|
|
|
Patient advocates say transparency will help the 340B program realign with its original intent of helping patients. Also, thoughts on supporting small molecules achieving big results. (735 words, 3 minutes, 40 seconds) |
|
|
|
Patient advocates urge 340B transparency |
|
|
Transparency is a first step in redirecting the ballooning 340B program back toward its original goal of helping patients in need, panelists told the BIO Patient Advocacy Changemaker Event (PACE).
Why it matters: 340B is supposed to help ensure patients in need receive access to free or discounted drugs and healthcare services. Unfortunately, providers and middlemen now reap big profits from the program without necessarily helping patients, who face a “complete black box,” panelists said.
What is known: There is no insight as to how 340B revenue is being used by providers, but abundant data exist that provider incentives created by the 340B Program are harming patients.
What they’re saying: “The intention was really for the savings to help safety net hospitals bolster access and help further serve vulnerable patients,” said Anna Hyde of the Arthritis Foundation. “But today, 340B is now the second largest federal prescription drug program behind Medicare Part D.”
“I think the low-hanging fruit is around transparency,” noted Hyde. Brian Connell of Blood Cancer United agreed: “Transparency seems to be where the oxygen is right now, at least on the federal level.”
BIO’s view: “BIO supports programs that create a stronger safety net for American patients. But the data is increasingly clear that the 340B program has not delivered,” BIO has said. “Regulators and policymakers must reassess whether the program is meeting not just its original intent, but the broader needs of Americans.”
Read more on Bio.News. |
|
|
|
Policy can help small molecules make big advances |
|
|
New Genetically Targeted Technologies (GTTs) are achieving remarkable results in neurologic and cardiometabolic diseases, but development is hampered by policy discouraging small molecule drugs.
What are GTTs? “In the central dogma of biology,” DNA makes RNA makes protein, but “most small molecules are targeting the protein,” explained Holly Kordasiewicz of Ionis Pharmaceuticals at PACE. GTTs target RNA, which can be a more straightforward and successful approach.
What can GTTs do? A new amyotrophic lateral sclerosis (ALS) treatment “is not just stopping the progression, but in 40% of people who are treated early in their journey, they're actually regaining function,” said Melanie Lendnal of the ALS Association, “something that was out of the realm of possibility in the neurodegenerative disease space.”
One family's story: KIF1A.ORG founder Luke Rosen described the impact on his daughter’s rare disease symptoms: “Three years without our child writhing in pain every morning.”
Policy challenges: Under the Inflation Reduction Act (IRA), small molecule drugs like GTTs have a maximum of nine years’ protection from price controls, even though GTTs are complex and novel—like biologics, which receive 11 years of protection.
Policy solutions: The EPIC Act would give all small molecule drugs 11 years of protection from price controls. Another proposal, the MINI Act, is specifically targeted at giving GTTs the same protections as biologics.
Read more on Bio.News.
|
|
|
|
|
FDA releases second list of Commissioner’s National Priority Voucher (CNPV) pilot program awardees. Six new products were granted priority review after the companies “agreed to increase affordability, domesticate manufacturing as a national security issue, or address an unmet public health need,” the FDA announced on Nov. 6. Novo Nordisk and Eli Lilly were awarded vouchers for priority review of their candidate obesity drugs in pill form after enrolling in a program to offer discounts for patients buying low doses of their obesity drugs directly from the soon-to-launch TrumpRx website, New York Times reports.
BIO on the American Road to Indiana. BIO President & CEO John F. Crowley will be leading the latest stop on this series of visits to learn more about the needs and attributes of biotech ecosystems around the country. The Nov. 11 trip to the Indianapolis area will feature visits to facilities operated by Novartis and Eli Lilly. Watch this space for coverage.
First drugs announced for HRSA pilot program on 340B rebates. The Health Resources & Services Administration (HRSA) on Oct. 30 announced the first nine drugs that will be included in its pilot program on 340B rebates. The pilot is intended to create “a fair and transparent 340B rebate model process,” HRSA has said. “Under the 340B Rebate Model Pilot Program, covered entities must continue to make purchases through their 340B wholesaler account and must ensure they are only requesting rebates on the above drugs dispensed to 340B eligible patients after a purchase is made,” according to HRSA. Read more here.
COVID-19 vaccine gives extra protection against other infections for children with eczema, study finds. In the latest indication of extra benefits from COVID-19 vaccination, a study presented at a Nov. 6 meeting of the American College of Allergy, Asthma and Immunology showed that children with eczema who received the vaccine had lower instances of several allergic conditions and infections than unvaccinated children. The research on nearly 6,000 patients follows another recent study showing people with advanced lung or skin cancer taking one of the new immune checkpoint inhibitors (ICIs) lived longer if they had a COVID mRNA vaccine within 100 days of initiating cancer therapy. Read more here. |
|
|
|
|
The Senate passed a measure yesterday that takes the first step in ending the shutdown by extending most federal spending through Jan. 30. The package would include funding of the Food and Drug Administration (FDA) through Sept. 30, 2026.
|
|
|
|
|
|
