Pharma Design Ltd has been a supporter of integrated Regulatory and Market Access advice in EU since its foundation in 2009. The progress made since then on a parallel EMA and HTA assessment is therefore welcome, although we feel that a lot of work still needs to be done and many hurdles overcome especially on the HTA side, before we can see a fully-functioning harmonized Market Access for Medicines in Europe. Anyway, last week EMA and EUnetHTA have reached an important milestone in bridging evidence-based analysis of results from both clinical efficacy as well as reimbursement-related effectiveness data. This has immediate tangible effects on how Companies will prepare for filings in EU.

As the first step to market access, a new medicine requires a marketing authorisation from a medicines regulatory agency. Following regulatory approval, Health Technology Assessment (HTA), providing evidence-based information and analysis, takes place at the national level States in accordance with national practices and legislative frameworks. Health Technology Assessment is then used to inform subsequent decisions on coverage (reimbursement) and price of an authorised drug at the national level.

Interactions between medicines’ developers, Regulators and Health Technology Assessment Bodies (HTABs) or other possible stakeholders to discuss the development plan means that evidence can be generated to meet the needs of respective decision-makers as efficiently as possible. Thus, a strong interaction between Regulators and HTABs/other relevant stakeholders is critical to facilitate patients’ access to important new medicines and hence for the overall benefit of public health.

The European Medicines Agency (EMA) is the EU body responsible for coordinating the existing regulatory and scientific resources put at its disposal by EU Member States for the evaluation, supervision and pharmacovigilance of medicinal products, including the provision of Scientific Advice for regulatory purposes.

The European Network for Health technology Assessment (EUnetHTA) was established to create an effective and sustainable network for HTA across Europe – working together to develop reliable, timely, transparent and transferable information to contribute to HTA in European countries, creating a sustainable system of HTA knowledge sharing, and promoting good practice in HTA methods and processes. Its main objective is to help to generate, all along the technology lifecycle, optimal and robust evidence for different stakeholders, bringing benefits for patient access and public health.

HTABs have performed several multi-HTABs Early dialogues (ED) in the framework of EUnetHTA Joint Action 2. Between 2013 and 2015, under the coordination of Haute Autorité de Santé (HAS), France, 14 HTABs took part in the Shaping European Early Dialogues for health technologies (SEED) project. Financed by the EC, the SEED project aimed to perform 10 Early Dialogues and explore possible scenarios for conducting Early Dialogues in the future. Associated with the SEED project, EMA took part in 4 of these dialogues as parallel EMA SEED advice procedures. Results from the EUnetHTA JA2, the SEED project, as well as the results of the Best Practice parallel regulatory-HTA Scientific Advice pilot since 2010 and the public consultation, have been taken into account to revise this workflow/process to better meet the needs and objectives of Parallel Scientific Advice/ Early Dialogues.

New EUnetHTA and EMA platform on evidence generation interactions

This new platform comprises enhanced collaboration for Parallel regulatory HTA Scientific Advice/Early Dialogues (henceforward referred to as Parallel Consultation) between EMA and EUnetHTA. Parallel Consultation provides a single gateway for requests for parallel discussions before the start of pivotal clinical trials on initial evidence generation for Marketing Authorisation Application/Reimbursement, and Post Licensing Evidence Generation (PLEG) involving EMA, EUnetHTA and HTA bodies. Partnership of EMA and EUnetHTA also allows for: streamlined logistics, improved HTA coordination through EUnetHTA ED Secretariat, greater participation via the involvement of EUnetHTA HTA Early Dialogue Working Party (EDWP) 2, and maximum gain from the parallel procedure by optimising opportunities for mutual understanding and problem solving between Regulators and HTAs. This facilitates optimal and robust evidence generation for different stakeholders bringing benefits for patient access and public health.

For all submitted requests, the EUnetHTA ED Secretariat facilitates centralised HTA recruitment, and selection criteria is applied by the EDWP in order to decide if the ED will follow the Consolidated or Individual pathway.

Therefore with immediate effect, all Parallel Scientific Advices will change to this Parallel Consultation Platform.

This guidance replaces the “Best practice guidance” on Parallel EMA-HTA Scientific Advice procedure (EMA/502692/2015) as of the date of publication.

Further updates of this guidance are expected with the advent of sustainable funding mechanisms for HTA early dialogues within JA3, and post JA3 in 2020.