The Need: Federal rare disease programs and projects occur across the entire government aimed to address challenges within the rare community such as the diagnostic odyssey, a limited rare disease therapy pipeline, and access to care and treatments.

The Proposed Solution: Interagency coordinating committees have been successful in assessing existing federal initiatives and identifying opportunities for improvements, ensuring one agency initiative can have maximum impact through coordination with other agencies, tracking metrics that can show progress or other areas where resources are needed.

The creation of an interagency coordinating committee will provide a central location for sustained coordination of the federal government’s rare disease initiatives.

Learn more about the Interagency Coordinating Committee.

The purpose of PRV’s is to expedite the development and promotion of drugs in the rare pediatric diseases. Developing drugs for rare pediatric diseases is particularly challenging due to the small populations affected, difficulties associated with conducting clinical trials for children, delays in diagnosis, and more. A majority of PRVs have gone to rare pediatric disease drugs, meaning the program has a large impact on the rare disease community.

It is vital to reauthorize the Priority Review Voucher program, which provides incentives to pharmaceutical companies for developing treatment for pediatric rare diseases. Companies can either use the voucher (or incentive) to accelerate the approval of another drug they are developing, or they can sell it to another pharmaceutical company.

House Co-Chairs: Representative Doris Matsui (CA) and Representative Gus Bilirakis (FL)

Senate Co-Chairs: Senator Roger Wicker (MS) and Senator Amy Klobuchar (MN)

Find out if your members of Congress are involved in the Rare Disease Caucus

Learn more about the Rare Disease Congressional Caucus

• Gives the rare disease community a permanent voice of Captiol Hill
• Holds quarterly Caucus briefings to update Congressional Staff on important policy issues
• We want every member of Congress to not just know about rare disease but to know a rare disease patient in their district

Currently, if a patient needs to see a provider outside of their state the provider must become a part of the patient’s state’s Medicaid program, which is a long-time consuming process.

This bill would change this process by making a new, faster process for doctors to enroll in another state’s Medicaid program by:

• Establishing a federal streamlined screening and enrollment pathway
  • Voluntary, meaning providers are not required to do this
  •  If providers opt to be screened through this pathway, they will be eligible to enroll in other state’s Medicaid programs for a 5-year period, assuming they remain in good standing
• Eligible providers must present the most limited risk of fraud and abuse, be enrolled in Medicare or their home state Medicaid program, and not be excluded from participating in any federal health programs