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We’re getting ready for Rare Disease Day (February 28) with a look at how policy can support patients and how BIO is engaging with the FDA. (960 words, 4 minutes, 48 seconds) |
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Rare Disease Day: BIO urges legislative and regulatory reforms |
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Ahead of Rare Disease Day (February 28), BIO explains how policy can support patients.
Why it matters: More than 10,000 different rare diseases impact roughly 1 in 10 Americans, and about half of them are children. Yet very few rare diseases have treatments.
The challenge: “It is taking longer and longer to stand up clinical trials, to develop products, and to bring them to market successfully,” said BIO Senior Director of Patient Advocacy Karin Hoelzer at the Goodwin Rare Disease Symposium. A patient-oriented approach would foster reform.
BIO calls for policy and regulatory solutions: - Inflation Reduction Act (IRA) mitigation: The ORPHAN Cures Act, introduced by Reps. John Joyce, M.D. (R-PA) and Don Davis (D-NC), would enable faster drug discovery by extending price control protections to rare disease drugs approved for more than one rare disease. The EPIC Act, reintroduced last week, would remove arbitrary penalties against small molecule drugs.
- The Pediatric Priority Review Voucher, awaiting reauthorization, encourages the development of orphan drugs with vouchers that can be redeemed to receive priority review for a different product.
- Accelerated Approval allows conditional approval of drugs that meet urgent unmet needs and are proven safe and effective against surrogate endpoints. Insurance coverage for these drugs is essential.
- Regulation: The patient’s perspective and regulatory certainty are key to success.
More rare disease legislation: On Tuesday, Reps. Josh Gottheimer (D-NJ), Don Bacon (R-NE), Jimmy Panetta (D-CA), and Tom Suozzi (D-NY) introduced H.R. 1414, Cameron’s Law, to restore the Orphan Drug Tax Credit (ODTC) to 50%.
BIO’s view: “There’s so much opportunity, but there’s also so much unmet need,” said Hoelzer. |
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FDA listens to BIO on Rare Disease Innovation Hub |
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When BIO spoke about how to encourage new treatments for rare diseases, the Food and Drug Administration (FDA) Rare Disease Innovation Hub was listening.
The FDA Rare Disease Innovation Hub promotes innovation in developing rare disease treatments. In the few months since its creation, the initiative has already meaningfully engaged patients and partners like BIO, culminating in the Strategic Agenda for 2025, which included many of BIO’s recommendations.
BIO recommendation: Make the hub a meeting point for education and knowledge-sharing among all stakeholders.
FDA response: The Hub “will design and implement multi-partner education and engagement opportunities.”
BIO recommendation: Innovate to improve the regulatory process.
FDA response: We’ll make sure the Hub is “further advancing regulatory science of rare disease therapies.”
BIO recommendation: “Create a clear framework for the Accelerated Approval Pathway and how to best utilize surrogate endpoints and biomarkers.”
FDA response: The Hub will enhance “opportunities for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods.”
BIO recommendation: “Address the inconsistencies across the federal regulators” through “a system where these agencies could cross-talk with each other.”
FDA response: The Hub will promote “knowledge-sharing around rare disease treatment review … ensuring that our comprehensive drug reviews are conducted within the context of other applications, approval decisions, and related information.”
BIO’s view: “BIO and our members are very excited about the launch of the FDA Rare Disease Hub and that many of our recommendations and feedback were incorporated into their Strategic Agenda,” said Dr. E’Lissa Flores, BIO’s Director of Science & Regulatory Affairs. |
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What Else to Read This Week |
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Patients have until March 1 to give input on drug price “negotiations.” The next 15 drugs set for price controls in 2027 include four cancer drugs and other treatments. During BIO's February Patient Advocacy Coffee Chat, experts discussed why the patient perspective is important, how to avoid unintended consequences of the CMS process, and how patients can engage. “The patient voice is critical and necessary when we start talking about how people are treated, how they access their medications and therapies that they need,” Stewart Perry, Board Chair of the Diabetes Leadership Council explained. Read more on Bio.News.
“Egg prices keep rising. Fact-based policy can help bring them down,” writes Beth Ellikidis, BIO’s VP for Agriculture & Environment, in the Missoulian. As avian flu drives up egg prices, Ellikidis warns against Montana’s House Bill 418, which would ban animal vaccines. “Animal vaccines are subject to overlapping layers of review and control, including a requirement that treated animals complete a ‘withdrawal period’ after receiving a vaccine such that no component of it can be found in them prior to slaughter or milking,” she explains. “For no benefit, then, this ban would penalize farmers and consumers alike, prohibiting farmers from deciding how to protect their animals against deadly illnesses and encouraging the viral spread that is pushing up prices for consumers on grocery aisles.”
FFA Week promotes careers in biotech for agriculture. Beyond inspiring tomorrow’s farmers, FFA (formerly Future Farmers of America) is also promoting careers in biotechnology. During FFA Week (Feb. 15-22), the organization encouraged young people to learn more about agricultural biotech. “You might have to go back to the first tractor, the moldboard plow or even hybridization to find something that’s shaped today’s agriculture more than biotechnology,” says the FFA. Read more on Bio.News.
FDA gives priority review to Gilead’s drug candidate for twice-yearly HIV prevention. Lenacapavir is Gilead’s twice-yearly injectable HIV-1 capsid inhibitor for the prevention of HIV as pre-exposure prophylaxis. “The FDA will review the applications under priority review and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 19,” BIO member Gilead announced.
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