title: Available Tools to Facilitate Early Patient Access to Medicines in the EU and the USA: Analysis of Conditional Approvals and the Implications for Personalized Medicine.
abstract: Scientific knowledge and our understanding of the human body and diseases have limited any possible treatment tailoring to each patient. The technological advances enabling the integration of various data sets (e.g. '-omics', microbiome, epigenetics and environmental exposure) have facilitated a greater understanding of the human body, the molecular basis of disease and all the factors influencing disease onset, progression and response to treatment, thereby ushering in the era of personalized medicine. We evaluate the regulatory approaches available to facilitate early patient access to efficacious and safe compounds in the EU and the USA in order to make more informed recommendations in the future as to the gaps in regulations for early patient access. An in-depth analysis of conditional approvals (EU) and accelerated approvals (USA) is performed based on the publicly available information (European public assessment reports and a summary review of products approved under both programmes). The types of product, indications, time to approval and type of evidence submitted were analysed. Between 2007 and early 2015, 17 products were conditionally approved in the EU and 25 in the USA, most of them in the area of oncology and based on evidence from phase II clinical trial data. Early approval of promising products based on data from early phases of development is already possible in the EU and the USA. Some of the improvements could entail implementing a rolling assessment of evidence in Europe and extending the scope of early dialogues.
authors: Maastricht University, Maastricht, The Netherlands.
authors: Lada
authors: L
authors: Leyens
authors: Étienne
authors: É
authors: Richer
authors: Øyvind
authors: Ø
authors: Melien
authors: Wolfgang
authors: W
authors: Ballensiefen
authors: Angela
authors: A
authors: Brand
mesh_terms: D035843:Biomedical Research / Q000191:economics / Q000458:organization & administration; D002986:Clinical Trials as Topic; D003362:Cost-Benefit Analysis; D017277:Drug Approval / Q000191:economics / Q000379:methods / Q000458:organization & administration*; D055808:Drug Discovery / Q000191:economics / Q000331:legislation & jurisprudence*; D005060:Europe; D005062:European Union; D005544:Forecasting; D006297:Health Services Accessibility; D006801:Humans; D008389:Marketing of Health Services / Q000191:economics / Q000331:legislation & jurisprudence / Q000458:organization & administration; D057285:Precision Medicine; D018570:Risk Assessment; D012955:Societies, Medical; D014481:United States; D014486:United States Food and Drug Administration