivize the development of drugs for rare diseases and has demonstrated overwhelming evidence of success.

The final word: The RDCC is disappointed in CMS’s revised guidance and remains committed to working with stakeholders in the Administration and Congress to find ways to mitigate the impact of this policy on Americans living with rare diseases.

The Rare Disease Company Coalition (RDCC) supports Cameron’s Law and the Rare Disease Clinical Trial Pandemic Disruption Act (now called Leo’s Law), two important bills reintroduced this week by Representatives Josh Gottheimer and Don Bacon in honor of Rare Disease Day. These legislative proposals would help to close the innovation gap for rare disease research by encouraging more upfront investment in R&D and by promoting flexible clinical trials that encourage the pursuit of promising treatment options for rare disease patients.  

   

40 years ago, the Orphan Drug Act (ODA) recognized the critical need to provide incentives for rare disease research. The RDCC has called on policymakers to preserve policies that will continue to attract and maintain long-term investment in the development of advanced treatments and diagnostics for rare diseases, and the reintroduction of both these bills is a critical step toward this goal.

Specifically, Cameron’s Law would restore the tax credit for clinical testing expenses from 25% back to the original 50%. Increasing the tax credit to its original parameters would make it more economically feasible for drug companies to develop rare disease treatments and provide needed certainty for continued investment.     

The ODA also established the importance of exclusivity for drugs with orphan designation as a critical incentive for rare disease drug development. The Rare Disease Clinical Trial Pandemic Disruption Act (now called Leo’s Law) would extend exclusivity for rare disease clinical trials stalled during the pandemic and ensure promising cures get to the patients who need them.

The RDCC thanks Representatives Josh Gottheimer and Don Bacon for their bipartisan leadership that will directly benefit the lives of the one in 10 people living with a rare disease—half of whom are children. As a coalition of life science companies committed to discovering, developing, and delivering rare disease treatments for the patients we serve, the RDCC is eager to further engage and use our #OneRareVoice to move these bills forward in the 118th Congress.

The Rare Disease Company Coalition today issued the following statement from Executive Director Amanda Malakoff in response to the three payment models released by Health and Human Services for testing by the Centers for Medicare & Medicaid Services. The proposal, which aims to reduce Medicare spending for some accelerated approval drugs, undermines the intent of the pathway and mischaracterizes the extent of sponsors’ clinical research. Any attempt at indication-based pricing by the US government would have enormous impacts on rare disease patients, as well as on the companies investing in critical research.


Amanda Malakoff, Executive Director, Rare Disease Company Coalition

The FDA’s accelerated approval pathway recognizes that a “one-size-fits-all” traditional model for the approval of new medicines cannot always work – especially for rare diseases. This well-established, proven, scientifically rigorous path forward enables rare disease companies to make promising new and improved treatments available to patients at the earliest possible point in time for use in life-threatening and serious rare diseases.

2023 marks the 40th anniversary of the Orphan Drug Act (ODA) and kicks off a year of renewed hope and promise for developing rare disease medicines.

WASHINGTON, DC – January 3, 2023 – As 2023 marks the 40th anniversary of the Orphan Drug Act (ODA), the Rare Disease Company Coalition (RDCC) recognizes the advances made over the past four decades and calls on Congress to renew its support for policies that promote investment and innovation in rare disease treatments.

The ODA has spurred biopharmaceutical  companies to pursue treatments that would otherwise be financially unfeasible. ODA policies reduce the risk associated with rare disease research and development (R&D) and incentivize companies to discover new treatments. Since its enactment in 1983, the number of orphan drugs approved by the Food and Drug Administration (FDA) has increased by 1,576% – from just 38 to more than 600 treatments for more than 1,000 rare diseases. Today, RDCC members collectively have over 200 promising rare disease treatments currently in development.

As we recognize the ODA’s role in incentivizing biomedical innovation, we also acknowledge that the vast majority of rare disease patients still lack any FDA-approved treatment options, necessitating urgent policy considerations to bring hope to the 1 in 10 Americans suffering from a rare disease.

“Over the past 40 years, the ODA has established a strong foundation for the biotech community to invest in and develop new medicines for rare diseases,” says Amanda Malakoff, Executive Director, Rare Disease Company Coalition. “We need to build on that strong foundation so we can ensure continued innovation for the 95% of rare diseases that currently have no approved treatments.”

Researchers and developers rely on steady and supportive policy and regulatory environments to reduce the risk of investing in new treatments for small patient populations. While the ODA has supported a stable environment for the past 40 years, recent policy changes threaten ODA-established incentives like the Orphan Drug Tax Credit. New one-size-fits-all policy proposals fail to fully address the nuances of rare disease research and development and would drastically undermine this progress.

“When you look beyond rare diseases that are currently being treated or have the potential for new therapies, you see millions of people who now have hope thanks to the ODA,” said Nevan Charles Elam, JD, Chief Executive Officer & Founder, Rezolute Bio, a member of the RDCC. “While the disease may be rare, the people are not, and without the ODA, the incentive to research and develop new therapies for patients in need would simply not be there.” 

Continued progress requires policymakers be active and intentional about enacting and protecting policies that build on 40 years of the ODA. The RDCC calls on policymakers to:

“In recognizing the 40th anniversary of the ODA, it is more important than ever for policymakers, patients, patient advocacy groups, providers, researchers, and manufacturers to come together and reaffirm their commitment to the rare disease community by showing steadfast support for upholding and strengthening the ODA,” Malakoff concludes.

To learn more about the Orphan Drug Act and policy priorities from the Rare Disease Company Coalition, please visit www.rarecoalition.com

About the Rare Disease Company Coalition (RDCC)

Founded in May 2021, the Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes so that critical innovation can continue and positive changes can be enacted for the rare disease community. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Washington, DC – June 6, 2022 – The Rare Disease Company Coalition (RDCC), a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, recently welcomed Amanda (Schechter) Malakoff as its next executive director. Malakoff started in her new role May 31, 2022, and will be a key spokesperson for the RDCC and an advocate for companies dedicated to rare disease treatment development.

“Amanda brings a terrific blend of experience in association management and working on member companies’ legislative and regulatory issues,” said John Jackimiec, chair of the RDCC. “She will play a critical role in extending the reach of our coalition and advancing the policy priorities of the RDCC through our education and advocacy work.  We are all looking forward to her leadership and contributions.”

Prior to joining the RDCC, Malakoff was senior director of member relations for the National Association of Manufacturers (NAM), where she was responsible for representing the policy interests of mid-size manufacturing companies and advising industry executives on government relations strategies. Malakoff previously held client relations roles at Bloomberg Government and the Advisory Board Company, working with pharmaceutical and healthcare leaders on legislative and regulatory monitoring as well as operational best practices. She also worked on Capitol Hill in the office of Congressman John Carney, Jr. Malakoff will graduate in 2023 with a master’s in public administration from the George Washington University’s Trachtenberg School of Public Policy and Public Administration with a focus in regulatory policy.

With a diverse and growing membership, the RDCC informs key policy stakeholders and members of Congress on the unique considerations of life science companies when developing and manufacturing rare disease therapies as well as the importance of improving patient access following U.S. Food & Drug Administration (FDA) treatment approval. The Coalition believes that constructive dialogue with well-informed policymakers will lead to policies and regulations that enable continued innovation and the timely development and commercialization of treatments for the one in 10 Americans living with rare diseases, half of whom are children.

“I am thrilled to join the RDCC during this pivotal time of growth and impact, and honored to step into the position of executive director,” said Amanda Malakoff, executive director of the RDCC. “With more than 90% of rare diseases without a viable treatment, there is an urgent opportunity to advocate for the needs of life science companies working to deliver hope to patients.”

Over the past year, the RDCC has experienced growth in both size and scope. Collectively, RDCC represents 21 company members, totaling $12.4 billion in research and development investments for rare disease treatments. The companies are united around a shared mission to improve the lives of people living with rare diseases.

About the Rare Disease Company Coalition (RDCC)

Founded in May 2021, the Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes so that critical innovation can continue and positive changes can be enacted for the rare disease community. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:

Anna Stallmann
media@rarecoalition.com
708-476-1258

WASHINGTON – February 3, 2022 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced appointments to its Executive Committee for 2022, serving a one-year term.

With a diverse and rapidly growing membership, the Coalition informs key policy stakeholders and members of Congress on the unique considerations of life science companies when developing and manufacturing rare disease therapies as well as the opportunities for improving patient access following FDA treatment approval. The Coalition believes that constructive dialogue with well-informed policymakers will lead to policies and regulations that enable continued innovation and the cost-effective and timely development and commercialization of treatments for the one in 10 Americans living with rare diseases, half of whom are children.

The Coalition is governed by a Board of Directors and supported by an Executive Committee that provides oversight of the day-to-day operations of the Coalition. In addition to named elected positions, the Executive Committee has representation from both commercial and pre-commercial Coalition members. 

The following company representatives were elected by the Board of Directors to serve as Executive Committee members for the 2022 calendar year of the Coalition:

  • Chair: John Jackimiec, Aeglea BioTherapeutics
  • Vice Chair: Deirdre Parsons, Alnylam Pharmaceuticals
  • Secretary: Betsy Ricketts, Ultragenyx
  • Member-at-Large: Diane Berry, Sarepta Therapeutics
  • Member-at-Large: Christine Harrison, Orchard Therapeutics

“I am honored to represent the Rare Disease Company Coalition as we embark on our second year and build on our successes from 2021,” said John Jackimiec, newly appointed Chair of the Rare Disease Company Coalition. “In 2022, the Coalition can maximize our impact through an increase in membership, improving our reach at both the national and state level to ensure that national- and state-level policies support continued innovation for the millions of patients and families affected by rare diseases.”

In addition, the following individuals have been appointed Committee Chairs for 2022: 

  • Federal Policy Committee Co-Chairs: Katie Jones, Alexion Pharmaceuticals; Geoff Werth, Harmony Biosciences 
  • State Policy Committee Chair: Kate Segal, Sarepta Therapeutics
  • Communications Committee Chair: Jessi Rennekamp, Agios Pharmaceuticals
  • Operations Committee Chair: Bruce Bloom, Healx

To learn more about the Coalition and the need for informed policy and legislative discourse and actions related to rare disease treatment development and commercialization, please visit rarecoalition.com.

About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Coalition Welcomes New Company Members Healx and Zogenix, Inc.

WASHINGTON, D.C., – December 15, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced the addition of two companies to the Coalition. Following its launch in May of 2021, the Coalition is rounding out its inaugural year boasting a total membership roster of 21 companies. 

New members joining the Rare Disease Company Coalition are:

The Coalition strives to inform key policy stakeholders and members of Congress on the unique considerations of life science companies with respect to the development and manufacture of rare disease therapies as well as access and reimbursement following approval. As the federal government continues to consider policy reforms that could impact the ability of companies to innovate and develop groundbreaking treatments, the work of the Coalition and its member companies is essential on behalf of the one in 10 Americans living with rare diseases whose needs often go unmet. 

“As we conclude an incredible inaugural year, we welcome two new members to our coalition and remain fiercely dedicated to our work ahead as we continue to facilitate ongoing education and engagement with policymakers about the needs of companies dedicated to the challenging work of developing treatments for rare diseases,” said Betsy Ricketts, Chair of the Rare Disease Company Coalition. “These efforts would not be possible without such an impressive group of life science companies with whom we are grateful to be working. The foundation we have established has us optimistic about the opportunities for continued member growth and greater understanding of rare disease.”

Life science companies that develop treatments for rare disease patients face numerous challenges that include smaller patient populations, variable disease progression or limited understanding of progression, and lack of consensus around meaningful clinical endpoints. As a result, a comparatively higher percentage of operating expenses is dedicated to research and development at these life science companies. In 2020, Rare Disease Company Coalition members invested more than $4.5 billion in research and development, representing on average more than half of their annual operating budgets, with over 225 treatment development programs in process.

This necessary commitment to research and development underscores the importance of supportive policy and regulatory frameworks that can foster a greater understanding of rare diseases and recognizes the distinct considerations associated with the development of and access to rare disease treatments. 

To learn more about the Coalition and the need for informed policy and legislative discourse and actions related to rare disease treatment development and commercialization, please visit rarecoalition.com.

About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:

Anna Stallmann
media@rarecoalition.com
708-476-1258

WASHINGTON – November 17, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, welcomes the introduction of the bipartisan “Cures 2.0,” legislation by Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) and encourages further engagement with Congressional leaders as it advances through the committee process.

“We applaud the continued leadership by Congresswoman DeGette and Congressman Upton to build on the success of the 21st Century Cures Act, and advance legislation that can better deliver modern treatments and cures to the countless Americans and rare disease patients that await options. As a diverse coalition representing life science companies dedicated to developing and delivering treatments for rare diseases, we look forward to continuing to work with Congressional stakeholders to provide feedback on the unique challenges and opportunities to unleash the promise of our R&D efforts aimed at tackling some of the most complex and devastating rare diseases,” said Betsy Ricketts, Chair of the Rare Disease Company Coalition.

About

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258

Coalition Calls on Congress to Not Stall Medical Progress for Those Urgently Awaiting Rare Disease Treatment Development  

WASHINGTON – November 3, 2021 The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today issued the following statement condemning the proposed legislation to amend the Orphan Drug Tax Credit included in the Build Back Better Act, citing the urgent need for continued investment in research and development for the 93 percent of rare diseases that do not have a single treatment available. 

“The language released today by the House Budget Committee is disappointing. We have come to the table with solutions and options to address or alleviate potential concerns related to the Orphan Drug Tax Credit,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “Unfortunately, the language provided today puts politics over patients. We will continue to fight to ensure continued innovation remains viable for those desperately seeking access to lifesaving treatments, and we hope those in Congress put politics aside, come to the table and do what is right.” 

For more information, please find the following resources available:

The 1983 Orphan Drug Act was passed and built upon to help incentivize investment in rare disease research and therapeutic development where there was little research being done and nearly no treatment options for those diagnosed with a rare disease. Over the last several decades, the Act has proven to be a universal success, enabling life science companies to address a growing number of unmet needs for people living with rare diseases that once were untreatable. Since implementation of the Orphan Drug Act in 1983, there have been more than 1,000 FDA approvals for rare disease treatments, with over 25 percent of those approvals occurring in the last three years. The Orphan Drug Tax Credit, already diminished in 2017 under the Tax Cut and Jobs Act from 50 percent to 25 percent, remains a critical program for sustained innovation and investment for innovator companies that exclusively focus on life-changing development programs for treatments for rare diseases. 

The Rare Disease Company Coalition was established in 2021 to help advance policies and regulations that enable the cost-effective and timely development and commercialization of rare disease treatments by educating policymakers on the distinct considerations of life science companies operating in the rare disease space. To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.

About

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:

Anna Stallmann
media@rarecoalition.com
708-476-1258

Coalition Welcomes Amryt Pharma, Fulcrum Therapeutics and Signature Biologics, LLC

WASHINGTON – October 12, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced the addition of three new companies to the Coalition. Launching in May 2021, the Coalition now boasts a total membership roster of 18 companies and provides a unified voice for innovator companies dedicated to rare disease treatment development. 

New members that join the Rare Disease Company Coalition include:

  • Amryt Pharma
  • Fulcrum Therapeutics
  • Signature Biologics, LLC

As Congress considers policy reforms that could impact the ability for companies to develop treatments for the one in ten Americans living with rare disease and have extraordinarily high unmet needs, the Coalition continues to help inform policymakers on the unique circumstances impacting therapy development for the 93% of rare diseases that have no FDA-approved treatments. 

“We welcome the three new members to our Coalition, and applaud their commitment to developing rare disease treatments through ongoing research and development while often taking on significant risk and uncertainty,” said Betsy Ricketts, Chair of the Rare Disease Company Coalition. “The sustained growth of the Coalition is reflective of the need for ongoing education and engagement with policymakers on the importance of well-informed policy that can support continued progress for developing treatments for people with rare diseases.” 

Life science companies that develop treatments for rare disease patients face numerous challenges from clinical trial enrollment through proving meaningful endpoints given the smaller population sizes and the more limited knowledge known about the disease progression. As a result, a comparatively higher percentage of operating expenses is dedicated to research and development at these life science companies. In 2020, Rare Disease Company Coalition members invested more than $4.1 billion in research and development, representing on average more than half of their annual operating budgets. 

This necessary commitment to research and development underscores the importance of supportive policy and regulatory frameworks that can foster a greater understanding of rare diseases and recognizes the distinct considerations associated with the development of and access to rare disease treatments. 

To learn more about the Coalition and the need for informed policy and legislative discourse and actions related to rare disease treatment development and commercialization, please visit rarecoalition.com.

About

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:

Anna Stallmann

media@rarecoalition.com

708-476-1258

WASHINGTON – September 14, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today issued the following statement criticizing the draft legislation to amend the Orphan Drug Tax Credit included in the Build Back Better Act which will be considered in the U.S. House of Representatives Committee on Ways and Means this week. 

“As one of America’s most underserved medical populations with extraordinarily high unmet medical needs, people living with rare diseases simply cannot shoulder the burden of American economic recovery and infrastructure development, and it is disappointing that Congress continues to advance policies that would limit their access to life-changing therapies,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “The proposed changes to the Orphan Drug Tax Credit would directly limit the ability for innovator companies to invest in research and development for the nearly 30 million people battling debilitating and potentially fatal rare diseases, of which 93 percent do not have available treatments. The Rare Disease Company Coalition strongly urges Congress to consider the devastating impact of this legislation.”

The 1983 Orphan Drug Act was passed and built upon to help incentivize investment in rare disease research and therapeutic development where there was little research being done and nearly no treatment options for those diagnosed with a rare disease. Over the last several decades, the Act has proven to be a universal success, enabling life science companies to address a growing number of unmet needs for people living with rare diseases that once were untreatable. Since implementation of the Orphan Drug Act in 1983, there have been more than 1,000 FDA approvals for rare disease treatments, with over 25 percent of those approvals occurring in the last three years. The Orphan Drug Tax Credit, already diminished in 2017 under the Tax Cut and Jobs Act from 50 percent to 25 percent, remains a critical program for sustained innovation and investment for innovator companies that exclusively focus on life-changing development programs for treatments for rare diseases.

The Rare Disease Company Coalition was established in 2021 to help advance policies and regulations that enable the cost-effective and timely development and commercialization of rare disease treatments by educating policymakers on the distinct considerations of life science companies operating in the rare disease space. To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.

About

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:

Anna Stallmann

media@rarecoalition.com

708-476-1258

WASHINGTON – August 12, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today issued the following statement in response to President Joe Biden’s remarks outlining a policy proposal that would allow Medicare to negotiate the price of certain prescription drugs. 

“Ninety-three percent of rare diseases have no viable, FDA-approved treatment option. President Biden’s remarks calling on Congress to adopt price controls for drugs that have no competition in the market create the wrong incentives for further innovation to treat these devastating diseases,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “Research and development for rare disease treatments present unique challenges and require substantially different business models than therapies for larger patient populations. We, as rare disease companies, are committed to working with Congress to address patient affordability, but a policy that seemingly targets innovators who are bringing medicines to patients with high unmet need seems unworkable and, worse yet, could mean fewer innovative medicines for the patients who need them most.”

The Rare Disease Company Coalition was established in 2021 to help advance policies and regulations that enable the cost-effective and timely development and commercialization of rare disease treatments by educating policymakers on the distinct considerations of life science companies operating in the rare disease space. To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.

About

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:

Anna Stallmann

media@rarecoalition.com

708-476-1258

WASHINGTON – July 29, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today sent a letter to the leadership of the U.S. Senate Health, Education, Labor & Pensions (HELP) Committee and the U.S. House of Representatives Energy & Commerce Committee. In this letter, the Coalition expresses support for the Food and Drug Administration’s (FDA) Accelerated Approval Program while citing concerns with recent policy recommendations put forth by the Medicaid and CHIP Payment and Access Commission (MACPAC).

“The accelerated approval pathway is a critical mechanism that enables expedited availability of innovative treatments for patients with serious and often life-threatening diseases, including rare diseases that may be too challenging to study using a traditional pathway. In these cases, accelerated approval provides a promising roadmap to make possible the delivery of safe and effective rare disease therapies without compromising FDA’s stringent, science-based approval standards,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “As a coalition of companies dedicated to rare diseases, we support policies that preserve the intent of the accelerated approval pathway and give all patients who have debilitating diseases a chance to be treated with cutting edge science. The Coalition urges members of Congress to recognize the harmful consequences of undercutting reimbursement, coverage and patient access to therapies developed under the Accelerated Approval Program.”

Throughout the letter, the Rare Disease Company Coalition highlights how the accelerated approval pathway has enabled the timely delivery of innovative treatments, particularly oncology drugs, and provides a proven regulatory framework that can appropriately enable access to therapies for challenging rare disorders, many of which might not be developed otherwise. The Coalition cites ill-informed policy proposals, including the recent recommendations put forth by MACPAC that selectively target accelerated approval drugs as a threat to patient access and the development of rare disease treatments. 

The full text of the letter may be found here. A recent op-ed on the importance of the Accelerated Approval Program for rare disease treatment written by Dr. Emil D. Kakkis, founder of Ultragenyx Pharmaceutical and a company member of the Rare Disease Company Coalition, can be found here. 

About

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:

Anna Stallmann

media@rarecoalition.com

708-476-1258

Coalition Welcomes Atara Biotherapeutics, Chiesi Global Rare Diseases, Horizon Therapeutics, and Stealth BioTherapeutics 

WASHINGTON – June 30, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced the addition of four new companies to the Coalition. With a total membership roster of 15 companies, the Coalition continues to articulate its voice on the importance of well-informed policies and regulations to enable the cost-effective and timely development and commercialization of rare disease treatments for patients. 

New members that join the Rare Disease Company Coalition include:

  • Atara Biotherapeutics
  • Chiesi Global Rare Diseases
  • Horizon Therapeutics
  • Stealth BioTherapeutics

“We are pleased to welcome four new companies to our growing Coalition,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “The Coalition is committed to fostering greater understanding of the unique circumstances associated with the development of rare disease treatments so that legislation and policies can better support continued development of these therapies as well as patient access and reimbursement to address the incredible unmet need of rare disease patients.” 

Life science companies that develop treatments for rare disease patients face numerous challenges from clinical trial enrollment through proving meaningful endpoints given the smaller population sizes and the more limited knowledge known about the disease progression. As a result, a comparatively higher percentage of operating expenses is dedicated to research and development at these life science companies. In 2020, Rare Disease Company Coalition members invested more than $4.6 billion in research and development, representing on average more than half of their annual operating budgets. 

This necessary commitment to research and development underscores the importance of supportive policy and regulatory frameworks that can foster a greater understanding of rare diseases and recognizes the distinct considerations associated with the development of and access to rare disease treatments. 

To learn more about the Coalition and the need for informed policy and legislative discourse and actions related to rare disease treatment development and commercialization, please visit rarecoalition.com.

About

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258

WASHINGTON – June 22, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, commented on the release of “Cures 2.0” draft bipartisan legislation by U.S. Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) and welcomes the opportunity to further engage and provide feedback as the legislation is considered.  

“The Rare Disease Company Coalition applauds Congresswoman DeGette and Congressman Upton on recognizing the importance of building upon their landmark, bipartisan 21st Century Cures Act and continuing to advance legislation that aims to better deliver modern treatments and cures to the countless Americans seeking options,” said Betsy Ricketts, Chair of the Rare Disease Company Coalition. “As Congress now considers ‘Cures 2.0’ and other legislative and regulatory proposals, we look forward to further engaging and providing feedback on the unique challenges in the development of rare disease treatments so that forthcoming legislation and policies facilitate and do not inadvertently harm the ability to successfully provide treatments for these medically underserved patient populations, now and in the future.”

The Rare Disease Company Coalition was established in 2021 to help advance policies and regulations that enable the cost-effective and timely development and commercialization of rare disease treatments by educating policymakers on the distinct considerations of life science companies operating in the rare disease space. To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.

About

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258

 For Immediate Release

WASHINGTON – June 8, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced appointments to its Executive Committee for 2021, following the Coalition’s launch on May 13, 2021.

The Rare Disease Company Coalition was established to educate policymakers on the distinct considerations of life science companies operating in the rare disease space. The Coalition believes that constructive dialogue with well-informed policymakers will lead to policies and regulations that enable continued innovation and the cost-effective and timely development and commercialization of resulting treatments for the one in 10 Americans living with rare diseases. 

The Coalition is governed by a Board of Directors and supported by an Executive Committee that provides oversight of the day-to-day operations of the Coalition. In addition to named elected positions, the Executive Committee has representation from each of the membership classes of the Coalition. 

The following company representatives were elected to serve as Executive Committee members for the inaugural year of the Coalition: 

  • Chair: Betsy Ricketts, Ultragenyx Pharmaceutical 
  • Vice Chair: Greg Chesmore, Acceleron Pharma 
  • Secretary: Tim Kanter, Orphazyme US 
  • Member-at-Large: Christine Harrison, Orchard Therapeutics 
  • Member-at-Large: Deirdre Parsons, Alnylam Pharmaceuticals 
  • Member-at-Large: Geoff Werth, Harmony Biosciences 

Supporting the work of the Coalition, Taylor Mason was appointed to serve as Executive Director of the Coalition. In addition, the following individuals have been appointed Committee Chairs for 2021: 

  • Policy Committee Chair: Diane Berry, Sarepta Therapeutics 
  • Operations Committee Chair: John Jackimiec, Aeglea BioTherapeutics 

To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.

About 
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com.

Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258

For Immediate Release

With one in 10 Americans living with an often devastating and debilitating rare disease and limited treatment options, a coalition of life science companies cites a “one-size-fits-all” approach to healthcare policy and regulations as detrimental to continued innovation that has potential to systematically improve quality of life for millions

WASHINGTON, DC – May 13, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced their organizational launch. This first-of-its-kind alignment of organizations will inform and help educate policymakers on the unique circumstances facing life science companies when developing and bringing to market therapies for very small and differentiated patient populations. The Coalition will also advocate for policies and regulations that enable cost-effective and more timely delivery of treatments by recognizing these differences.

Collectively, the Rare Disease Company Coalition represents life science companies that have brought to market 22 treatments and are presently working on more than 160 rare disease development programs, many of which would be first-to-market therapies if approved. These diseases are devastating and often life-threatening: 80 percent of rare diseases are genetic in origin, 50 percent impact children, and 30 percent of those children won’t live to see their 5th birthday. The Coalition is composed of companies continuing to change those statistics by discovering, developing and bringing valuable treatments – and even potential cures – to market for these patients awaiting a treatment approved by the Food and Drug Administration (FDA).

The pharmaceutical industry continues to lead the nation in research and development expenditures by dedicating on average between fifteen to twenty percent of sales to fostering innovation. Companies focused on rare diseases take on an outsized commitment to and risk in research and development. In 2020, Rare Disease Company Coalition members invested more than $4.1 billion in research and development, representing approximately 65 percent of their annual operating budgets. Moreover, the majority of Coalition members spend more annually in research and development than revenues generated, including the over one-third of members that are in the preclinical stage and currently do not generate revenue.

Building on the results of these capital investments, smaller companies are now driving the groundbreaking innovation happening across multiple disease areas. As reported by the Congressional Budget Office, 70 percent of the nearly 3,000 therapies in Phase III clinical trials are from small drug companies or those with annual revenues of less than $500 million. Moreover, about one-third of new treatments approved by the FDA have been developed by companies with annual revenues of less than $100 million.

Expanding on years of advocacy work through successful industry associations and rare disease patient-focused organizations, the Rare Disease Company Coalition will specifically work to:

  • Inform and help educate policymakers on the unique circumstances of the rare disease company business model, specifically focusing on the different issues created by working with small and differentiated patient population sizes;
  • Advocate for public policy and regulatory frameworks that account for and recognize the distinct considerations of life science companies operating in the rare disease space;
  • Work with policymakers to establish long-term, consistent, equitable and sustainable research incentives for rare diseases and access to the resulting innovation;
  • Increase awareness of the innovation happening within rare disease companies.

Founding members of the Coalition include:

  • Acceleron Pharma
  • Aeglea BioTherapeutics
  • Agios Pharmaceuticals
  • Alnylam Pharmaceuticals
  • Harmony Biosciences
  • Orchard Therapeutics
  • Orphazyme US, Inc.
  • Sarepta Therapeutics
  • Taysha Gene Therapies
  • Ultragenyx Pharmaceutical

In the coming month, the Coalition will engage with policy stakeholders on potentially impactful drug and healthcare policies and regulations currently under discussion, including prescription drug pricing, to highlight the consequences that blanket legislation can have on continued innovation for rare disease treatments.

About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com

Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258