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 act on genetic biomarkers rather than any specific type of cancer. Even though EAPs and novel therapeutic approaches have brought huge benefits for public health, their implementation is limited by several challenges, including the high risk of safety-related label changes for medicines authorized through the accelerated procedure, the high costs, and the reimbursement and access concerns. In this context, regulatory agencies should provide the best conditions for the implementation of the described new tools.
Highlights
The development of a new medicine is a long, expensive and risky process
Despite the progress made in the field of clinical research, unmet therapeutic needs are still identified in several clinical areas (Miller, 2009; Taiwo et al, 2010; Aceves, 2014; Markowitz, 2015; Morrow et al, 2017)
Recent developments in clinical research have placed a series of challenges for regulatory agencies, which are required to create the best conditions for the implementation of the described new tools
Summary
The development of a new medicine is a long, expensive and risky process. The entire time that passes from the R&D phase until the drug’s marketing approval can last up to 15 years, and it is characterized by extremely high costs, usually exceeding $1.2 billion (Saadi and White, 2014). Once the new medicine is authorized, based on data demonstrating the positive benefit/risk profile, the realworld effectiveness and safety of the drug is assessed during phase IV studies (Auricchio et al, 2017; Mascolo et al, 2017). In this last phase, pharmacovigilance is included. During the premarketing phase, the efficacy and safety data are frequently evaluated using a non-inferiority or equivalence study design and surrogate outcomes Considering these limitations, the real value of a new drug can be confirmed only when it will be used in real life conditions (Oyinlola et al, 2016)
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