One area of successful European leadership is the adoption of the ‘adaptive pathways’ or ‘medicines adaptive pathways to patients’ (MAPPs) approach, which forms the basis of the European Medicines Agency’s (EMA) efforts to improve timely access to medicines. Simply put, MAPPS / adaptive pathways involves the early European-wide approval of a medicine for a restricted patient population based on small initial clinical studies, followed by progressive expansion of the market authorisation and patient access based on confirmatory real-world evidence and further research. Such adaptive approaches have been developed to meet demands from patients with serious unmet medical needs and to facilitate drug development as medical advances such as genomics and personalised medicine increasingly fragment indications and patient populations.
In principle, adaptive pathways will generate data to fulfil both the efficacy requirements for authorisation and the effectiveness needs of national health technology assessments (HTA), while providing patients with needed therapies in the most efficient timescale and trial size possible. This is not merely a new form of licensing, but involves a prospective plan, tailored for the product and disease, encompassing clinical development, licensing, reimbursement, clinical use and monitoring. As such, adaptive pathways can help strengthen confidence and aid financial projection over the product’s life-span, whilst avoiding the current ‘all or nothing’ approach to drug development.
Adaptive pathways require agreement between all stakeholders regarding the benefit/risk assessment of a new medicine, the remaining uncertainties, and the necessary evidence-gathering plan following the initial approval. This collaborative approach represents an opportunity for industry to engage with regulators, HTA authorities, payers, practitioners and patients organisations throughout the development process. The approach is already supported by the European Federation of Pharmaceutical Industries and Associations (EFPIA) and other pharma industry bodies, e.g. the Association of the British Pharmaceutical Industry (ABPI), BioIndustry Association (BIA) and the Centre for the Advancement of Sustainable Medical Innovation (CASMI).
In February 2015, EMA closed Stage I of its adaptive pathways pilot project, from which selected applications will progress into in-depth discussions. EMA reported its experience to the Safe and Timely Access of Medicines to Patients (STAMP) expert group of the Pharmaceutical Committee. Going forward, EMA will only consider new applications that are “very well developed and present elements for discussion by all stakeholders”. There are certainly a number of challenges that will need to be addressed for the concept to function within the current regulatory framework and to be accepted by payers and HTA bodies, but the EMA pilot project is a promising development that needs to be watched closely.