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
Read More >

Congress Can Unite to Help Kids Living with Rare Diseases
Read More >

Groups Hope New Congress Will Pass Bipartisan Bills Centered on Rare Diseases
Read More >

New Study Finds Patient Access to Rare Disease Drugs Would Decline Sharply Under Proposals to Limit Medicaid Reimbursements
Read More >

Recent Updates

RDCC Proposes Policy Solutions to Remove Barriers for Rare Disease Patients Seeking Treatment Options and Increase Equity of Access
Read More >

RDCC Responds to New HHS Payment Models Targeting Accelerated Approval Therapies
Read More >

RDCC Urges Swift Action to Reauthorize PDUFA in Letter to Congressional Leaders
Read More >

Comments Submitted to Colorado PDAB on Draft Proposed Rule Concerning the Affordability Review of Prescription Drugs
Read More >

Featured Policy Positions

Accelerated Approval image of article

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.

Learn More

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.

Learn More

Advancing Patient Access to Rare Disease Therapies

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.

Learn More

What They Said

Learn more.

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.

Join our mailing list.


By submitting this form, you are consenting to receive marketing emails from: . You can revoke your consent to receive emails at any time by using the SafeUnsubscribe® link, found at the bottom of every email. Emails are serviced by Constant Contact

©2023 The Coalition for Research, Access, Reform, and Equality for Rare Diseases (RARE). All rights reserved.