Congress has passed a year-end spending package that reflects the strong engagement from our rare disease community. Relationships with elected officials matter and your outreach and personalization of the issues of importance to our community have shone through as Congress made tough choices at the end of 2022. This spending bill, intended to ensure the government remains funded through September 2023, includes multiple healthcare policy riders that were originally in the FDA user fee reauthorization (PDUFA) legislation earlier this year, including many of those that were brought forward by our rare disease community.
Highlights that are especially noteworthy to the rare disease community include:
- Increased funding for FDA, ARPA-H, and NCATS for research in the development of new therapies and diagnostics for rare diseases;
- Critical funding to support a National Academy of Medicine study of the current newborn screening landscape, which reflects recommendations for enhancements to the entire newborn screening system. (This language had been a central part of the Newborn Screening Saves Lives Reauthorization Act);
- Reauthorization of the Orphan Products Grants Program, which includes new language adding regulatory science challenges in future grant funding opportunities (Note that the new regulatory science authority originated from the STAT Act) *;
- Accelerated Approval reform* to strengthen and enhance this vital regulatory pathway used to get innovative therapies to patients at the earliest possible moment;
- Policies aimed at improving clinical trial diversity*;
- Guidelines for pre-approval payor and drug manufacturer communication engagement. (This language accomplishes one of the goals in the STAT Act) *;
- Appropriations report language to advance the coverage of genetic testing for children served by Medicaid, aligning with goals of the Precision Medicine Answers for Kids Today Act;
- Language requiring several new studies and reports from the FDA to better understand how external expertise is incorporated in the review of rare disease treatments (a core component of the HEART Act); and
- A two-year extension for telehealth flexibilities that have enabled increased access to telehealth for Medicaid and Medicare beneficiaries since the start of the COVID-19 public health emergency.
* When the User Fee package passed in September, both the House and Senate stated that they wanted to utilize a year-end package to pass many of the healthcare policy riders that had been included within either the House or Senate PDUFA legislation. These provisions were among those policy riders originally considered for PDUFA.
Many rare disease communities, including RDLA partners, had significant wins included in the omnibus bill. Among them, we celebrate the passage of the Lymphedema Treatment Act, a product of years of advocacy led by the Lymphedema Advocacy Group. In addition, the bill secures appropriations to continue the operations of the Undiagnosed Diseases Network, a lifeline for so many in the rare community who are left without answers on their diagnostic odyssey.
We are grateful for the progress achieved in 2022 thanks to your advocacy efforts and we look forward to engaging the new Congress in 2023 as we celebrate the 40th anniversary of the Orphan Drug Act to ensure that we continue to move rare disease priorities forward.
Please be on the lookout on January 4th for the opening of registration for Rare Disease Week and join us on Capitol Hill to keep our momentum going!
