At the beginning of July 2026, the U.S. Food and Drug Administration approved for young children with .
While this is good news for children in the U.S. born with this illness, the FDA’s approval may also benefit .
About 70% of countries around the world have .
So most rely, in whole or in part, on reviews conducted by agencies that the World Health Organization has designated as . These authorities from 38 countries plus the European Union that meet the highest international regulatory standards for experimental drugs.
I am a and clinical pharmacologist who has researched the and and for nearly 30 years.
, this model has been a win-win proposition for the countries, their citizens and the pharmaceutical companies. However, some could hurt international confidence in their objectivity and rigor.
An arduous process
WHO-Listed Authorities oversee the approval of several types of experimental drugs, including , standard such as aspirin, diuretics, statins and antibiotics, and injectable large-molecule called . Biologics are used to treat conditions including cancer, and, in the case of the newly approved gene therapy, sickle cell disease.
The approval process for these experimental drugs is expensive and labor-intensive. In the case of the FDA, when a pharmaceutical company believes their cell and animal studies are promising enough to warrant human testing, they fill out an .
This is followed by three phases of trials – the first phase entails studies in normal, healthy volunteers, then Phase 2 is dose-finding clinical trials in patients, followed by large-scale clinical trials in Phase 3. It takes to conduct all three phases of clinical trials, and it typically costs about US$1 billion dollars.
If the drug shows a benefit to patients in these trials, the pharmaceutical companies then fill out a voluminous amount of paperwork called a . This application is then reviewed by a team of FDA reviewers . Once those reviews are complete, an external panel of experts does another review of the application and the FDA reviewers’ preliminary findings. That panel then makes a recommendation to the agency, which has the final say about whether a drug is approved.
Only about entering clinical trials are ultimately approved by the FDA.
Other WHO-Listed Authorities for experimental drugs seeking approval to those of the FDA. In fact, the FDA and European Medicines Agency, the regulatory body for the European Union, reach the same approval determination .
However, there can be important differences. For instance, the FDA has an for experimental drugs for serious diseases without effective treatment options. The European Medicines Agency has a similar , but it requires stronger evidence before approval.
These procedural differences aren’t just a matter of paperwork: A 2024 study found that only 43% of cancer drugs with accelerated approval by the FDA .
How other countries use these agencies
Many countries that of experimental drugs but make their own final decision about which drugs to approve for use. Other countries automatically recognize the approval from a WHO-Listed Authority in their own country.
Still others take a , where they might require a pharmaceutical company to submit a unique packet for approval for certain types of drugs, but they rely on data from an agency on the WHO list to inform their decisions about other drug types.
Benefits for developing nations
There are to using a WHO-Listed Authority rather than developing its own drug submission process from the ground up.
First of all, pharmaceutical companies may simply choose not to dedicate their time and resources to completing approval applications for countries where the drug won’t make a profit. In that case, citizens of those countries would never have access to the treatment.
Using existing regulatory reviews is also much more cost-effective than hiring experienced reviewers and vetting an experimental therapy internally. An for a new drug application includes at minimum a project manager, medical officer, toxicologist, statistician, medicinal chemist and two pharmacologists – one basic and one clinical.
Finally, relying on existing data from trusted regulatory bodies reduces approval timelines so that new treatments reach patients more quickly. This is important, because research participants in experimental drug trials are , but there is a long delay in new drugs being available there.
Even with the WHO list, from the time a drug first becomes available for use in any country, the median time to availability is three years for high-income countries but .
In the case of sickle cell disease, the hope is that developing nations will be able to use the FDA’s approval to approve the product for use in their own countries.
Changes at the FDA
Changes to the FDA at the beginning of President Donald Trump’s second term in 2025, including major staff cuts, have caused some public health experts to effectively.
Then, in June 2025, the FDA rolled out a program called the . It to get accelerated approval even if their experimental drug is not intended to treat life-threatening conditions for which no adequate therapy exists.
Since then, have been invited to participate in this program, in exchange for agreeing to terms such as increasing drug affordability or bringing their manufacturing facilities to the U.S. These manufacturers may obtain review periods as short as one to two months.
Some U.S. policymakers may see this as a good way to and .
But others worry that this program does not give the FDA enough time to adequately assess an experimental drug for safety and effectiveness before rendering a decision about approval. Many healthcare workers, health policy organizations and politicians have expressed concern, including , , and .
Outside the U.S., these changes to the FDA could affect its standing as a WHO-Listed Authority, which is valid .
At this point, the WHO has not signaled that it is considering a reevaluation of the FDA. But other countries may be reticent to use the agency’s reviews in the future if changes like this pilot program compromise the agency’s ability to ensure the safety and effectiveness of experimental drugs entering the market.
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