title: Temporary authorization for use: does the French patient access programme for unlicensed medicines impact market access after formal licensing?
abstract: BACKGROUND
  To reach the French market, a new drug requires a marketing authorization (MA) and price and reimbursement agreements. These hurdles could delay access to new and promising drugs. Since 1992, French law authorizes the use of unlicensed drugs on an exceptional and temporary basis through a compassionate-use programme, known as Temporary Authorization for Use (ATU). This programme was implemented to improve early access to drugs under development or authorized abroad. However, it is suspected to be inflationary, bypassing public bodies in charge of health technology assessment (HTA) and of pricing.
  
  
  OBJECTIVE
  The aim of this study is to observe the market access after the formal licensing of drugs that went through this compassionate-use programme.
  
  
  METHODS
  We included all ATUs that received an MA between 1 January 2005 and 30 June 2010. We first examined market access delays from these drugs using the standard administrative path. We positioned this result in relation to launch delays observed in France (for all outpatient drugs) and in other major European markets. Second, we assessed the bargaining power of a hospital purchaser after those drugs had obtained an MA by calculating the price growth rate after the approval.
  
  
  RESULTS
  During the study period, 77 ATUs were formally licensed. The study concluded that, from the patient's perspective, licensing and public bodies' review time was shortened by a combined total of 36 months. The projected 11-month review time of public bodies may be longer than delays usually observed for outpatient drugs. Nonetheless, the study revealed significant benefits for French patient access based on comparable processing to launch time with those of other European countries with tight price control policies. In return, a 12 % premium, on average, is paid to pharmaceutical companies while drugs are under this status (sub-analysis on 56 drugs).
  
  
  CONCLUSIONS
  In many instances, the ATU programme responds to a public health need by accelerating the availability of new drugs even though this study suggests an impact of the programme on the market access of these drugs for which the standard administrative path is longer than usual. In addition, pharmaceutical companies seem to market compassionate-use drugs with a presumed benefit/risk ratio at a price that guarantees a margin for future negotiation.
authors.affiliation: Laboratory of Economics and Management of Health Organizations (LEGOS), Paris-Dauphine University, Place du Maréchal de Lattre de Tassigny, 75016, Paris, France. albane.degrassat-theas@eps.aphp.fr
authors.forename: Albane
authors.initials: A
authors.lastname: Degrassat-Théas
authors.forename: Pascal
authors.initials: P
authors.lastname: Paubel
authors.forename: Olivier
authors.initials: O
authors.lastname: Parent de Curzon
authors.forename: Claude
authors.initials: C
authors.lastname: Le Pen
authors.forename: Martine
authors.initials: M
authors.lastname: Sinègre
mesh_terms: D057176:Compassionate Use Trials / Q000191:economics / Q000331:legislation & jurisprudence*; D003365:Costs and Cost Analysis; D017277:Drug Approval / Q000191:economics / Q000331:legislation & jurisprudence*; D004345:Drug Industry / Q000191:economics / Q000331:legislation & jurisprudence; D005602:France; D006297:Health Services Accessibility / Q000191:economics / Q000331:legislation & jurisprudence*; D006301:Health Services Needs and Demand; D006801:Humans; D004364:Pharmaceutical Preparations / Q000191:economics / Q000600:supply & distribution*; D012051:Reimbursement Mechanisms; D013673:Technology Assessment, Biomedical / Q000331:legislation & jurisprudence; D013997:Time Factors
chemical_list: D004364:Pharmaceutical Preparations