The New Paradigms in Clinical Research: From Early Access Programs to the Novel Therapeutic Approaches for Unmet Medical Needs

Frontiers in Pharmacology
Cristina ScavoneAnnalisa Capuano

Abstract

Despite several innovative medicines gaining worldwide approval in recent years, there are still therapeutic areas for which unsatisfied therapeutic needs persist. For example, high unmet clinical need was observed in patients diagnosed with type 2 diabetes mellitus and hemophilia, as well as in specific age groups, such as the pediatric population. Given the urgent need to improve the therapy of clinical conditions for which unmet clinical need is established, clinical testing, and approval of new medicines are increasingly being carried out through accelerated authorization procedures. Starting from 1992, the Food and Drug Administration and the European Medicines Agency have supported the so-called Early Access Programs (EAPs). Such procedures, which can be based on incomplete clinical data, allow an accelerated marketing authorization for innovative medicines. The growth in pharmaceutical research has also resulted in the development of novel therapeutic approaches, such as biotech drugs and advanced therapy medicinal products, including new monoclonal antibodies for the treatment of asthma, antisense oligonucleotides for the treatment of Duchenne muscular dystrophy and spinal muscular atrophy, and new anticancer drugs that...Continue Reading

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Citations

Nov 7, 2019·Applied Health Economics and Health Policy·Caridad PontesBrian Godman
May 12, 2020·Frontiers in Pharmacology·Rachel J TysonDaniel Gonzalez
Oct 30, 2020·The Journal of Pharmacology and Experimental Therapeutics·Kelly A FaderVishal S Vaidya
Oct 15, 2020·International Journal of Molecular Sciences·Dmitry A FilimonovVladimir V Poroikov
Mar 20, 2021·Cell Chemical Biology·Rebecca E HughesNeil O Carragher

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Methods Mentioned

BETA
SMA
antisense oligonucleotides

Software Mentioned

Adaptive

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