The Food and Drug Administration is generally achieving the balance between ensuring safety and providing access to investigational drugs through compassionate use programs, according to results from a new analysis that found that most of these drugs are available within 6 months of an application to the FDA.
But that balance may be threatened by the recently enacted Right to Try Act, Jeremy Puthumana, of Yale University, New Haven, Conn., and his colleagues reported in an article published on JAMA Network Open. They said the new law encourages sponsors to make investigational drugs available earlier in the clinical development period, potentially jeopardizing safety.
“These findings suggest the FDA and the pharmaceutical industry have established a balance between investigational new drug access and protection of patients from therapies without established safety, which may be compromised by policy makers seeking to speed access to investigational medicines through the Right to Try Act by removing the requirements for FDA oversight and approval of expanded access requests,” the researchers wrote.
The cross-sectional study examined all expanded access programs registered with ClinicalTrials.gov through Aug. 1, 2017. Of 92 expanded access programs for investigational drugs, 69.6% were initiated within 6 months following (43.5%) or preceding (26.1%) submission of a new drug application. Ninety of the 92 drugs ultimately went on to receive FDA approval.
Of the most common uses of the 92 drugs registered in expanded access programs between September 1996 and June 2017, half were used for the treatment of cancer; 16 drugs were used to treat metabolic, endocrine, and genetic diseases; and 14 drugs were used for the treatment of infectious diseases. But there were no significant differences in the timing of program initiation by therapeutic characteristics, the researchers found.
President Donald Trump signed the Right To Try Act of 2017 (S. 204) into law on May 30, 2018, despite opposition from physician and patient groups who expressed concerns that the law will remove FDA safeguards.
The study was funded by a grant from the National Institutes of Health. The researchers reported research support from the Laura and John Arnold Foundation, Johnson and Johnson, Medtronic, the Blue Cross-Blue Shield Association, the FDA, and other federal agencies.
SOURCE: Puthumana J et al. JAMA Network Open. 2018 Jun 15. doi:10.1001/jamanetworkopen.2018.0283.