Good Day BIO: BIO testifies on PDFUA – plus, why RWE matters for Alzheimer's drugs

BIO’s Dr. Cartier Esham and HHS Secretary Xavier Becerra appeared before Senate Committees yesterday. As CMS prepares to announce its final coverage decision for a revolutionary Alzheimer’s drug, a study looks at why the outcome matters for this drug and future drugs requiring real-world evidence. (854 words, 4 minutes, 16 seconds)

BIO's Dr. Esham: PDUFA is ‘most critical to advancing innovation’

Reauthorization of FDA user fee agreements will strengthen dialogue and advance innovation, BIO’s Chief Scientific Officer Dr. Cartier Esham told the Senate Health, Education, Labor & Pensions (HELP) Committee—here’s a recap.

What happened: The Senate HELP Committee held a hearing yesterday, FDA User Fee Agreements: Advancing Medical Product Regulation and Innovation for the Benefit of Patients.

Why reauthorize the Prescription Drug User Fee Act (PDUFA) and Biosimilar User Fee Act? They “ensure effective and timely reviews, improve drug and biologics safety monitoring, enable the Agency to keep pace with medical and scientific advancements, allow for earlier and more frequent FDA-sponsor engagement to identify and resolve drug and biologic development challenges, and provide the support necessary to ensure that advanced medicines are available to patients as efficiently and safely as possible,” said Dr. Esham in her opening statement.

PDUFA VII is “most critical to advancing innovation,” she continued, “enhancing and strengthening scientific dialogue between sponsors of applications and FDA,” especially around decentralized clinical trials and digital health tech, which moved forward during the pandemic.  

What promise does Accelerated Approval provide for companies investing in novel treatments and rare disease? asked Ranking Member Richard Burr (R-NC).

“It is critical for continued investment in innovation in these disease areas that are complex and where there are little or evolving precedents,” Dr. Esham responded. “If it did not exist or its not able to function as intended, the path forward for timely access to improved care for these devastating diseases will be limited and delayed.”

Watch:

AMR also came up—with Sen. Bob Casey (D-PA) highlighting the need for The DISARM Act.

“Antimicrobial resistance is a public health crisis and no single solution is going to fix the problem,” said Liz Richardson, Director of The Pew Charitable Trusts’ Health Care Products Project. “We need creative ideas that can realign current market incentives to encourage drug developers to stay in this business,” including DISARM and The PASTEUR Act.

Why Alzheimer’s drug research requires real-world data

As Centers for Medicare and Medicaid Services (CMS) prepares to announce its final coverage decision for a revolutionary Alzheimer’s treatment, a new study looks at why the outcome matters for this drug and future drugs requiring real-world evidence.

ICYMI: The FDA granted accelerated approval for Biogen’s Aduhelm—the first disease-modifying treatment for Alzheimer’s. But CMS wants to restrict coverage to patients participating in clinical trials, which would put it "out of reach for millions of patients and may disproportionately exclude disadvantaged communities," as 41 bipartisan Members of Congress recently wrote.

Worse, the decision would extend to future FDA-approved Alzheimer’s treatments in this class of drugs—even though FDA said the drug “consistently and very convincingly” reduced plaques believed to contribute to the disease.

In addition to clinical trial data, we also need real-world evidence (RWE)—“to include larger patient populations that are historically underrepresented in randomized controlled trials, including, for example, patients who are ethnically/racially/geographically diverse, have comorbid conditions or are taking concomitant medications,” say Biogen and the National Minority Quality Forum (NMQF).

This is especially true for Alzheimer’s, where trials “historically have struggled to enroll” diverse participants, says a study from Biogen and NMQF. Real-world data can address the gaps and provide information “that cannot be generated in the clinical trial setting.”   

Biogen intends to conduct a post-marketing trial AND collect RWE—“including claims and billing activities, product and disease registries, and patient-generated data including those captured in home-use settings.”

But we won’t get that data if drug’s only covered for seniors in trials—and we’ll limit access to the fortunate few who live close to a trial site and have the ability to participate. 

The next steps: CMS intends to announce its final decision by Monday, April 11—we’ll be watching. 

The Alzheimer’s Disease Policy Task Force, a coalition of Alzheimer’s-focused advocacy organizations recently held a rally for access in Washington, D.C.—watch the stirring video, featuring patients living with Alzheimer's disease, and learn how you can weigh in ahead of the CMS decision:

President Biden’s Wednesday: He’ll address the North America’s Building Trades Unions Legislative Conference at 12:45 PM ET, then sign H.R. 3076, the Postal Service Reform Act of 2022, which will “shore up the U.S. Postal Service's finances, providing support for the struggling agency and also ensuring service six days of the week,” per NPR.

What’s Happening on Capitol Hill: The Senate continues consideration of Supreme Court nominee Judge Ketanji Brown Jackson, who could be confirmed this week, according to CNN. HHS Secretary Xavier Becerra will appear before the House Education and Labor Committee and the House Budget Committee, while EPA Administrator Michael Regan will appear before the Senate Committee on Environment and Public Works. 

ICYMI:The Senate Finance Committee held a three-hour hearing yesterday on the HHS FY23 budget request with HHS Secretary Xavier Becerra. Topics included addressing mental and behavioral health, regulating pharmacy direct and indirect remuneration (DIR) fees, and potentially expanding the Affordable Care Act, along with questions regarding Medication-Assisted Treatment (MAT), Title 42, public health emergency waivers, and more. Notably, a few Senators brought up drug price controls, with Chair Ron Wyden (D-OR) touting the need to give Medicare “the power to negotiate drug pricing for brand-name drugs” as well as caps on insulin and Part D out-of-pocket costs in his opening statement. We’ll keep an eye on this.