RDCC, Author at Rare Coalition

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.

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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.

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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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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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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.