Innovation and access to new medicines is one of the 11 key business priorities of the HMA Multi-annual Work Plan (MAWP).
Ensuring timely access to new beneficial and safe medicines for patients is considered essential by the network. It involves several steps as early detection of promising products, guiding an appropriate clinical development, tailored access adjusted to current knowledge during pre-authorization phases though clinical trials and/or compassionate use programs, efficient and fair regulatory processes and integration of all these activities with subsequent price and reimbursement decisions and the pharmacovigilance activities for the entire life cycle of medicines.
The contribution of this new subgroup will be of relevance for the progression of these initiatives in conjunction with HTAs, payers and other interested parties such as the industry, patients and Health Care professionals associations, to ensure an adequate and cooperative work. The integration of these actions at national level is needed for the correct functioning of the system.
The creation of a new HMA subgroup focused on timely access has been proposed in the MAWP to directly deal with a number of actions which have been proposed by HMA for achieving this goal, listed below:
- HMA should further explore the flexibilities that the EU regulatory framework offers for the early access of innovative products at national level. i.e. the participation in clinical trials (either phase II, phase III or expanded access clinical trials), compassionate use (either named-patient basis or cohort basis approaches), or off-label use as well as contributing to the further optimisation of conditional approval, exceptional circumstances approval or accelerated assessment (pathfinder support scheme) through the participation in the STAMP group and support to prime/adaptive pathways.
- In conjunction with EMA explore ways to harmonize the regulatory requirements of registries and defining circumstances for use of real world data (RWD) that may be potentially used in the authorization or modification of marketing authorizations.
- HMA will work in the following years to explore how to improve the involvement of patients/users, Health Care professionals and academic community in those regulatory activities which have an impact on them or on which they can influence. Moreover, the collaboration with other key bodies (such as HTAs, pricing and reimbursement authorities and payers) has to be reinforced to enable appropriate decision making and exchange of information to allow optimal market access.
- HMA should consider the necessity, feasibility and capability of agencies to generate independent data or reanalyse raw data on issues necessary to ensure the reliability of regulatory outcome.
Members and Representatives
ES, DE, NL, NO, HU, UK, BE, and the European Commission.
Maria Jesus Lamas, Spanish Agency of Medicines and Medical Devices (AEMPS)
Tel.: + 34 91 822 50 28
Activities and Achievements
In order to support the innovation and timely access to new medicines, the following activities are proposed:
- Discuss and generate a common framework of activities related with timely access at HMA level
- Explore early access activities at EU level such as compassionate use programs and/or clinical trials to identify potential areas for collaboration
- Explore the possibility of increasing the cooperation in off-label use and potential ways to integrate it into the regulation
- Collaborate with the EMA to optimize the timely access regulatory tools and integrate them into national circumstances
- Strength the collaboration with the different partners: HTAs bodies, pricing and reimbursement authorities, payers, Health Care Professionals and patients
- Assessment of emerging trends: RWE & registries.
Compassionate Use Programme in the MS | pdf