As Prescribed

Taking a step toward fulfilling its requirements under Section 506K of the Federal Food, Drug, and Cosmetic Act (Section 506K), FDA has issued Draft Guidance describing how the agency intends to implement the Platform Technology Designation Program. Section 506K was established by Section 2503 of the Prepare for and Respond to Existing Viruses, Emerging New Threats, and Pandemics Act of 2022 and enacted as part of the 2023 Consolidated Appropriations Act.

Pharmaceutical drug pricing and reimbursement continues to make headlines nationwide. In a recent development, Maryland’s Prescription Drug Affordability Board (PDAB) finalized its list of selected drugs for affordability review. The board will engage in what it contends will be a comprehensive “cost review” where it will seek public comments, additional information, and data over a 60-day period to determine if the selected drugs will be subject to state-prescribed upper payment limits.

In what is being touted as a significant win for pharmaceutical drug manufacturers, the DC Circuit affirmed on May 21, 2024 that Section 340B of the Public Health Service Act does not categorically prohibit manufacturers from imposing distribution restrictions on covered drugs to covered entities. By rejecting the US government’s position that the 340B Drug Pricing Program categorically prohibits manufacturers from imposing any contractual conditions on the distribution of covered outpatient drugs, the court opened the door for new contract relationships between program manufacturers and covered entities.

At a time when pharmaceutical drug prices are at the height of political scrutiny, manufacturers can breathe a small sigh of relief as the Centers for Medicare and Medicaid Services (CMS) announced on May 15, 2024 that it will not, at this time, finalize its best price stacking provision in its May 23, 2023 Medicaid Drug Rebate Program (MDRP) proposed rule.

Pharmaceutical drug pricing and reimbursement remains a bipartisan focus as we draw closer to the November presidential elections, with politicians remaining steadfast in their efforts to turn up the heat on pharmaceutical manufacturers. Politicians and other groups from across the political spectrum have coalesced around a 40-year-old statute known as the Bayh-Dole Act—intended to facilitate the public’s beneficial use of patented inventions by securing intellectual property (IP) rights for inventors—and are seeking to transform the statute’s never-before-used “march-in” rights to influence the price of drug products.

2024 is shaping up to be a big year for prescription drug affordability boards (PDABs). Like state price transparency reporting laws, state legislation establishing PDABs continues expansion. In response to rising prescription costs and overall state-level health care spending, numerous states have established PDABs to review certain high-cost prescription drugs and determine if states should take action to reduce those prices.

Effective April 1, 2024, California will expand its existing prescription drug price transparency requirements by enacting legislation to amend and clarify requirements on wholesale acquisition cost increase reporting and notice submissions as well implement further processes for manufacturer penalty hearings.

The US Food and Drug Administration (FDA) issued draft guidance, providing recommendations to sponsors who are considering submitting a non-interventional study (i.e., an observational study) to FDA to support the demonstration of substantial evidence of effectiveness and/or evidence of safety of a drug or biologic, as another chapter in its evolution toward the use of real-world evidence (RWE).

The ball has dropped on the healthcare and life sciences industry’s unique New Year tradition, the J.P. Morgan Healthcare Conference, so all eyes turn to 2024. To help define a path forward, Morgan Lewis FDA and healthcare partners Jacqueline Berman, Rebecca Dandeker, Maarika Kimbrell, and Kathleen Sanzo have assembled an in-depth report on drug and biologic developments at the FDA.

FDA’s Center for Biologics Evaluation and Research (CBER), the locus of regulating cell and gene therapies among other expanding areas of biotech, is poised for change on a number of important fronts—leadership, significant growth, and plans to address challenging scientific and regulatory issues. With a well-earned reputation of stability, and slow, plodding progress, moments with so many elements in flux at FDA don’t come around often, so it is worth keeping an eye on how these begin to unfold to have a sense of where CBER—central to many of the exciting developments in industry—is headed.