EURORDIS contributes to early dialogue in Health Technology Assessment
Patients and other rare disease stakeholders are calling for early dialogue in medicinal product development, including the processes to determine value and price.
What is HTA?
Health Technology Assessment (HTA) involves assessing a medicinal product for potential coverage/reimbursement in healthcare programmes and is defined by the EUnetHTA as a “multidisciplinary process that summarises information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner. Its aim is to inform the formulation of safe, effective, health policies that are patient focused and seek to achieve best value“.
The EUnetHTA was established in response to Article 15 of Directive 2011/24/EU on the application of patients’ rights in cross-border healthcare, which stipulates that the EU support and facilitate cooperation and the exchange of information via a voluntary network between Member State designated national authorities or bodies responsible for Health Technology Assessment (HTA).
The objectives of the second EUnetHTA Joint Action, of which EURORDIS is a stakeholder, include early dialogue between HTA bodies and relevant stakeholders. Early dialogue pilots have been conducted in this framework.
Planting the SEED of Early Dialogue
In addition to cooperation and collaboration between the national HTA agencies, early and sustained dialogue is important between HTA agencies, health technology developers, payers, patient organisations, and regulatory bodies.
The SEED Consortium (Shaping European Early Dialogues for Health Technologies) is developing early dialogue pilots between HTA agencies and medicinal product developers to take place during the development phase of medications and medical devices. Early dialogue between HTA bodies and medicinal product developers could help facilitate faster, more equitable access to rare disease medicinal products. EURORDIS Treatment Information and Access Director and Health Policy Advisor François Houÿez is participating in the SEED Early Dialogues. The SEED Consortium, composed of 14 European HTA agencies with the French Haute Autorité de Santé as consortium leader, runs for 22 months, during which time ten early dialogues will be conducted. Since September, EURORDIS has already participated in one early dialogue.
Amongst the outcomes of the SEED Consortium are recommendations for a permanent model for conducting early dialogues, including methodological protocols and codes of conduct. Other organisations participating in the SEED Consortium, or in specific early dialogues, include the European Medicines Agency, regulatory bodies, payers, health professionals, other HTA agencies, and patient organisations.
HTA-EMA cooperation and parallel scientific advice
The European Medicines Agency (EMA) also collaborates with the EUnetHTA, including work on improving information on benefit-risks that could be optimised for HTA use.
The EMA has also established a pilot project of parallel scientific advice with HTA bodies, allowing developers of medicinal products to receive simultaneous feedback from regulators as well as HTAs on their development plans. In August, 2014, the EMA published a draft guidance on best practices for pilot parallel scientific advice procedures, based on outcomes from a workshop held in November 2013. EURORDIS Chief Executive Officer Yann Le Cam was present at this workshop, voicing the needs of rare disease patients across the EU for fast access to safe, effective, affordable medicinal products. He also called for increased and earlier dialogue between regulators and HTA bodies and greater patient involvement.
Louise Taylor, Communications and Development Writer, EURORDIS