A study carried out by Bioethics International has found that 76% of the drugs provided to patients through Expanded Access eventually go on to receive FDA approval.
Bioethics International is a company interested in the ethics, patient-centricity, and governance of how medications are researched, developed, marketed, and then made available to patients, both domestically and on a global scale. Their work addresses the roles of the multiple players involved in the healthcare industry including drug companies, hospitals, pharmacies, pharmacy benefit managers (PBMs), universities and research organizations, ethics committees, patient advocacy groups, etc.
The objective of this recent study released by the company was to “determine the characteristics of ‘expanded access’ and ‘compassionate use’ programs registered in ClinicalTrials.gov and to determine the percentage of drugs provided through these programs that ultimately received FDA marketing approval.”
To meet this objective, the company cross-referenced the drugs found in Expanded Access or Compassionate Use Programs registered on ClinicalTrials.gov with the drugs found on the FDA-approved drug list on Drugs@FDA, which shows all drugs approved since 1939.
The study found that, as of July 2016, there were 389 Expanded Access or Compassionate Use Programs registered on ClinicalTrials.gov. These 389 programs were administered to cover over 460 unique conditions, with 71% of the programs providing access to drugs, 11% providing access to biologics, and 10% providing access to medical devices.
However, the big takeaway from this study, especially from the drug manufacturer point of view, is that the majority of the drugs provided through Expanded Access do go on to receive FDA approval. The programs registered on ClinicalTrials.gov provided access to 210 unique investigational drugs, 160 (76%) of which are now approved and commercially available to patients. Taking into account the fact that some of the drugs included in this study are still in the development stage and are not yet being reviewed for approval by the FDA, a 76% approval rating is a positive find.
The results of this study should help alleviate concerns that providing experimental drugs through expanded access can hurt drug manufacturers’ chances of eventually receiving marketing approval for their products.