Legislation Calls for FDA to Ensure Expedited Drugs are Safe for Women

Regulatory NewsRegulatory News | 06 May 2015 |  By 

New legislation introduced last week in the House of Representatives would require greater gender equity in some clinical trials overseen by the US Food and Drug Administration (FDA).

The bill, the Research for All Act, was previously introduced in 2014 by Reps. Jim Cooper (D-TN) and Cynthia Loomis (R-WO), and was reintroduced in the House on 29 April 2015.


At its core, the bill aims to address something long known by regulators and medical research officials: Women are sometimes under-represented in clinical trials and medical research. The concern is that if women are under-represented in these venues, regulators and healthcare professionals may not know if a drug is safe or effective for females.

For example, in 2013, FDA announced it would require manufacturers of sleep drugs containing zolpidem to lower the recommended doses in the drugs after research found the drug affected women for a longer period of time than men.

Concerns about under-representation of women in medical research ultimately led to legislators including provisions in the Food and Drug Administration Safety and Innovation Act (FDAISA, Section 907) directing FDA to address "the extent to which clinical trial participation and the inclusion of safety and effectiveness data by [sex] is included in product applications submitted to FDA."

FDA's final report on the subject, known as the Section 907 Report, pledged to improve the quality and completeness of data on sex-specific differences, participation of women in clinical trials and the transparency of data on sex-specific differences.

However, as FDA noted in an accompanying post on its website:

"There is no single 'right' target number or percentage for subgroup analysis.  For the large majority of cases, we do not require any specific numbers for subgroups. The FDA may be able to determine if a medical product is safe and effective for both women and men with enrollment of women at a level less than half and others may be more than half. Statistically, it is possible to determine that a medical product is safe and effective for both men and women in a study with fewer patients than it would be to compare how a medical product performs in women vs. how it performs in men."

In plain terms: While women make up about half of the US population, FDA doesn't require that women make up half of all clinical trial participants (though in many cases women do). FDA requires many companies to assess whether sex-specific differences exist when evaluating the results of clinical trials, and is working on extending this list of products further.

New Legislation

Cooper and Loomis' Research for All Actseeks to go one step further than FDA in this regard. FDA would be required "to ensure that the design and size of clinical trials for products granted expedited approval … are sufficient to determine the safety and effectiveness of such products for men and women using subgroup analysis."

That could be hard to accomplish. Many drugs granted expedited approval are approved on the basis of relatively small trials or surrogate endpoints indicating safety and efficacy.

Increasing the size of these clinical trials could increase the cost of drug development, lengthen the clinical development time or slow enrollment—all critical factors considering expedited approval measures are almost exclusively used for drugs intended to treat "serious or life-threatening disease[s] or condition[s]."

Interestingly, the law would also broaden the current definition of which drugs are eligible for expedited approval. For example, FDA would be allowed to expedite the approval of a drug which is substantially safer or more effective for either "men or women than a currently available product approved to treat the general population or the other sex."

While it's not clear such approvals are prohibited under the current construct of the law—companies often seek approval of a drug in a subset of the population, including a specific sex—the Research for All Act would explicitly allow the practice.

A significant portion of the legislation also deals with sex-specific differences in medical research. For example, NIH is directed to increase the amount of study it conducts using female animals, tissues and cells.

Finally, the Government Accountability Office (GAO) is instructed to update a report on the number of women participating in clinical research.


Research for All Act


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