A “one-size-fits-all” approach to drug pricing and value assessment frameworks undermines development of therapies for people – very often children – with rare diseases. As rare disease companies, we champion policies that help people with high unmet needs by developing innovative cutting-edge medicines and working with federal and state policymakers to address access and affordability.

Learn more about the importance of restoring equity and patient focus when valuing rare disease treatments in this overview.

The Orphan Drug Tax Credit is a vital lifeline for rare disease patients and should be protected to preserve hope for the rare disease community. Given that approximately 95% of rare diseases of rare diseases lack an FDA-approved drug, any proposal to reduce the ODTC would have a devastating impact on orphan drug development in the U.S. and on millions of Americans living with a rare disease. We must preserve innovation and not lose the gains that have been made since the passage of the ODA.

Learn more about the Orphan Drug Tax Credit and its role in rare disease treatment development in this overview. 

FDA’s accelerated approval pathway recognizes that a “one-size-fits-all” traditional model cannot always work – especially for serious and unique rare diseases with very small population sizes that progress slowly and variably from patient to patient. Therapies approved through the accelerated approval pathway are subject to the same stringent, evidence-based clinical review and approval standards as the traditional FDA approval process, as well as to post-approval confirmatory trials to verify clinical benefit. 

While less than 10% of all accelerated approvals in its 30-year history have been used for nononcology rare diseases, today’s increased understanding of rare disease and advances in targeted drug development are making it possible to realize the promise of accelerated approval for rare diseases where current treatments don’t yet exist.

As policymakers consider policy proposals that impact the trajectory of rare disease treatment development, we advocate for an approach that considers the unique circumstances of rare diseases and caution against punitive measures that could chill future investment and development of rare disease treatments.

Click here to download this overview on the benefits of the accelerated approval pathway for rare diseases.

Building on our letter submitted to the Oregon Health Authority (OHA) on the proposed renewal and amendment of its Section 1115 waiver, RDCC submitted comments to the Department of Health and Human Services Secretary Becerra and Centers for Medicare and Medicaid Services (CMS) Administrator Brooks-LaSure on the OHA Section 1115 Demonstration Waiver. In this letter, the RDCC expresses concern with the potential impact that waiving certain provisions of Section 1927 of the Social Security Act would have on the discovery and development of new medicines, particularly on treatments and cures for patients with rare diseases who currently have limited or no treatment options.

Limiting Medicaid coverage for viable treatments would have a devastating impact on patients benefiting from this innovation and create a greater healthy equity divide, all while resulting in limited budget savings. This proposal is the wrong solution for patients.

The Rare Disease Company Coalition (RDCC) is proud to support the bipartisan Better Empowerment Now To Enhance Framework And Improve Treatments (Benefit) Act (H.R. 4472 / S. 373). We applaud the work Congress has done to date to advance patient engagement in drug development and this legislation introduced by Representatives Doris Matsui (CA-6) and Brad Wenstrup (OH-2) and Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) demonstrates a continued interest in doing so.

The BENEFIT Act addresses the need to reflect the patient perspective and experience in the FDA’s risk-assessment tool. This policy would play a critical role in the FDA evaluation and approval decision regarding drugs and other medical products and will allow the FDA to capture meaningful patient experience, PFDD, and related data as part of the benefit-risk assessment.

The RDCC believes this legislation will advance patient engagement, which ultimately can help advance the development and delivery of
treatments to those living with rare diseases. We applaud Rep. Matsui, Rep. Wenstrup, Sen. Wicker and Sen. Klobuchar for championing policies to improve the regulatory process and supporting the voice of the rare disease patients we serve.

Read our full endorsement letter here.

Newborn screening is a vital and proven public health program that screens approximately four million U.S. newborns each year. In the first 24 to 48 hours of a baby’s life, a small blood sample is taken to detect serious, often fatal, genetic conditions that can be treated if diagnosed early. We believe equitable access to newborn screening can ensure that all babies have the best chance for a healthy life. Join us in working towards a modern newborn screening system that provides equal access to a timely diagnosis for all babies.

Learn more in this educational resource.

Click here to download an overview of our Policy Focus Areas.

Earlier this month, our Coalition expressed concern to the Oregon Health Authority (OHA) on the proposed renewal and amendment of its Section 1115 waiver. We are a Coalition of biotech companies dedicated to the research, development and delivery of innovative treatments for rare diseases with over 225 rare disease development programs in process, we are deeply concerned about the impact of the proposed closed formulary provisions on the rare disease community.  The proposal would limit coverage to one drug per therapeutic class and exclude drugs approved via the accelerated approval pathway.  93% of rare diseases do not have a single FDA approved treatment and limiting coverage for Medicaid beneficiaries that live with a rare disease jeopardizes patient health while threatening to curtail innovation.

We urge our Oregon-based partners, advocates, individuals living with rare diseases, and their caregivers to contact the Governor and their state legislators to voice their concern with this proposed amendment. Limiting Medicaid coverage for viable treatments would have a devastating impact on patients benefiting from this innovation and create a greater healthy equity divide, all while resulting in limited budget savings. This proposal is the wrong solution for patients.

Contact Information:
Governor Kate Brown
Submit comments online at: https://www.oregon.gov/gov/Pages/share-your-opinion.aspx
Call directly at (503) 378-4582

Click here to download the letter submitted from our Coalition to OHA on January 7, 2021.

Watch this video to learn more about the unique challenges in rare disease treatment development, and why “one-size-fits-all” approaches to healthcare policy reform are harmful to continued investment and innovation for rare disease treatments.

Click here to download this overview.