What to watch on Rare Disease Day

A leap year means we have an extra day in the month to talk about rare disease patients and R&D. Plus, Agriculture Secretary Tom Vilsack talked about biotech on the Hill and BIO’s David Thomas writes about the vaccine pipeline in International Business Times. (849 words, 4 minutes, 19 seconds)

What we’re watching on Rare Disease Day

Today is Rare Disease Day—a chance to stand up for patients and innovation for the 30 million Americans who live with a rare disease.

What’s happening: A House Energy & Commerce Health Subcommittee hearing, Legislative Proposals to Support Patients with Rare Diseases. They will consider 18 bills, including the ORPHAN Cures Act (H.R. 5539), the Maintaining Investments in New Innovation (MINI) Act (R.R. 5547) and the Creating Hope Reauthorization Act (H.R 7384) —three of BIO’s legislative priorities.

The ORPHAN Cures Act would fix a flaw in the Inflation Reduction Act (IRA) to ensure orphan drugs are exempt from price controls, even if approved for two or more indications. (BIO, member companies, and American voters support it.)

Why it matters: “Of the more than 280 orphan drugs approved since 2003, nearly a quarter were later approved to treat additional diseases. Some medicines earned approval for four or more follow-on indications,” said BIO Chair, Dr. Ted W. Love, in a Real Clear Health op-ed published in January.

What they’re saying: “It’s critical that Congress acts to support research and development into treatments and therapies that address rare diseases and further work to ensure that patients have access to the care and to the treatment that they need,” said Rep. John Joyce, M.D. (R-PA), one of the co-sponsors of the ORPHAN Cures Act.

The MINI Act would align the eligibility timeline for Medicare drug price controls for drugs made with genetically targeted technology, often referred to as complex small molecules, with that of large molecules, at 13 years post-FDA approval.

Why it matters: Small molecule drugs are exempt from price controls for 9 years, while large molecule drugs (biologics) are exempt for 13 years, a distinction intended to capture the complexity of manufacturing, longer development timelines, and technical hurdles large molecule drugs face.

The Creating Hope Reauthorization Act would reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) program, which supports treatments to address rare diseases that disproportionately impact children.

Why it matters: About 70% of rare diseases are exclusively pediatric onset.

The EveryLife Foundation for Rare Disease was among the patient advocacy groups on Capitol Hill advocating for legislation including the Creating Hope Reauthorization Act—here’s a photo they shared of some of the hundreds of patients and advocates at the Capitol! (Photo Courtesy EveryLife Foundation for Rare Disease)

More reading about rare disease: The FDA could help save my son from a rare disease—instead, the agency keeps safe treatment out of the hands of patients with no other options, writes former Health and Human Services official Judy Stecker in the Wall Street Journal.

More Health News: 

International Business Times (Opinion): We’ve just scratched the surface of what vaccines can achieve
“Despite this enormous promise, efforts to create new and more effective vaccines still face daunting challenges. Overcoming these barriers will require new approaches to vaccine development—as well as a concerted effort to build public confidence in these life-saving medicines,” says David Thomas, Senior Vice President of Industry Research and Analysis at BIO. “Unfortunately, mere entry into the drug development pipeline is no guarantee of success. New vaccines only have an 11% likelihood of winning FDA approval after entering phase 1 clinical trials. This implies we need 10 vaccines entering clinical trials for each pathogen, which is far more than the current number for most."

USDA Secretary Vilsack talks climate-smart farming, LanzaJet’s new facility

Speaking before the Senate Agriculture Committee, Agriculture Secretary Tom Vilsack highlighted the power of biotech to address climate change—and called out a BIO member’s new investment.

The hearing: Agriculture Secretary Tom Vilsack testified before a hearing of the full Senate Agriculture Committee, where he provided an update on work happening at the U.S. Department of Agriculture (USDA).

The question: “It appears that the private sector in this country, at least some parts of it, are taking very seriously climate change and changing the way they’re going to do business in the future. What do you think we need to do to bring the farming sector into that mindset?” asked Sen. Dick Durbin (D-IL)

The response: USDA is funding 141 climate-smart farming projects, many of which involve the conversion of agricultural waste, such as manure, to sustainable aviation fuel or biobased products. “You’ve created not only a new income source and a new commodity for that farmer; you’ve also created jobs for rural communities.”

The question: Sen. Chuck Grassley (R-IA) asked about USDA’s role in bolstering sustainable aviation fuel and the feedstock required to develop it.

The response: USDA advocates for “a rule that allows a broad array of feedstocks” and specifically incorporating the GREET model (which BIO supports) to determine lifecycle reductions. USDA also provides “the science and data behind the availability of feedstock, the logistics for the supply chain, how you can accelerate adoption and commercialization,” Vilsack said, noting he recently attended the opening of LanzaJet’s ethanol-to-SAF production plant in Georgia.

President Biden’s Thursday: Heading to Brownsville, Texas, to receive a briefing from U.S. Customs and Border Protection, USCIS, and ICE at the border.

What’s Happening on Capitol Hill: We’ll be watching a House Energy & Commerce Health Subcommittee hearing, Legislative Proposals to Support Patients with Rare Diseases.