Shenango Valley News

WASHINGTON, D.C. -- U.S. Rep. Mike Kelly (R-PA) has co-sponsored a resolution marking February 29, 2024, as Rare Disease Day, acknowledging the challenges faced by over 25,000,000 Americans living with rare diseases. In a statement, Rep. Kelly emphasized the importance of raising awareness and advancing medical causes to support treatments and cures for rare diseases.

"While doctors and industry leaders are making new lifesaving discoveries every day, too often people with rare and ultra-rare diseases don’t have the opportunity to benefit from these new cures," Rep. Kelly stated.

The resolution introduced by Rep. Kelly aims to shed light on the needs of individuals living with rare diseases and to push forward initiatives that promote medical advancements in the field.

In October 2023, Rep. Kelly played a pivotal role in co-leading the Providing Realistic Opportunity To Equal and Comparable Treatment for Rare (PROTECT Rare) Act. This legislation is designed to assist patients with rare and ultra-rare diseases in accessing evidence-based, medically necessary care covered by their health insurance.

Highlighting the severity of the situation, it is noted that over 90% of rare diseases currently lack FDA-approved treatments, leaving patients in a precarious position where they may have to rely on off-label use of drugs approved for more common conditions. The PROTECT Rare Act seeks to enable Medicare and Medicaid to consider additional sources, such as peer-reviewed literature, in determining if rare disease therapies meet the standard for "medically accepted indications" for coverage. Furthermore, the legislation will require private payers to establish an expedited review pathway for formulary exceptions and appeals related to coverage denials for drugs prescribed to patients with rare disorders.