Washington, D.C. – Last week, the Rare Disease Company Coalition (RDCC), on behalf of 29 leading rare disease biotech companies, sent a letter to Department of Health and Human Services Secretary Robert F. Kennedy Jr. and the Trump Administration. The RDCC called on the Administration to prioritize the urgent needs of the 30 million Americans affected by rare disease and support policies that increase innovation and access to life-changing treatments.
The RDCC offers the following policy recommendations to ensure the 1 in 10 Americans living with a rare disease have access to safe and effective therapies:
The RDCC is ready to serve as a resource to policymakers to create a brighter and healthier future for American families impacted by rare diseases.
Read the full letter below.
By Delman Lebel, head of U.S. Corporate Affairs at Alexion, AstraZeneca Rare Disease and Chair of the Rare Disease Company Coalition
Finding cures for rare diseases is a fundamentally nonpartisan pursuit. That’s why members of the Rare Disease Company Coalition (RDCC) are calling on our elected leaders to seize this unique opportunity for bipartisanship, reach across the aisle and unite on common-sense legislation to offer hope to millions of Americans living with rare diseases.
Right now, over 30 million Americans, many of whom are children, live with a rare disease. These aren’t abstract medical conditions; they’re the daily realities for families facing diagnoses that too often carry a devastating prognosis – and a glaring lack of treatment options.
A staggering 95% of these 10,000 diseases lack FDA-approved therapies. Diagnosis can take years, and during that time the disease can quickly progress. For those in need of treatment the clock is ticking, and the key to progress lies with our lawmakers.
As Chair of the RDCC, an advocacy-focused coalition representing life science companies committed to discovering, developing, and delivering rare disease treatments for patients, I understand the unique challenges that come with advancing rare disease therapies. Developing treatments for rare diseases involves creating animal models from scratch, difficulty in recruiting participants for clinical trials, and navigating high costs and risks. It often takes over a decade of sustained investment to move from bench to bedside. Because each disease affects a small population, traditional market incentives are insufficient. This is why a robust and supportive ecosystem with key incentives is crucial. To foster this kind of ecosystem, we need the support of Congress.
Three bipartisan, common-sense legislative priorities offer a concrete path forward to ensure scientific breakthroughs continue and the roughly 30 million Americans living with a rare disease are not left behind.
First, we need to remove barriers that hinder scientific progress, such as the disincentive within the Inflation Reduction Act (IRA) that hinders rare disease innovation. The IRA’s Orphan Drug Exclusion provides a price negotiation exemption for orphan drugs that treat only one orphan, or rare, condition. However, 1 in 5 orphan drugs are FDA-approved for more than one use, and 60% of those second indications are for another rare disease. This unnecessary hurdle discourages companies from further pursuing promising research that could lead to additional treatment options. The ORPHAN Cures Act would expand the single-orphan exclusion, allowing companies to follow the science and invest in promising treatments using existing research. This means more treatment possibilities would be within reach.
Second, we must preserve incentives to develop therapies for rare diseases. The rare pediatric disease priority review voucher (PRV) program has been a proven success, encouraging drug development for rare pediatric diseases by expediting the regulatory process for qualifying products. Since its inception, the PRV program has led to the approval of dozens of life-changing therapies for children, benefiting over 200,000 patients and addressing high unmet needs across 47 rare pediatric indications. However, as of December 20, 2024, the PRV Program has lapsed and begun to expire. Without urgent Congressional action, this incentive, along with hope for millions of children, may vanish for good.
Finally, we must restore the Orphan Drug Tax Credit (ODTC) to its original value. This credit is essential for many small biotech companies leading rare disease research. It promotes research by lowering development costs for manufacturers, reducing the significant financial cost and risk that comes with creating new therapies for small patient populations.
The ODTC was cut in half in 2017 and we are already seeing a chilling effect, making it harder for these companies to pursue potentially life-saving research. 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. Restoring the credit isn’t just smart policy; it’s a moral imperative. Without it, many promising projects will simply never get off the ground, leaving patients with little hope for treatment.
These aren’t just legislative checkboxes; they are essential to preserve the delicate ecosystem of rare disease research and development, ensuring that scientific progress can continue despite the challenges. They create hope for families who have spent far too long searching for answers.
Policymakers have a chance to make a real difference, to show the American people that bipartisanship isn’t dead and that progress is still possible. By prioritizing these common-sense measures, our lawmakers can send a powerful message: the rare disease community matters, their struggles are seen, and healthier futures for patients living with a rare disease are worth fighting for.
Cameron’s Law Would Restore the Critical Orphan Drug Tax Credit (ODTC), Enabling Future Innovation in Rare Disease Drug Development
Washington, DC – The Rare Disease Company Coalition (RDCC) supports H.R. 1414, Cameron’s Law, a critical bill that would restore the Orphan Drug Tax Credit (ODTC) to its original 50 percent. The RDCC thanks the bill sponsors, Representatives Josh Gottheimer (D-NJ-05), Don Bacon (R-NE-02), Jimmy Panetta (D-CA-19), and Tom Suozzi (D-NY-03) for their leadership on this important issue.
As Rare Disease Week approaches, Cameron’s Law will help spur rare disease research & development (R&D) encouraging more upfront investment in the pursuit of promising treatments for patients living with a rare disease.
The ODTC is a pillar of the Orphan Drug Act (ODA), a cornerstone of rare disease policymaking for over 40 years. Before the ODA was enacted in 1983, there were only 38 available treatments for rare diseases. Now, there are over 650 approved treatments – but with 95 percent of rare diseases still lacking an FDA-approved treatment, there is much more work to be done.
“For RDCC member companies developing life-changing treatments for rare diseases, it’s crucial to reduce the barriers to drug development and encourage upfront investment. Cameron’s Law will restore an integral component of the rare disease ecosystem and make it more economically feasible for drug companies to develop rare disease treatments and provide much-needed certainty for continued investment,” said Stacey Frisk, Executive Director of the Rare Disease Company Coalition. “The RDCC is proud to endorse this important bill and urges policymakers to ensure Cameron’s Law becomes law in the 119th Congress.”
The ODTC is a vital lifeline for rare disease patients. The tax credit promotes research for rare diseases by lowering qualifying development costs for manufacturers. It is an important incentive for companies working to find rare disease treatments, reducing the significant financial cost and risk that comes with the research and development of new therapies for small patient populations.
However, recent policy and regulatory changes – including the reduction of the ODTC by half in 2017 – have substantially reduced investments in rare disease companies. Recent research shows the trading index for rare disease companies has declined by nearly 7% per year over the last 5 years, compared to a 1.3% decline for non-rare disease companies. 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.
The journey from bench to bedside is longer and more challenging for rare disease treatments than mass-market drugs, yet rare disease companies remain industry leaders in R&D investment. Both clinical- and commercial stage rare disease companies invest over twice as much in R&D as their non-rare counterparts. To stay afloat, however, small and emerging rare disease companies often rely on venture capital and outside investment as they pursue promising treatments, which can take 3 to 5 years longer than mass-market drugs.
The RDCC thanks the bill sponsors for their bipartisan leadership that will directly benefit the lives of the 1 in 10 Americans living with a rare disease – half of whom are children. The RDCC urges policymakers to ensure Cameron’s Law becomes law in the 119th Congress.
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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
Crucial PRV Program Lapsed in December, Will Sunset Without Congressional Action. Give Kids a Chance Act Would Revive Program, Provide Necessary Incentives to Spur Research and Development of Rare Disease Treatments
Washington, DC – The Rare Disease Company Coalition (RDCC) applauds the introduction of the Give Kids a Chance Act, which would reauthorize the rare pediatric disease priority review voucher (PRV) program. The program began to lapse on December 20, 2024 and without urgent Congressional action, hope for millions of children may vanish for good. We thank Representative Michael McCaul (R-TX) for his leadership on this bill, and original sponsors, Representatives Gus Bilirakis (R-FL), Debbie Dingell (D-MI), Kim Schrier (D-WA), Diana Harshbarger (R-TN.), Kathy Castor (D-FL), Mike Kelly (R-PA), Doris Matsui (D-CA), Dan Crenshaw (R-TX), Lori Trahan (D-MA) and Randy Weber (R-TX), for their support.
The rare pediatric disease PRV program is critical to providing hope for the millions of children in the United States living with a rare disease, 30 percent of whom will not live to see their fifth birthday. The program has led to new innovations benefiting over 200,000 patients and addressing high unmet needs across 47 rare pediatric indications, at little to no cost to taxpayers.
“Since just before Christmas last year, millions of children and their families have faced uncertainty about the future of the lifesaving rare pediatric disease PRV program, a proven tool that has led to groundbreaking innovations in rare disease therapies,” said Stacey Frisk, Executive Director of the Rare Disease Company Coalition. “Small, emerging biopharmaceutical companies rely on the PRV program to stabilize investment in new treatments. Every minute the PRV program remains expired jeopardizes investment in critical innovations that could transform the lives of children living with rare, often fatal and progressively debilitating, diseases.”
Passage of the Give Kids a Chance Act would ensure that the rare pediatric disease PRV program will continue to benefit hundreds of thousands of patients living with a rare disease. The program has received broad, bipartisan, and bicameral support since its inception in 2012. Most recently in the 118th Congress, the program enjoyed unanimous passage through the House of Representatives and was included in the health title of the end-of-year package. Further, the Food and Drug Administration (FDA) has supported the reauthorization of the program via inclusion in their anomalies list. A diverse array of stakeholders, from Congress to the Administration, from patient advocates to academia, and from the investor community to rare disease innovators, understand that the PRV program is a proven innovation-driving and cost-effective policy that spurs research & development in rare pediatric diseases.
The Rare Disease Company Coalition applauds Congressman McCaul and all cosponsors for their leadership on the Give Kids a Chance Act, and urges Congress to act quickly to restore the rare pediatric disease priority review voucher program and ensure children can get access to the treatment they desperately 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
Without urgent Congressional action, hope for millions of children may vanish for good.
Washington, D.C. — Leaders from 19 biotechnology companies are urging Congress to support the swift reauthorization of the rare pediatric disease priority review voucher (PRV) program.
Immediate restoration of the rare pediatric disease PRV program is critical to achieving our important mission to address the unmet medical needs of the rare disease community. Programs like the rare pediatric disease PRV are crucial to mitigating the risks associated with investment in the rare disease space; without it, investors may view rare disease drug development as too risky – and that could make the difference between bringing a product to market and shuttering a program.
Children with rare, often fatal, and progressively debilitating diseases rely on this program in the hope of finding a treatment for their conditions. Every minute the PRV program remains expired further jeopardizes these children’s health and livelihood.
We cannot allow hope for these children to vanish for good. As leaders in the rare disease community, we urge policymakers to prioritize the reauthorization of this critical program as swiftly as possible.
Read the full letter below.
Bipartisan Bill Enables Rare Disease Companies to Pursue Promising Research to Uncover More Treatment Options
Washington, D.C. — The Rare Disease Company Coalition (RDCC) strongly supports the ORPHAN Cures Act (H.R.946) introduced by Representatives John Joyce (R-PA-13) and Don Davis (D-NC-01). The bipartisan legislation will help to encourage critical rare disease research and development (R&D) by addressing a disincentive within the Inflation Reduction Act (IRA) that has hindered 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, thereby discouraging rare disease companies from further exploring promising research that could lead to additional treatment options. In practice, however, 1 in 5 orphan drugs are FDA-approved for more than one use, and 60 percent of those second indications are for another rare disease.
The current limits imposed by the IRA have created a roadblock to rare disease innovation, and in turn, progress towards developing treatments for the more than 95 percent of rare diseases without approved therapies. The ORPHAN Cures Act would open the door for the research and development of existing products that could help find treatments by expanding the single-orphan exclusion.
“The ORPHAN Cures Act is common sense legislation that will remove unnecessary hurdles towards developing treatments for individuals with rare diseases. RDCC member companies understand the importance of investing back into research and development for rare diseases therapies, but in order to maximize the pursuit of promising research we must expand the IRA’s single-orphan exclusion,” said Stacey Frisk, Executive Director of the RDCC. “We thank Representatives Joyce and Davis for understanding the importance of delivering these desperately needed therapies to underserved patient and caregiver communities by introducing this legislation.”
Rare disease drug development is uniquely challenging, and the current policy does not reflect the unmet need for rare disease treatments. By ensuring developers can continue to direct much needed resources towards rare disease research, the ORPHAN Cures Act will build upon the 40 years of progress enabled by the Orphan Drug Act (ODA).
One in ten people live with a rare disease, and the ORPHAN Cures Act provides an opportunity to address the urgent need for innovation in rare disease R&D by breaking down barriers that are restricting biopharmaceutical companies from meeting the moment. The RDCC thanks Representatives John Joyce and Don Davis for leading the charge and Representatives Kevin Hern (R-OK-1), Scott H. Peters (D-CA-50), Mariannette Miller-Meeks (R-IA-1), William R. Keating (D-MA-9), Richard Hudson (R-NC-9), Shri Thanedar (D-MI-13), Gus M. Bilirakis (R-FL-12) and Josh Gottheimer (D-NJ-5) for co-sponsoring this legislation and supporting the rare disease community.
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Washington, DC – Rare Disease Company Coalition (RDCC) Executive Director Stacey Frisk released the following statement on the continuing resolution to fund the federal government, which includes a five-year extension of the life-changing rare pediatric disease priority review voucher (PRV) program.
The RDCC strongly urges Congress to advance the continuing resolution to support key programs like the rare pediatric disease priority review voucher (PRV).
The rare pediatric disease PRV program has already spurred extraordinary progress for pediatric rare disease treatments and supported groundbreaking innovation for patients. The RDCC’s latest research found that over the past decade, the program has led to new innovations that benefit over 200,000 rare disease patients and addressed unmet medical needs across 47 rare pediatric indications. Over 90 percent of all rare pediatric disease PRVs were awarded to therapies for indications with no approved therapy on the market.
Now, thanks to our champions on Capitol Hill, Congress has the opportunity to ensure innovative companies like our members can continue to discover, develop, and deliver treatments for children, while reinvesting critical dollars into additional R&D for rare disease treatments. Through the continuing resolution, Congress can bring stability to innovators, encourage further investment and create the conditions for groundbreaking innovations in rare pediatric disease drug development.
Read our letter to Congress here.
Washington, DC – With so much at stake for the 1 in 10 Americans living with a rare disease, the FDA’s Rare Disease Innovation Hub could spur the development of new innovations for the 95 percent of rare diseases with no FDA-approved treatment. It has incredible potential to increase collaboration with patients, leverage cross-agency expertise and advance regulatory science on behalf of individuals living with rare diseases; prioritizing the right activities will ensure that happens.
The comment letter below presents our feedback on the Hub’s priorities. We are confident that with these recommendations in mind, the FDA’s new model for rare disease collaboration can fulfill its mission and promise.
We look forward to continuing to work with FDA to support the Hub and our shared commitment to improving the lives of Americans living with rare diseases.
Read the full letter here.
Washington, DC – On September 12, the Rare Disease Company Coalition sent a letter to Congressional leadership urging them to support the swift passage of S.4583/H.R. 7384, the Creating Hope Reauthorization Act of 2024.
The bill would reauthorize the rare pediatric disease priority review voucher (PRV) program, a proven policy that has benefitted hundreds of thousands of patients living with a rare disease. Over the past 12 years, the program has been an innovation-driving and cost-effective policy that spurs research & development (R&D) in rare pediatric diseases.
If Congress allows it to expire on September 30, this critical program will be out of reach for many rare disease treatments.
Read the full letter below.
Washington, DC – The Rare Disease Company Coalition (RDCC) has submitted a response to the 21st Century Cures and Cures 2.0 Request for Information, offering recommendations for policy improvements to facilitate the discovery, development, and delivery of treatments for people living with a rare disease.
RDCC applauds the longstanding leadership of Rep. DeGette and Rep. Bucshon in developing and advancing policy solutions on behalf of the 1 in 10 Americans living with a rare disease. 21st Century Cures and Cures 2.0 yielded notable progress in advancing the development and availability of treatments for rare diseases, but there is a critical need for further action to address the unmet needs associated with rare disease. In particular, the RDCC recommends that the 21st Century Cures initiative ensures:
By supporting these initiatives, Congress can help create an environment where effective treatments for rare diseases are developed and delivered without delay. We look forward to working with policymakers to advance legislation to accomplish these goals.
Read the full letter here.
Washington, DC – The Rare Disease Company Coalition (RDCC), representing 24 life science companies committed to discovering, developing, and delivering treatments for individuals living with a rare disease, today issued the following statement from RDCC Executive Director Stacey Frisk in response to FDA’s announcement of a plan to establish the Rare Disease Innovation Hub (the Hub):
“At this critical moment for the future of rare disease research and development, the RDCC commends Dr. Patrizia Cavazzoni and Dr. Peter Marks and their colleagues at the FDA for their leadership in advancing a new model for coordination and collaboration to spur the development of new innovations for people with rare disease.
The RDCC looks forward to working with the FDA as it implements the Rare Disease Innovation Hub. With so much at stake for the 1 in 10 Americans living with a rare disease, it is critical that the FDA partner with a wide range of rare disease stakeholders to realize the full potential of the Hub’s vision. We are incredibly optimistic that, alongside stakeholders across the rare disease community, the FDA’s new Hub will increase collaboration with patients, leverage cross-agency expertise and advance regulatory science on behalf of individuals living with rare diseases.”
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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 Rare Disease Company Coalition (RDCC), with support from IQVIA, investigated the history and impact of the rare pediatric disease priority review voucher (PRV) program. The findings clearly demonstrate that this program is a proven incentive that has spurred the development of new innovations that benefit more than 200,000 patients across 47 rare pediatric indications. But while there remains an urgent unmet medical need for new treatments for rare pediatric diseases, the future of this vital tool is uncertain.
Without Congressional action, the program will begin to expire on September 30, 2024. Expiration of the rare pediatric disease PRV program would result in reduced incentives for the discovery of new treatments, jeopardize programs already under development, and destabilize investment in new treatments. Preservation is necessary to further investment in and development of new rare disease treatments.
The RDCC applauds Congress for the significant progress in improving the lives of children with rare disease through the creation and subsequent reauthorization of the Rare Pediatric Disease PRV program in 2012, 2016 and 2020, respectively. A long-term extension of the PRV program will support the stability of research & development and ultimately lead to new treatment advances for rare pediatric diseases.
Former Director of Regulatory Strategy at Sarepta Therapeutics, Frisk Brings Expertise on Rare Disease Policy
Washington, DC – The Rare Disease Company Coalition (RDCC) on Wednesday announced Stacey Frisk as Executive Director of the Coalition. Joining the RDCC from rare disease company Sarepta Therapeutics, Stacey has spent the past decade steeped in the issues that matter most to rare disease companies. She will help to further advance the RDCC’s mission to stimulate development of and access to rare disease treatments. The RDCC provides a unified voice for life science companies committed to discovering, developing, and delivering rare disease treatments.
“The RDCC plays a pivotal role in advancing policy that supports research and development of rare disease treatments, and I am thrilled to be joining them in furthering their mission to bring life-changing therapies for patients,” said Stacey Frisk, Executive Director of the RDCC. “I’m excited to continue my advocacy for policies that take into account the challenges associated with rare disease drug development and provide the needed flexibilities and incentives to support continued innovation. With only 5 percent of rare diseases having an FDA-approved treatment, the RDCC’s work is more important than ever.”
While at Sarepta Therapeutics, Stacey played a crucial role in developing and executing regulatory strategies and advancing policies for rare disease programs. Through her experience developing and advancing legislation to address key issues impacting rare disease development and approval, Stacey has a strong understanding of the rare disease policy landscape and understands both its challenges and opportunities. Prior to her role at Sarepta, Stacey served in government affairs and policy roles at the Cystic Fibrosis Foundation and Cigna. She is a graduate of Boston University.
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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
2024 will be a critical year for the rare disease community – with crucial policy challenges and opportunities, a competitive election cycle, and continued economic uncertainty, it is now more important than ever for the RDCC to be a fierce advocate for rare disease innovation and access.
Our 2024 Outlook contains ambitious goals to address concerns and opportunities within both pillars, and empowers the RDCC to harness the support and engagement of our member companies and broader rare disease community. With the partnership of champion policymakers, patient advocacy organizations, and key third-party stakeholders, we maintain our commitment to providing hope for the patients living with one of the 95 percent of rare diseases with no FDA-approved treatment.
Read our full outlook below.
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
By Curt Oltmans, Chief Legal Officer of Fulcrum Therapeutics and Chair of the Rare Disease Company Coalition
For people living with a rare disease, many feel as though they have to face their journey alone. With over 10,000 distinct disease states — 95 percent of which have no cure — the path from diagnosis to care can be complicated, frustrating and costly.
But rare disease is not rare. Over 30 million Americans live with a rare disease, the same number of people in the U.S. living with diabetes. But patients living with a rare disease are often under-researched, underserved and misunderstood, and it doesn’t have to be that way.
Last week was Rare Disease Week, an annual occasion to come together with a hopeful vision for the future of rare disease research and patient care. It is an occasion we use to remind ourselves, and our lawmakers, that there is no time to lose in our mission to discover, develop and deliver treatments to patients living with a rare disease. Half of all rare disease patients are children, and a third of those children won’t live to see their fifth birthday.
Rare disease drug developers must continue to pursue scientific innovation and breakthroughs, but unfortunately, policymakers are currently on a dangerous path of creating obstacles to innovation, threatening the existence of rare disease companies.
As Chair of the Rare Disease Company Coalition (RDCC) and Chief Legal Officer at Fulcrum Therapeutic, I’ve spent years meeting with patients whose stories break my heart, and whose strength fuels our mission to improve the lives of patients with genetically defined rare diseases.
At Fulcrum, one of our key areas of focus is around Sickle Cell Disease – a condition that primarily affects African-Americans. In a community that is already subject to widespread healthcare inequity and mistreatment within the broader healthcare system, those who suffer from Sickle Cell Disease are faced with a painful, life-shortening disease with few treatment options.
We’re able to invest in this kind of research in large part due to the passage of the Orphan Drug Act in 1983, which created a set of incentives for researching rare disease treatments in addition to treatments for common diseases and conditions. Over the past 40 years, the number of orphan drugs skyrocketed by 1,576% – from just 38 to more than 600 treatments for more than 1,000 rare diseases.
While the scientific landscape has transformed over the last 40 years, opening up a wide range of new possibilities for our work, the legal and regulatory environment has not kept up.
Right now, there are several avenues that federal lawmakers and regulators can take to shape and improve the next decade of this work for the better. For instance, the ORPHAN Cures Act would encourage rare diseases research and development by expanding the IRA’s Orphan Drug Exclusion to include multiple rare disease indications. This change allows rare disease companies to follow the science and explore promising research that could lead to additional treatment options. For RDCC members, who spend on average half their annual revenue on R&D, this is a critical fix.
Additionally, it is critical that Congress reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) program. The program provides a crucial incentive to direct research and resources toward drug development for our most vulnerable population – our children. Over the past decade, the PRV program has proven itself to be a critical incentive for decision-makers in the rare disease space – from CEOs to venture capitalists – to pursue research in pediatric populations. And it comes at no cost to taxpayers. By reauthorizing the program, Congress can help rare disease drug developers help children living with a rare disease.
Improving, or even saving, the lives of people living with a rare disease is a nonpartisan issue. On the heels of a powerful Rare Disease Week, we are reminded that while these conditions may be “rare,” the community we serve is large. And while research and treatment challenges we face may be enormous, the need for solutions is even bigger.
At Fulcrum and among all rare disease innovators in the RDCC, we remain committed to the rare disease community, and strongly advocate for smart policy to ensure we can continue doing the work we love. I have never been more optimistic about the opportunities we have in the current scientific landscape. Now, we just need support to meet the moment – and it’s up to Congress to rise to the occasion.
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.