The following was cross-posted by MassBio CEO and President Kendalle Burlin O’Connell on LinkedIn:
The IRA needed (and needs) fixing. Even as lawmakers are speaking out about the need to address the ‘pill penalty,’ there are others who want to undue the fix that was finally passed in July—ORPHAN Cures. More on that, the latest from FDA, 340B, and the shutdown in this week’s update:
ORPHAN Cures Under Threat: The CBO released a new estimate earlier this week on the expanded orphan drug exclusion in the “One Big Beautiful Bill”, suggesting it could cost Medicare as much as $8.8B over 10 years, or about $3.9B higher than previous estimates. In response, Senators Welch, Wyden, and Cortez Masto introduced the No Big Blockbuster Bailouts Act, which would allow negotiation for orphan drugs with multiple indications if their annual Medicare spend exceeds $400M. MassBio supported ORPHAN Cures because it will help deliver hope to the 30 million Americans who are impacted by the 6,000+ rare diseases for which there are no current treatment options available. ORPHAN Cures fixes IRA’s price negotiation provisions, which have had a demonstrated impact on reducing investment and development of treatments for rare diseases. In our view, any “savings” found by repealing the legislation will only ultimately cost patients through reduced innovation and delayed cures.
FDA’s New Commissioner’s National Priority Voucher (PRV) Program: FDA named the first 9 recipients of the new Commissioner’s PRV Program last week. Staff within each FDA review division were given the opportunity to select one recipient based on specific criteria addressing major national priorities (public-health crises, innovative treatments, large unmet need, onshoring, affordability). FDA suggests the voucher could cut NDA/BLA review timelines from 1 year to just 1–2 months. At MassBio, we will continue to work with FDA to identify what’s driving 12-month reviews and propose fixes to shorten timelines across the board.
FDA Approvals Slowing: A recent analysis found a significant drop in approvals, more complete response letters, and more missed PDUFA dates through Q3. The rising delays are linked to disruptions across FDA infrastructure, including short staffing, leadership turnover, and inspection backlogs. The delays may be exacerbated due to the government shutdown since the FDA is not accepting new submissions. Has your company been impacted? Let us know.
Senate HELP 340B Hearing: On October 23, the HELP Committee will examine 340B’s growth and patient impact. The program has been criticized for expanding its scope beyond its original intent, lack of transparency, and potential for abuse. We’ll be following closely and are expecting questions on the program, drug pricing, and patient access.
Shutdown Continues: As we inch closer to the one-month mark of the government shutdown, negotiations between Democrats and Republicans have largely stalled. However, rumors are circulating of a year-end health policy package once the shutdown ends. Such a package may include scaled-back ACA subsidies along with other health-related items. MassBio will continue to advocate for maintaining the orphan drug exclusion and pushing for passage of the EPIC Act to end the pill penalty, along with PBM reforms.