By Patrick Cooney, for AMRC
FEDERAL AGENCY NEWS
Single Pivotal Trial for Drug Approvals
Reaction from Congress has been muted since the U.S. Food and Drug Administration (FDA) announced its decision to move toward a single pivotal trial for drug approvals. No new legislation has been introduced, but several lawmakers have expressed scrutiny of FDA decision-making more broadly, which could extend to this change. At this stage, congressional reaction appears to be cautious oversight rather than outright opposition. Many lawmakers historically support policies that accelerate medical innovation and reduce regulatory barriers, but they also tend to monitor whether safety standards are maintained.
On February 19, 2026, the FDA announced a significant shift in its drug approval framework, establishing one adequate and well-controlled clinical trial—supported by confirmatory evidence—as the default standard for approval of new drugs.
The policy was outlined in a New England Journal of Medicine article authored by FDA Commissioner Dr. Martin Makary and Dr. Vinay Prasad, Director of the Center for Biologics Evaluation and Research. The announcement marks a notable departure from the long-standing expectation that sponsors conduct two pivotal clinical trials to support marketing authorization.
For decades, the FDA typically required two independent trials to demonstrate safety and effectiveness. This approach helped reduce the risk of statistical false positives and provided additional confidence in clinical findings. However, the agency notes that this framework emerged during an earlier era of drug development when scientific understanding and clinical trial methodologies were less advanced.
In practice, the two-trial standard has already been applied flexibly. Since 1997, the FDA has had statutory authority to approve therapies based on a single adequate and well-controlled study combined with confirmatory evidence. In some therapeutic areas—particularly oncology—single-trial approvals have become increasingly common.
Confirmatory evidence may include:
- Mechanistic or biological evidence
- Data from related indications
- Animal models
- Evidence from drugs in the same class
- Real-world data
- A second adequate and well-controlled study
Under the new policy, the FDA will generally consider one well-designed pivotal study plus confirmatory evidence as sufficient for marketing authorization.
The FDA makes clear that the new policy does not eliminate the possibility of requiring multiple trials.
Additional studies may still be necessary if a clinical trial:
- Relies on a poorly understood or nonspecific mechanism of action
- Uses surrogate or short-term endpoints with uncertain clinical relevance
- Contains design limitations or methodological concerns
Bringing a new drug to market currently takes more than seven years on average. By allowing a single pivotal trial in many cases, the FDA expects the policy to reduce development timelines and lower research costs. However, pivotal trials will remain expensive.
The FDA suggests the policy could shift the agency’s focus from reviewing multiple trials to ensuring the quality and rigor of a single pivotal study. For sponsors and clinical research organizations, this may result in greater emphasis on trial design, comparator quality, and endpoint selection.
Regulators anticipate that lowering development barriers may stimulate additional investment in drug development, particularly among smaller biotechnology firms that previously faced significant financial constraints associated with conducting multiple large trials.
FDA Moves to Streamline Biosimilar Approval Process
On March 9, 2026, the U.S. Food and Drug Administration (FDA) announced another major step in its initiative to streamline the development of biosimilar medicines. In new draft guidance, the agency recommended streamlining unnecessary clinical pharmacokinetic (PK) testing when scientifically justified. The FDA states that this change could save biosimilar developers up to 50% of their PK study costs and help lower drug costs.
“Streamlining biosimilar development reflects our ongoing commitment to lowering drug costs for everyday Americans,” said FDA Commissioner Marty Makary, M.D., M.P.H. “Using common sense, we are embracing more precise analytical testing approaches than have been used in the past.”
Biologic medicines can be powerful treatments for many diseases, including autoimmune diseases and cancer, but are often expensive. Despite accounting for just 5% of prescriptions, biologics account for 51% of drug spending, and commonly cost hundreds of thousands of dollars per year. Biosimilars, like generic drugs, can give patients more affordable treatment options and increase access to medications that are otherwise unaffordable.
Today’s announcement builds on a prior FDA effort in October, when Commissioner Makary announced another draft guidance that reduces certain unnecessary comparative efficacy studies.
The new draft guidance, “New and Revised Draft Q&As on Biosimilar Development and the BPCI Act (Revision 4)” is intended to inform prospective applicants and facilitate the development of proposed biosimilars and proposed interchangeable biosimilars. It revises and replaces the draft guidance for industry entitled “New and Revised Draft Q&As on Biosimilar Development and the BPCI Act (Revision 3)” issued September 17, 2021, and includes revisions to certain Q&As that appeared in a previous version of the final guidance entitled “Questions and Answers on Biosimilar Development and the BPCI Act.”
Specifically, this draft guidance provides updated recommendations to prospective biosimilar applicants seeking to use data from a comparator product approved outside the U.S. (“non-U.S.-licensed comparator product”) as evidence that a proposed product is biosimilar to the U.S.-licensed product. The recommendations describe scenarios in which a biosimilar applicant may use clinical data from outside the U.S. without additional data from a three-way PK study (using the proposed biosimilar, the U.S.-licensed reference product, and the non-U.S.-licensed comparator product). The revisions also remove the earlier recommendation for at least one clinical PK study that directly compares the proposed biosimilar with the U.S.-licensed reference product to support a demonstration of biosimilarity; instead, a PK study can use a comparator product approved outside the U.S. if scientifically justified.
Decision on Moderna Flu Vaccine Expected August 5th
On February 18, 2026, the FDA’s Center for Biologics Evaluation and Research (CBER) reversed course and stated it would review the application for an mRNA flu vaccine for those 50 and older. Moderna is seeking full FDA approval for adults 50 to 64 years of age and accelerated approval for adults 65 and older, along with a post-marketing requirement to conduct an additional study in older adults. The FDA accepted the application for review, with a deadline of August 5, 2026.
The FDA had refused to review the original application because it said Moderna’s phase 3 study was not “adequate and well-controlled.” Most in question was the use of a standard-dose flu shot in the comparator arm of the trial for those 65 and older, instead of a high-dose seasonal flu vaccine. Moderna said CBER had previously approved the study design.
CAPITOL HILL NEWS
Health Policy on Capitol Hill in Q2 of 2026
As Congress moves into the second quarter of 2026, several health policy issues are expected to command attention on Capitol Hill. While broad health reform legislation is unlikely in an election cycle, lawmakers are advancing targeted initiatives that could have meaningful implications for clinical research, drug development, and the health care delivery system. For organizations engaged in clinical trials and medical innovation, developments related to FDA oversight, Medicare policy, research funding, and workforce issues will be particularly important to monitor.
Members of Congress are expected to continue oversight of FDA review practices through hearings and bipartisan legislation related to clinical trial modernization, decentralized trial models, and the use of real-world evidence. These issues may also be incorporated into broader legislative efforts, sometimes referred to as “FDA Modernization Act 3.0.” For the clinical research enterprise, these discussions may shape future regulatory expectations and the design of clinical studies.
Federal funding for biomedical research will remain a central issue as Congress begins work on fiscal year 2027 appropriations. Lawmakers are expected to review funding levels for agencies such as the National Institutes of Health and other health research programs while balancing broader federal budget constraints.
Although major funding increases may be difficult in the current fiscal environment, bipartisan support for biomedical innovation remains strong. Congressional committees are likely to examine how federal research investments translate into new therapies, economic growth, and improved health outcomes. For clinical trial sponsors and research organizations, the appropriations process will be an important indicator of federal priorities in areas such as precision medicine, oncology, rare diseases, and advanced biologics.
Payment policy within the Centers for Medicare & Medicaid Services is expected to be a significant topic of congressional activity during the coming months. Concerns about declining physician reimbursement and the financial stability of provider organizations have prompted lawmakers in both parties to explore legislative solutions to stabilize Medicare payment updates.
In addition, Congress is continuing to examine Medicare coverage policies related to innovative therapies and diagnostics. Policymakers have increasingly focused on how coverage decisions affect patient access to emerging treatments and participation in clinical trials. Discussions around value-based care and evidence-based coverage may also influence how new medical technologies enter the health care system.
Taken together, the second quarter of 2026 is likely to feature a mix of targeted legislative initiatives and oversight activity rather than sweeping health reform.
