Characterizing expanded access and compassionate use programs for experimental drugs

Abstract

ObjectiveWe sought 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.ResultsWe identified 398 expanded access and compassionate use programs (hereafter referred to as expanded access programs) registered on ClinicalTrials.gov. Industry funded 61% (n = 241) of programs individually or collaboratively, while NIH and the US Federal Government rarely funded programs (3% [n = 11] and 2% [n = 6], respectively). Most programs provided access to drugs (71% [n = 282]), 11% to biologics (n = 43), and 10% to medical devices (n = 40). These programs covered 460 unique conditions, the most common being HIV (n = 26), leukemia (22), and multiple myeloma (n = 14). Only 2% of programs reported results in ClinicalTrials.gov. Most programs (82%) were open to enrolling adults and seniors (n = 326). These programs provided access to 210 unique experimental drugs, of which 76% have received FDA approval.

Physician perspectives on compassionate use in pediatric oncology

“…For example, recombinant activated coagulation factor VII (rFVIIa, Novoseven), used in patients with congenital factor VII deficiency, has pediatric use labeling which was supported in part with data from compassionate use studies. 37,38 This study has several important limitations. The results may not be representative of the experiences of the greater pediatric oncology community as the response rate was lower than previous surveys we conducted.…”

“…Though we did not define clinical benefit, our survey data underscore the need to track clinical efficacy of compassionate use cases, particularly for children, so that industry, government, and academic researchers can be best informed about investigational agents. For example, recombinant activated coagulation factor VII (rFVIIa, Novoseven), used in patients with congenital factor VII deficiency, has pediatric use labeling which was supported in part with data from compassionate use studies …”

Hidden duplicates: 10s or 100s of Indian trials, registered with ClinicalTrials.gov, have not been registered in India, as required by law

“…Otherwise, there will be a bias in favor of the results from records that are present in multiple registries. Aside from studies that may contribute to clinical guidelines, other analyses of large sets of registry records have also aimed to avoid double counting [22,[31][32][33].…”

Taking Psychedelics Seriously

“…33 , 34 Similarly, the FDA's expanded access or compassionate use provisions may make use of drugs that have not been approved available to patients who are otherwise facing death. 35 , 36 By diminishing a desire to die among people with severe depression, anxiety, PTSD, and those with terminal cancers, genetic and neurodegenerative diseases, psychedelics may have greater life-saving effects than other drugs that have earned right-to-try and expanded access status. 37 …”