London, 8th December 2016: The European Medicines Agency is hosting a stakeholder workshop on
The European Medicines Agency is
organising this workshop in collaboration with the European Commission to
gather views and proposals from stakeholders on the adaptive pathways
approach, in light of the practical experience gained during the pilot project
EMA ran between March 2014 and August 2016, and to plan the next steps in the
exploration of this concept.
The basis for considering adaptive
pathways for evaluating medicines is to meet the challenges underlying how to
resolve high unmet medical need with then aim of licensing products likely to
have a major impact on patient morbidity and/or life expectancy.
The desire is to find ways to reduce
unavoidable uncertainties as rapidly as possible for serious/rare debilitating
and life-shortening illnesses. There are clear ethical and scientific
challenges in using novel approaches to evaluate medicines without a decline in
the quality of evidence on the effectiveness and safety of new
treatments.
Products which might be considered for
use of adaptive evaluation pathways include both conventional medicines and
advanced therapy medicinal products (ATMPs). ATMPs are medicinal product which
involve either a gene therapy medicinal, a somatic cell therapy or a tissue
engineered product [see more on ATMPs at Directive 2001/83/EC as amended by the
ATMP Regulation 1394/2007].
Adaptive pathways contribute to an
expanded toolbox for evidence generation where conventional randomized
controlled trials are not-appropriate or practical. Principles include using
processes to allow rapid reaction to uncertainty by iterative development based
on ongoing analysis of pathway data [“rapid cycle analysis”]. As
secondary end-points are welcomed, these pathways require pre-planning across
the entire product life-span, including the post-marketing paths ie active
monitoring and management of on-market use.
The adaptive pathway approach is
dependent on earliest stage and ongoing multi-stakeholder networking,
underpinned by risk management plans.
Critical issues include
- resolving scientific trust in real world data and the need to develop mature,
robust registries
- defining and prioritising unmet medical needs
- agreement on acceptable risks and benefits early access worth it (vs.
- maintaining safety standards
- ensuring that benefits outweigh risks
- avoiding unrealistic expectations
- establishing practical criteria for reimbursement, especially when secondary
outcome measures form the basis for market authorization
The EACPT was founded 24 years ago and
now includes as members all national organisations for clinical pharmacology in
Europe, as well as organisations from further afield internationally. The EACPT
aims to provide educational and scientific support for the more than 4000
individual professionals interested in clinical pharmacology and therapeutics
throughout the European region, with its congresses attended by a global
audience. The EACPT also advises policy makers on how the specialty can
contribute to human health and wealth.
