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