EFPIA response to the EMA Regulatory Science Strategy to 2025

EFPIA welcomes the opportunity to comment on the Agency’s Regulatory Science Strategy (RSS) to 2025. Within our response EFPIA has identified a series of priorities that the innovative biopharmaceutical industry believes will have the most substantive benefit for European citizens, as well for the global community, by ensuring that Europe remains at the frontier of innovation in healthcare. EFPIA represents 39 research-intensive biopharmaceutical companies committed to researching, developing and bringing new medicines to patients to improve patients’ health and quality of life. EFPIA also includes 36 National Association members.

In total, there are over 7000 medicines in development (1). In Europe, these 40 companies invest 35 billion Euros in R&D (2), which comprises over three-quarters of all health industries R&D (44.8 billion Euros) (3). Once effectively implemented, EMA’s RSS 2025, will be a key enabler for bringing this promising next wave of medical innovation to patients who continue to face the burden of unmet medical need.

EFPIA supports advances to the regulatory environment essential for innovation, focused on four key themes: ensuring a competitive world class system; evolving the framework for innovation; elevating patient engagement; and expanding global convergence. This strategy forms the foundation for industry’s input to the RSS consultation. EFPIA would like to stress that these highlighted priorities are equally applicable to the direction, urgency and future skills required by the EU national competent authorities and HMA through the Multi-Annual Work Plan.

All five strategic goals within RSS 2025 address important priorities for the advancement of medicines and therapeutic care in Europe. In many cases, the recommendations set out by EMA are interrelated and interdependent. In practice, pursuing one recommendation may imply the need to progress others. Following EMA’s reflections from this consultation, industry anticipates the release of EMA’s 5-year implementation work plan, which will detail the Agency’s priority operations to action its regulatory science strategy.

EFPIA has concentrated on prioritising the objectives that the innovative pharmaceutical industry believe will best enable the delivery of novel medicines to patients – through the most effective, efficient, technologically advanced, informed means possible. While EFPIA acknowledges all of EMA’s proposals, EFPIA’s identified top priorities align with its mission to bring a continuously improved wave of innovative medicines to patients. EFPIA’s overall top three priorities from amongst EMA’s “core recommendations” are found in the first three of the five strategic goals.

EFPIA’s Top Three Priorities for RSS 2025 are: [Number of the recommendation indicated in parenthesis]
  • Foster innovation in clinical trials (2.2)
  • Diversify and integrate the provision of regulatory advice along the development continuum (1.7)
  • Promote use of high-quality real-world data (RWD) in decision making (3.4)

Along with these priorities, EFPIA wishes to emphasise the RSS 2025 recommendation to Support developments in precision medicines, biomarkers and ‘omics’ (1.1) with a view to advance the concept of personalised healthcare.

EFPIA also highlights the recommendations to Facilitate the implementation of novel development and manufacturing technologies (1.4) and Optimising Capabilities in Modelling, Simulation and Extrapolation (2.6). Manufacturing of medicines is evolving to embrace new models such as continuous manufacturing and stakeholders should further collaborate to advance these approaches.

Moreover, EFPIA recognises the importance of addressing better coordination in the provision of advice between regulatory authorities and HTA bodies. EMA has undertaken considerable efforts to bridge the coordination gaps between decision makers, and there have been some gains achieved. However, in order to deliver a step-change in consistent, aligned decision-making for the benefit of patients, it will require a refreshed approach (including greater involvement of patients and healthcare professionals), recognizing that regulatory, HTA and reimbursement processes and decisions are separate and occur at different stages of development.

EFPIA continues to value and rely upon EMA’s delivery of 5-year strategic and work plans. The process to deliver RSS 2025 has been the most comprehensive regulatory science strategy development to date including the public workshop and extended consultation period. This has allowed the industry to undertake a robust approach to offer these priorities and comments. As such, EFPIA would value the continuation of the EMA’s stakeholder engagement, including the full participation of HMA/NCAs, frequent status updates and outreach technology platform meetings, throughout the 5-year implementation phase of the RSS 2025 plan.




(3) Table 3.2 p. 45