Should patients in need be given access to experimental drugs?

Physician perspectives on compassionate use in pediatric oncology

“…The requests most often submitted via the compassionate use pathway are for oncology and hematology drugs, representing 37% of all compassionate use applications for an individual patient . Although some ethical, legal, and practical issues of accessing unapproved drugs have been previously discussed, little is known about pediatric oncologists’ experience with and knowledge about compassionate use . The US Government Accountability Office published a report that stated physicians, particularly those with little experience with compassionate use, may find the FDA website and application form difficult to understand .…”

“…5 Although some ethical, legal, and practical issues of accessing unapproved drugs have been previously discussed, little is known about pediatric oncologists' experience with and knowledge about compassionate use. 2,[6][7][8][9][10][11][12][13][14][15][16][17] The US Government Accountability Office published a report that stated physicians, particularly those with little experience with compassionate use, may find the FDA website and application form difficult to understand. 18 Previously speculated challenges include limited knowledge about compassionate use mechanisms, the administrative burden and potential confusion of applying to multiple entities and obtaining IRB approval, and differing policies among pharmaceutical companies.…”

Ethics review in compassionate use

“…Indeed, the value of data collected during the conduct of compassionate use is limited, especially compared to that of randomized controlled trials, the contemporary gold standard of drug efficacy and safety studies [19]. Of the two major regulatory agencies, the FDA does not consider compassionate use to be a significant source of reliable safety and efficacy data; while adverse event reporting is required, this does not affect the FDA’s decisions on eventual drug approval [4, 20]. The EMA allows safety data to be collected during compassionate use programs, while at the same time stressing that compassionate use cannot replace clinical trials for investigational purposes [6].…”

“…It is beyond the scope of this article to discuss the specific information that should be included on this form, but it should contain adequate information about the potential benefits and risks associated with the use of drugs with unproven safety and efficacy. While it is commonly accepted that informed consent should be required for compassionate use [4, 17, 27], independent review would ensure that informed consent forms really fulfill their purpose [4]. In practice, RECs have extensive experience of reviewing informed consent forms, and although they are principally involved in the evaluation of those used in biomedical research, the overall objective of the consent form in compassionate use is the same: to present a patient with all the risk/benefit information they need to make a truly informed choice about participation in a program.…”

“…In the 1980s, the original idea underlying the development of compassionate use was to administer an investigational drug outside of a clinical trial for the direct therapeutic benefit of a patient [3]. At present, such treatment can be performed in patients with very serious, including life-threatening diseases, who cannot be satisfactorily treated with approved drugs [4–6]. Compassionate use regulations have already been introduced in the USA, Canada, most European Union countries (e.g.…”