Early access schemes for pharmaceuticals have been increasingly implemented worldwide with the objective of satisfying unmet medical needs or facilitating market access for certain innovative drugs. It allows for pharmaceuticals which have not yet obtained their marketing authorization (MA) to be administered and reimbursed to a pre-defined population of patients. Under the French regulatory framework, this scheme consists in a window of unregulated prices, which stands in contrast with the system of administered prices for reimbursed pharmaceuticals. Our study aims at filling a gap in the literature by analyzing the French Temporary Authorization for Use (TAU) scheme since its implementation in 1994 up to 2016. This long time span allows to document and describe the TAU scheme and its impact on prices. In this article, we review the price difference between the freely set price under TAU and the post-MA price, after negotiation with the French Pricing Committee. Our main result shows that the 2007 regulatory change, which compelled pharmaceutical companies to reimburse the difference between the TAU price and the post-MA price, is significantly correlated with decreasing or stable post-MA-to-TAU price ratios.
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)…
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