On July 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar for July. As always, these recurring meetings help provide critical updates to the rare disease community on legislation and other political initiatives that are relevant to their needs. Speakers for this webinar program included:
- Heather Ferguson, Lymphedema Advocacy Group
- Dylan Simon, EveryLife Foundation for Rare Diseases
- Brett Meeks, Horizon Government Affairs
- Jamie Sullivan, EveryLife Foundation for Rare Diseases
Heather provided updates about the Lymphedema Treatment Act, a piece of legislation specifically intended to help address the treatment of lymphedema, a rare disease. Lymphedema can require a specific list of prescription supplies in order to be treated effectively. Unfortunately, this has become a nightmare for patients because insurance companies often refuse to pay for them.
Patients can sometimes successfully appeal these decisions, but in many cases they may be stuck with a bankruptcy-inducing medical bill if they want to get the treatment that they need. This law will affect Medicare recipients by requiring coverage of prescription supplies and is intended to set a standard for Medicaid and private insurers to follow. The bill has broad bipartisan support, with the latest iteration passing a vote out of the House Energy and Commerce Committee. A full House floor vote is expected in the near future. Advocates hope that the legislation will move to the Senate side before year’s end.
Dylan talked about FY2023 appropriations, with six appropriations bills having already been passed in the House. The FDA’s funding is projected to increase by $343 million, including $5 million increase for the Orphan Products Grants Program. This includes a specific priority for ALS.
The National Center for Advancing Translational Science (NCATS) will receive $901 million. This includes $3 million for the CDC Quality Assurance Program and $1 million to fund a study on newborn screening to determine future improvements and needs.
Jamie provided updates about the next iteration of the Prescription Drug User Fee Act (PDUFA), a critical component of drug development regulation. Compared to the situation a few weeks ago, the passage of PDUFA has gotten more bogged down with debate surrounding the formula shortage and the importation of drugs from overseas. Further provisions added by the Senate are likely to slow the full passage of PDUFA.
Additionally, a ‘clean version’ of the PDUFA bill has also been introduced, but this is effectively identical to the old version and would be an extension of the status quo if passed. This would eliminate the opportunity to make improvements.
The Inflation Reduction Act that has been making headlines (and is effectively a scaled down version of the ‘Build Back Better’ bill from a few months ago) also eliminates a previously included proposal to get rid of the Orphan Drug tax credit, which is a valuable incentive for pharma companies to develop rare disease treatments.
Click here to watch a recording of the full length webinar.