In most countries, medicinal products must obtain regulatory approval before being marketed. The regulatory process and evidence requirement for market access of health technologies have been shown to be substantially lengthy in some countries (Hirako et al., 2007; Downing et al., 2012; Nitin Kashyap, Gupta, and Raghunandan, 2013). The health technology appraisal, from the compulsory benefit-risk assessment to the conditional cost-effectiveness evaluation, is the first step in the regulatory procedure. In addition, eligibility to reimbursement and the price setting procedure (with the negotiation of medicinal products’ prices) further delay the medicine’s market access. The price regulation and reimbursement policy of medicinal products directly impact patient access to pharmaceuticals. Countries with tighter price and reimbursement policies may thus suffer substantial delays in their effective access to pharmaceuticals (Ades et al., 2014; Richter, 2008; Danzon and Epstein, 2012). In this context, Compassionate Use Programs (CUPs) have been developed to reduce the delay in obtaining the drug in case of severe diseases, when no suitable authorized alternative therapy exists. CUPs are the only way of gaining an early access to promising but not yet market-authorized medicines for patients who are not eligible to clinical trials.
In Europe, France has the longest history of CUPs, with its Temporary Authorizations for Use (TAUs or Autorisations Temporaires d’Utilisation in French) scheme set up in 1994 (Law N°92-1279, 1992)…