Good Day BIO: ARPA-H, Accelerated Approval, and more

We have a heavy focus on health policy today, with key discussions on ARPA-H and the Accelerated Approval Pathway, plus a new study that shows how we might reduce or eliminate clinical trials involving children. (784 words, 3 minutes, 55 seconds)

Can ARPA-H advance new cures?

The purpose of ARPA-H, drug pricing, and antimicrobial resistance were topics discussed during yesterday’s House Energy & Commerce Health Subcommittee hearing with former Assistant Secretary for Health Admiral Brett P. Giroir and major research institutions—here’s a quick recap.

What is ARPA-H?President Biden proposed the creation of ARPA-H—the Advanced Research Projects Agency for Health—a new agency under NIH to focus on research for diseases including cancer, diabetes, and Alzheimer’s.  

Members debated the need for ARPA-H, with Rep. Kurt Schrader (D-OR) saying it should be complementary, not duplicative. (BIO previously said we need more details.) 

They also debated where it should live, with some saying NIH, while Admiral Giroir warned housing the agency under NIH could doom it to “mediocrity,” which America cannot “afford.”  

But there’s a disconnect: “On the one hand, some members are stressing the need for additional research and development and a new department or agency, while on the other hand actively working to potentially curtail the industry’s efforts to find new cures through price-control-type policies,” said Rep. Larry Bucshon, M.D (R-IN), citing a letter from small biotechs and investors.

“We have to listen to the biotech community,” said Admiral Girior. “We can absolutely not bias the system against any class of therapeutics.” (Watch the exchange around 2:13:00.) 

We also can’t ignore the “lack of new antibiotics in the pipeline,” said Rep. Fred Upton (R-MI). Esther Krofah, Executive Director of FasterCures and Center for Public Health at the Milken Institute, cited the need to fix the marketplace for new antibiotics through Cures 2.0/The PASTEUR Act.   

The next steps: Chair Anna Eshoo (D-CA) made it clear she intends to hold a markup for both Cures 2.0 and ARPA-H legislation—stay tuned.

What about accelerated approval?

Everyone’s talking about accelerated approval, particularly in the context of Aduhelm—here’s two opportunities for you to engage in the conversation.

To catch you up: The first treatment addressing a likely cause of Alzheimer’s was recently approved by the FDA via the Accelerated Approval Pathway, but Centers for Medicare and Medicaid Services (CMS) proposed to restrict coverage to patients participating in clinical trials, setting a dangerous precedent for Alzheimer’s and other hard-to-treat diseases.

Dr. Mark McClellan will speak with BIO about this topic next week. The former CMS Administrator and FDA Commissioner will join BIO President and CEO Dr. Michelle McMurry-Heath in conversation live in New York at the BIO CEO & Investor Conference on Tuesday.

The CMS decision is not just about Aduhelm—it sets a precedent for “rationing” Medicare beneficiaries’ access to treatments approved under the Accelerated Approval Pathway, as the Alliance for Aging Research will discuss in a free virtual event today.

The bottom line: Now is the time to “double down on” and “modernize” accelerated approval—one of the only regulatory pathways to address unmet need, as Dr. Michelle has said. 

Register:

• February 9 (Today!): Alliance for Aging Research Webinar
• February 14-17: BIO CEO & Investor Conference feat. Dr. Mark McClellan

Study: Pediatric drug development could benefit from ‘extrapolation’

Pediatric drug development lags adult drug development by about eight years, says a recent study co-authored by BIO—here’s what we can do about it. 

The study:Extrapolation as a Default Strategy in Pediatric Drug Development, published in January in Therapeutic Innovation & Regulatory Science, was authored by members of BIO’s Pediatric Drug Development Task Force. 

The key finding: By “extrapolating” efficacy, we could reduce or eliminate clinical trials involving children altogether and speed up pediatric drug development. 

What is “extrapolation”? A strategy “based on assessing the relevance of existing information in the adult population to the pediatric population, in terms of the disease, drug pharmacology, and clinical response to treatment,” the study says. 

The result: In some cases, pediatric trials could be completely avoided, or at least reduced in size and scope. 

The bottom line: “It is a clear ethical obligation to ensure that children are not enrolled in unnecessary clinical studies. This obligation compels us to explore the use of extrapolation, when scientifically and clinically appropriate, to the fullest extent possible when designing pediatric clinical development programs.”

Read the full study.

More Health Care News: 

Bristol Myers Squibb: BMS awards nearly $8 million in new health equity grants to improve access to care for medically underserved patients in U.S.
The grants are awarded to “24 U.S. non-profit organizations that aim to improve access and quality care to medically underserved patients and communities across oncology, cardiology, immunology, and hematology therapeutic areas.”

President Biden’s Wednesday: The White House COVID-19 Response Team will hold a briefing at 11 AM ET. President Biden will host a roundtable with CEOs of electric utility companies to discuss Build Back Better at 2:30 PM ET.

What’s Happening on Capitol Hill: The Senate Finance Committee will consider Health and Human Services (HHS) nominees.