The balance between investigational new drug access and protection of patients from therapies without established safety the FDA built during the past 20 years could be compromised by the new Right to Try law, a new study in JAMA Network Open suggests.
“To our knowledge, no studies have examined the timing and duration of drugs made available through expanded access programs to determine whether the program was serving its original purpose,” Jeremy Puthumana, MS, of the division of medical ethics at Yale School of Medicine, and colleagues wrote.
Researchers used ClinicalTrials.gov to locate investigational medicines made obtainable through expanded access and compassionate use programs prior to FDA approval and ascertained the start date of each program and several “key regulatory dates” — investigational new drug application activation, initial NDA submission and FDA approval — and timing and duration of expanded access availability in regard to NDA submission and FDA approval.
There were 92 drugs from September 1996 when the initial expanded access program began through Aug. 1, 2017, that researchers studied. Those used to treat cancer made up half of the drugs studied, whereas genetic, metabolic and endocrine diseases (n = 16) and infectious diseases (n = 14) made up the rest.
Puthumana and colleagues found that the median premarket expanded access availability was 10 months (interquartile range, 6-19.5), comprising a median of 14% (interquartile range, 7-25) of the premarket clinical development period (investigational NDA activation to FDA approval).
In addition, of the 92 expanded access programs:
- 64 were begun immediately prior or after NDA submission;
- 40 were begun in the 6 months after the submission; and
- 24 were begun during the 6-month period before the submission.
“For medicines that ultimately receive FDA approval, these findings suggest that the FDA and pharmaceutical industry have established a balance between investigational new drug access and protection of patients from therapies without established safety,” Puthumana and colleagues wrote.
The balance between investigational new drug access and protection of patients from therapies without established safety the FDA built during the past 20 years could be compromised by the new Right to Try law, a new study in JAMA Network Open suggests. Photo Source: Adobe
“This balance may be compromised by policymakers seeking to speed access to investigational medicines through the Right to Try Act,” they added.
Despite more than 100 advocacy groups, including the American Lung Association and American Society of Clinical Oncology sending a letter to Congress strongly opposing the law, the bill passed both chambers. President Donald J. Trump signed Right to Try into law on May 30. An expert who spoke to Healio before the bill signing indicated the effect of the law on patients remained unclear. – by Janel Miller
Puthumana reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures.