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Washington’s healthcare policy landscape is constantly evolving, and 2026 is shaping up to be another consequential year for the biotech industry.
Some developments are positive, including progress toward reauthorization of the Prescription Drug User Fee Act (PDUFA) and growing momentum for pharmacy benefit manager (PBM) reform. At the same time, companies are navigating uncertainty stemming from leadership changes at the Food and Drug Administration (FDA) and continued pressure from drug pricing policies, such as the Inflation Reduction Act (IRA), already being implemented and proposals for Most Favored Nation (MFN) pricing.
Against this backdrop, the Washington Policy Brief panel at the inaugural Biotechnology Innovation Organization (BIO) Investment and Growth (BIG) Summit in Miami brought together policy experts to discuss what these developments mean for the industry. Panelists examined the evolving roles of key federal agencies and what shifting policy dynamics could mean for research and development, investment, and patient access.
FDA: change vs. continuity
Anna Abram, Senior Advisor of Health Care Policy and Legislation at Akin, kicked off the conversation by discussing updates at the FDA.
“Change can bring perhaps short-term regulatory uncertainty, but longer-term certainty,” she said. “Sometimes, the policy-making process can be iterative.” But that iterative process often results from an agency trying to keep pace with modern scientific advances, as in the cases of PDUFA and accelerated approval.
As Abram put it, some of this change is driven by leadership asking the question, “How could the FDA do better? How can it be as modern and efficient, predictable and executing, in addition to both protect and promote public health?”
As one example, there was a lot of focus on rare disease. “There has been a lot of buzz around possible mechanisms, framework, pathway, and we saw the FDA release guidance as part of that,” Abram said.
PDUFA: The ‘must‑pass’ engine of regulatory certainty
PDUFA is the quiet backbone of the FDA, dictating both regulatory timelines and staffing decisions, explained Abram.
PDUFA VII is set to run through the end of FY2027 (September 30, 2027), meaning that the agency is currently negotiating with industry on PDUFA VIII before it goes to Congress.
“PDUFA does not prejudge or predetermine regulatory outcomes,” she said. “What it is intended to do is to provide more regulatory certainty about the process in the pre‑market review.”
Naturally, there has been a great deal of energy around its reauthorization.
“Ultimately, PDUFA is predicated on resource alignment between the expected workload and the resources,” she said. “And people and workforces are a key part of balancing that. The goal is to ensure as predictable and streamlined a review process as possible.”
PBMs: Rebate walls, net price, and the new reform triangle
PBMs have been a thorn in the side of the drug affordability question for years, but current reforms may finally shift incentives from list‑price, rebate‑driven models toward net-price transparency.
As Jim Meyers, Senior Advisor at Boston Consulting Group (BCG), explained, PBMs hurt patients in more ways than just affordability. They also depress innovation, leading to fewer cures down the line.
“They create these rebate walls, where it makes it impossible for an innovative company, even with good data, to come in and compete, because they simply don’t have the combination of the volume times that rebate,” he explained.
Meyers noted that the landmark Federal Trade Commission (FTC) settlement against Express Scripts Inc. (FTC–ESI), which required ESI to adopt fundamental changes to its business practices that increase transparency, was a step in the right direction, though limited.
“It affects a lot of change: decoupling pricing from PBM, compensation, rebate pass through, etc.” he explained. “But it only applies to the standard formula for ESI. ESI has seven or eight formulas that employers get to choose from, and I can assure you, those employers will still be choosing from the other six that aren’t this, because they actually have tremendous incentive to do so.”
IRA and MFN: International prices come home
There were a number of unintended consequences of the drug price controls enacted by the IRA, primarily when it comes to access and the cooling of drug development and innovation.
“When the IRA passed, it did include an exemption for orphan drugs,” noted Kelsey Kurth, Policy & Research of U.S. Policy and Government Affairs at Bristol Myers Squibb. “But that exemption was extremely narrowly scoped, so a product with multiple orphan designations was effectively still part of drug price negotiation.”
That narrow exemption effectively incentivized companies to pursue multiple designations for their orphan drugs, lest they lose their window to recoup their investment at market.
Luckily, when the Working Families Tax Cut Act was passed, Kurth continued, it clarified that products with multiple orphan designations can be exempt. However, industry still has work to do when it comes to educating policymakers on the pitfalls of drug price controls and their effects on access—especially when it comes to MFN.
“With MFN, they’re looking at the list price of the drugs in other countries,” explained Carl Schmid, Executive Director at HIV+Hepatitis Policy Institute. “It just doesn’t make any sense. We don’t price our hospital bills based on other countries.”
Ultimately, to solve an American challenge, you have to use an American solution, rather than importing a European system.
Patient-first policy
The panel concluded with agreement that good healthcare policy is always patient-first. That mindset is the north star of the biotech industry and its engagement with Washington.
“For the patient community, we just have to continue to speak up and fight and say: We need more treatments. We need cures. We need a vaccine that works,” said Schmid. “Better treatments, cures, vaccines make America healthy.”
