Center for Health System Sustainability

Equitable access to essential medicines, which the World Health Organization (WHO) defines as those that meet the priority needs of the population,1 is key to achieving universal health care coverage.(2) However, it is estimated that essential medicines are unaffordable or unavailable to 1 in 4 people worldwide.(3) As governments strive to achieve universal access to essential medicines, it is important to understand how the prices and affordability of these medicines vary between countries. High drug prices strain both personal and government budgets, especially in low- and middle-income countries, where patients often face high out-of-pocket costs.

Although many national surveys of medicine prices and affordability have been conducted, fewer analyses have compared these variables internationally, with most research dating back to 2011 or earlier.(4-20) Many of these studies have focused on high-income countries. Of the studies that also included low- and middle-income nations, most compared the prices of 15 or fewer medicines or included a small number of countries, limiting the generalizability of those findings. Therefore, little is known about the relationship between national income and drug prices. The question remains whether some poor countries routinely pay more than rich countries for the same prescription drugs, possibly owing to weaker pharmaceutical pricing policies.

This cross-sectional study used global data on pharmaceutical sales to compare the list prices of 549 essential medicines in 72 high-, middle-, and low-income markets (covering 87 countries) in 2022, both in nominal and purchasing power–adjusted terms. We also evaluated the affordability of 8 essential medicines used to treat major causes of death and disability globally.

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.