‘Right-to-Try’ Could Upset Access/Safety Balance: Study
Yale University researchers who looked at 92 expanded drug access programs registered at ClinicalTrials.gov over the last two decades say that the balance established by FDA and the drug industry between investigational new drug access and protection of patients from therapies without established safety could be compromised by policy makers seeking to spread access to investigational medicines through the Right to Try Act. Writing online in the Journal of the American Medical Association, the researchers say the law will remove the requirement for FDA oversight and approval of expanded access requests.
In what the researchers believe is the first study to examine the timing and duration of drugs made available through expanded access programs to determine whether the program was serving its original purpose, the study found that 69.6% of expanded access programs were started within the six months preceding or following NDA submission and overall the medicines were available to patients for 14% of their clinical development period.
“Our findings suggest that these medicines are generally made available after clinical research needed for FDA approval is completed or is nearing completion, around the time of NDA submission,” the report says. “Thus, for medicines that ultimately receive FDA approval, expanded access programs over the past two decades are providing access to investigational medicines for which safety and effectiveness have been established.”
The authors say that the Right to Try Act encourages sponsors to make investigative medicines available earlier in the clinical development period, in part because it shields companies from any liability associated with patient injury due to expanded access. “Nevertheless,” they say, “under both the FDA expanded access program and the proposed Right to Try Act, sponsors may still refuse access to their investigational medicines. Therefore, legislative efforts aiming to safely ensure drug availability should work to involve both investigational medicine manufacturers and FDA. Such legislation may help patients with life-threatening conditions gain access to investigational medicines without compromising patient safety or the process for evaluating drug efficacy and safety established by FDA.”