Center for Health System Sustainability

Uncertainties about the benefits and harms of new drugs are common at the time of drugs’ approval. It is unclear to what extent the Food and Drug Administration (FDA) communicates these uncertainties in the FDA-approved prescribing information (the drug label), which is the primary channel of communication between the FDA and physicians. Although physicians might not regularly consult the drug label for prescribing decisions, other information sources used by physicians either index or incorporate information from the label. We searched FDA review documents for uncertainties identified by FDA reviewers with new cancer drugs. We considered the subset of uncertainties highlighted in the FDA’s Benefit-Risk Framework as important to the FDA’s approval decision. During the period 2019–22, the FDA approved fifty-two new cancer drugs. In review documents, FDA reviewers identified a total of 213 clinical trial uncertainties with new cancer drugs, 50 percent of which were considered to be important uncertainties to the FDA’s approval decision. Labels for physicians reported information on 26 percent of all uncertainties and 48 percent of uncertainties that were important to the FDA’s approval decision. Communicating uncertainties about the evidence of drugs in the label is essential for informing physicians about drugs’ safe and effective use.

The Publication-of-the-Year Award recognizes the best and most relevant peer-reviewed, scientific work that the fields of health services research and health policy have produced and published in the prior calendar year.

Publication-of-the-Year Award
Differential Legal Protections for Biologics Vs Small-Molecule Drugs in the U.S.
Olivier J. Wouters, Ph.D.

In recent months, a new kind of partnership between telehealth companies and pharmaceutical manufacturers has come under scrutiny over concerns that such arrangements could lead to inappropriate prescriptions and poor care. Eli Lilly and Pfizer, along with five telehealth companies accessible through the pharma companies’ websites, have recently received letters from four senators echoing those concerns, asking questions about care, prescription volume, and the flow of data and money between the firms. The lawmakers want to determine whether pharma-telehealth deals may be violating the federal anti-kickback statute. That question has also piqued the interest of three health policy researchers at Brown University.

Americans have grown accustomed to direct-to-consumer pharmaceutical advertisements and the refrain, “If you think [this drug] is right for you, talk to your doctor.” But some recent online ads feature a new twist — a link to a clinician offering telehealth services. Several pharmaceutical companies have also launched online platforms that direct users to websites run by telehealth companies, where clinicians are available to prescribe medications after a virtual consultation.

The Inflation Reduction Act instructs Medicare to negotiate prices of top-selling drugs and sets statutory upper limits (“ceilings”) on negotiated prices. Medicare can negotiate prices below the ceilings based on how prices and clinical benefits of these drugs compare with those of therapeutic alternatives. In August 2024, Medicare published the negotiated prices for the first 10 drugs selected for negotiation; these prices will come into effect in 2026 and will apply to all Medicare Part D plans. We analyzed how negotiated prices in the US compared with net prices before negotiation, ceiling prices, and list prices in 6 other high-income countries.

Biologics approved by the US Food and Drug Administration (FDA) receive 12 years of guaranteed protection from biosimilar competition compared with 5 years of protection from generic competition for new small-molecule drugs. Under the 2022 Inflation Reduction Act, biologics are exempt from selection for Medicare price negotiation for 11 years compared with 7 years for small-molecule drugs. Congress codified these differing legal protections on the premise that biologics require more time and resources to develop and have weaker patent protection, necessitating additional protections for manufacturers to recoup their development costs and generate adequate returns on investment.