Generating evidence from expanded access use of rare disease medicines: challenges and recommendations 231 11 ♠ ‘compassionate use versus clinical trials’ to address this issue: 'From a methodological point of view, clinical trials are practically the only means of obtaining reliable and interpretable efficacy and safety data for a medicinal product. Although safety data may be collected during compassionate use programmes, such programmes cannot replace clinical trials for investigational purposes. Compassionate use is not a substitute for properly conducted trials.'2 But this section does not foreclose the use of expanded access data as a supplement to clinical trial data, rather than as a replacement for them. We are not aware of any evidence of companies or physicians bypassing trial guidelines and conducting expanded programs instead – some companies have refused expanded access requests to avoid jeopardizing trial enrollment.6 Some worry, however, that allowing limited use of expanded access data could lead to increasing calls to broaden use of expanded access data. Illustratively, Belgian authorities describe a ‘Frequently Asked Question’, ‘Could we apply for a Compassionate Use Program (CUP) or Medical Need Program (MNP) in place of an extension trial/open label study?’ Such concerns have led some countries to prohibit data collection through sponsors on expanded access studies. In earlier versions of this FAQ, the Belgian authorities responded that: ‘no other data except pharmacovigilance data can be gathered which will only be used for the evaluation of the (..) program.’43 This even precluded the use of safety data for purposes other than the evaluation of the expanded access program. In more recent versions, this has changed to: ‘data collected (…) that are necessary for the conduct of the program (e.g. to check inclusion/ exclusion criteria, to follow-up the B/R (benefit/risk) of a patient, pharmacovigilance data) could be used to enlarge the understanding of the treatment. It is not possible to collect more data than strictly needed for the conduct and evaluation of the program.’4 Similarly, Austria prohibits data collection in a named-patient setting (‘Heilversuch’) stating that: ‘named patient use is intended to facilitate the urgently needed treatment of a specific patient to avert a life-threatening or chronically debilitating situation. Systematic collection of data on safety and efficacy of the medicinal product used is not legally acceptable in this framework.’6 Through our correspondence with regulators, we learned that Sweden does not allow data collection at all, and that Canada does not ‘condone’ data collection. Nevertheless, several publications on expanded access programs originate from Austria, Sweden, Belgium, and Canada.44–51 These paradoxes demonstrate the unclear position of expanded access in evidence 6 https://www.fiercebiotech.com/biotech/biogen-holds-firm-denying-compassionate-use-for-experimental-als-drug
RkJQdWJsaXNoZXIy MTk4NDMw