Market access in the EU, do we have enough evidence?

• Autores: Teresa Gómez LLuch, María Dolores Fraga Fuentes, Clara Notario Dongil, Patricia Nieto-Sandoval Martín de la Sierra, José Luis Sánchez Serrano, Juan Carlos Valenzuela Gámez
• Localización: European journal of clinical pharmacy: atención farmacéutica, ISSN 2385-409X, Vol. 22, Nº. 1, 2020, págs. 39-43
• Idioma: inglés
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• Resumen

  • Objectives: The appearance of new drugs needs to represent 'a necessary option, however involves a challenge for healthcare decision makers. EMA is responsible for the scientific evaluation based on many aspects, but later there can be discrepancies between European drug approval and subsequent use. Our objective was to review EMA marketing authorizatioti of new active substance (NAS) and analyze the conditions and evidence of these. Also, we analyzed the opinion and reports of this NAS in The Spanish National Healthcare System. Method: We analyzed NAS who had received a positive opinion by EMA recommending the granting of a marketing authorization from 1st January 2014 to 31st December 2017, according to the dates of authorization details reflected on the EMA's website. We compiled: therapeutic area, indication, type of approval, route normally used, designation obtained and pivotal trials of drugs, according to their design features design, their clinical phase and the comparator, in order to evaluate the evidence provided. It was verified the existence of reports in the Spanish Dngs Agency and we analyzed its conclusions as well as the financing conditions. Results: A total of 136 NAS were approved, mainly in the ancology area. From them, 22 (16.2%) were conditional or exceptional approval. Otherwise, 22 (16.2%) of them were carried out by accelerated route. Of these drugs, 50 (36.8%) were authorized under orphan designation and 51 of the approved indications included at least a pivotal essay in phase I or II. Also, 54 trials included a placebo as a comparator and 27 are single-arm-essays. The Spanish-Drugs-Agency elaborated 132 reports. Most of them concluded that drugs were similar to than alternatives, considering their us; based on efficiency. Finally, 46 indications were not financed or financed under conditions. Conclusions: Our study shows that when a new drug is authorized, the evidence is low. This leads to uncertainties of efficacy and safety. The evaluation of the value is fundamental in the health system, and strategies are needed to improve the final outcomes with the limited available resources