Polsinelli Podcasts - Confusion to Clarity on the Future of the 340B Program
FDA’s Center for Biologics Evaluation and Research (“CBER”) recently issued a trio of new draft guidances to assist sponsors who are developing and conducting postapproval studies of cell and gene therapies (“CGTs”):...more
Through our Venable FDA Pulse series, we provide insight into key developments within the Food and Drug Administration (FDA or the Agency), emerging trends, enforcement activity, and other topics impacting stakeholders....more
On Wednesday, the U.S. Food and Drug Administration (FDA) published three new draft guidances that aim to offer greater clarity to sponsors of cell and gene therapies (CGTs) on issues related to clinical trial design,...more
In the latest episode of Health Law Diagnosed, Of Counsel Bridgette Keller is joined by Associate Hassan Shaikh for a discussion on noteworthy developments relating to the IRA and drug pricing. Together, Bridgette and Hassan...more
On September 3, 2025, the FDA announced the RDEP, a new framework intended to accelerate the approval of treatments for ultra-rare genetic diseases.1 The RDEP was jointly developed by the Center for Drug Evaluation and...more
On September 3, 2025, the US Food and Drug Administration (FDA) announced a new process to support the development of drugs intended to treat rare genetic diseases. The Rare Disease Evidence Principles (RDEP) process offers...more
In the latest episode of Health Law Diagnosed, Of Counsel Bridgette Keller is joined by Associate Hassan Shaikh for a discussion on noteworthy developments relating to the IRA and drug pricing....more
On September 3, 2025, the Food and Drug Administration (FDA) announced Rare Disease Evidence Principles (RDEP) with processes aimed at providing greater predictability and facilitating the development and review of drugs...more
On July 4, 2025, President Trump signed the One Big Beautiful Bill Act (OBBBA) into law. The act extends several provisions of the Tax Cuts and Jobs Act, expands Health Savings Account (HSA) eligibility, and creates new...more
The legal battle over the scope of orphan drug exclusivity continues, as the U.S. Food and Drug Administration (“FDA”) has recently lost a second case concerning the exclusivity provisions of the Orphan Drug Act (“ODA”)....more
The One Big Beautiful Bill Act ("OBBB") expands exemptions for orphan drugs from mandatory Medicare price negotiations and modifies their eligibility timeline....more
On July 4, 2025, President Trump signed into law the One Big Beautiful Bill Act, his signature policy legislation. Two aspects of this legislation are particularly relevant for drug pricing....more
The landscape for rare disease treatments appears to be evolving rapidly, thanks to a confluence of regulatory momentum and a booming market forecast. Statements from key federal figures in recent months combined with market...more
On March 19, 2025, Shanghai Henlius Biotech announced that the FDA has granted Orphan Drug Designation for HLX22, an anti-HER2 monoclonal antibody, for the treatment of gastric cancer. HLX22 binds to HER2 extracellular...more
President-elect Trump’s nominee for commissioner of Food and Drugs, Martin A. Makary, MD, MPH, argued in 2016 that Congress should reform the Orphan Drug Act (ODA) because drug companies “are gaming the system to use the law...more
In April 2023, we published an alert in relation to two European Commission legislative proposals: new Regulation 2023/0131 and new Directive 2023/0132, to replace the current EU regulatory framework for all medicines...more
The Inflation Reduction Act (IRA) was signed into law in August 2022 with the goal of curbing inflation by, among other things, lowering prescription drug prices. Notable prescription drug provisions of the IRA include the...more
This update is the final in a multi-part series exploring FDA approval of pet care drugs. In part one of our series we discussed that before an animal drug product can be legally marketed for use in animals, a New Animal Drug...more
On June 30, 2023, the Centers for Medicare and Medicaid Services (CMS) released its much-anticipated final guidance (“Final Guidance”) for the Medicare Drug Price Negotiation Program (“Negotiation Program”) through the...more
We recently published an alert in relation to the European Commission’s legislative proposals to replace the current EU regulatory framework for all medicines (including those for rare diseases and for children). One of the...more
Last month, the Food and Drug Administration’s Office of Orphan Products Development (“OOPD”) found that one prescription drug company was able to, essentially, breach another’s orphan drug exclusivity (and have its new drug...more
The Inflation Reduction Act of 2022 (IRA) establishes the Medicare Drug Price Negotiation Program (“Negotiation Program”), which permits the government to negotiate drug prices for certain high expenditure, single source...more
While many provisions of the FY 2023 Consolidated Appropriations Act (Omnibus) have received much attention, one has flown under the radar. In its explanatory statement on the Omnibus, the US Congress indicated that it is...more
Recently, the Food and Drug Administration published a Notification to clarify its policy about orphan drug exclusivity. The agency issued the announcement because the U.S. Court of Appeals for the 11th Circuit found that FDA...more