Abstract
To provide an overview of the practical requirements for patient access to new therapies before marketing approval (early access programs: EAP) and to compare recent implementation of these programs in three countries. Targeted narrative literature review was performed. Publications were identified on NCBI, PubMed, Google Scholar, grey literature and websites of regulatory agencies from France (ANSM), Germany (BfArM and PEI) and the UK (MHRA). Early access is possible in all three countries through compassionate use programs (CUP: ATU de cohorte, Härtefall-Programme, EAMS) or individual-basis prescriptions (IBP: ATU nominative, individuelle Heilversuche and supply of unlicensed medicinal products or “specials”), but their implementation differ. All countries require authorizations for CUP. IBP do not require a submission to regulatory agencies in Germany, while authorizations are required for “specials” in the UK. In Germany and in the UK, the manufacturer has to provide the drug free of charge for CUP without further national regulation about drug costs and reimbursement for IBP. In France, authorizations are needed for CUP and IBP and the healthcare system supports all related costs. In 2017 France's ANSM authorized 265 ATU, which were mainly targeting neurological diseases, severe infectious diseases, cancers, blood disorders and metabolic diseases. In Germany ongoing CUP in June 2018 are mostly targeting cancers and genetic diseases. Between April 2014 and November 2015, over 500 patients benefited from UK’s EAMS, which targeted cancers and heart failure. Reports regarding safety and efficacy of the drugs during CUP must be provided to regulatory agencies in all 3 countries. Early access programs in the 3 countries are similar in the types of access granted (for specific populations) and the diseases they target corresponding to high unmet medical needs. The main differences between countries are seen in the financing of these programs.
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