Accessibility

As rare disease companies work to find equitable, sustainable solutions for patients, we believe that it is paramount that accessibility be top of mind from concept through testing, development, and distribution. Even the most innovative and effective rare disease treatments are only as valuable as they are accessible. Accessibility must be considered at every stage of drug development to ensure solutions that work for all patients. We will continue to promote policies that encourage fair access so that all rare disease patients who are able to benefit from the new innovations have an opportunity to do so.

In the News

How QALY Discriminates Against People Living with Rare Diseases
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Congress Can Unite to Help Kids Living with Rare Diseases
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Groups Hope New Congress Will Pass Bipartisan Bills Centered on Rare Diseases
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New Study Finds Patient Access to Rare Disease Drugs Would Decline Sharply Under Proposals to Limit Medicaid Reimbursements
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Recent Updates

RDCC Proposes Policy Solutions to Remove Barriers for Rare Disease Patients Seeking Treatment Options and Increase Equity of Access
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RDCC Responds to New HHS Payment Models Targeting Accelerated Approval Therapies
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RDCC Urges Swift Action to Reauthorize PDUFA in Letter to Congressional Leaders
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Comments Submitted to Colorado PDAB on Draft Proposed Rule Concerning the Affordability Review of Prescription Drugs
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Featured Content

Capturing the Unique Perspective of Rare Disease in Value Assessment

The FDA’s Accelerated Approval Pathway

Cross-state Medicaid Access

Advancing Patient access to Rare Disease Therapies

Featured Policy Positions

Accelerated Approval

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

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Capturing the Unique Perspective of Rare Disease in Value Assessment

Value-assessment must account for the rarity, severity and progression of the disease throughout a patient’s life. It is imperative that inclusive methods be utilized to restore equity and patient focus when capturing value for rare disease patients. The development of alternative methods for value frameworks must be developed to accurately define how value is calculated for rare disease patients, including encouraging real-world data, prioritizing patient outcomes over payer costs, valuing innovative trial design, advancing health equity, and rejecting foreign price controls.

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Advancing Patient Access to Rare Disease Therapies

In this resource, the RDCC outlines six areas for improvement with specific policy solutions that will improve outcomes for the rare disease community.

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What They Said

“If we want to realize the promise of precision medicine, such as relying on genetics and proteins to treat diseases early, accelerated approval must be in FDA’s toolkit.”

Rep. McMorris Rodgers (R-WA)

“If you are trying to meaningfully reduce state budgetary healthcare expenditures, restricting access to accelerated approved therapies within Medicaid seems to be a strange place to focus your efforts, as in many cases that spending accounts for little more than a rounding error.”

Harry P Bowen, Vital Transformation

“Using the underlying genetics of rare diseases to identify biomarkers that are reasonably likely to predict the benefit of novel therapies should make it possible to rely on these biomarkers for accelerated approval. My colleagues and I have advocated for greater access to appropriate use of this pathway for more rare disease treatments that might not be developed otherwise and proposed a scientific framework in 2015 for qualifying and assessing biomarkers as primary endpoints.”

Emil D. Kakkis, CEO, Ultragenyx

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The Rare Disease Company Coalition helps educate key policy stakeholders on the unique considerations of life science companies when developing and bringing to market therapies for small and differentiated patient populations. If you are a life science company dedicated to rare diseases and interested in joining us, please contact info@rarecoalition.com.

If you are a member of the media interested in learning more about the Rare Disease Company Coalition, please contact us at media@rarecoalition.com.

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