Demystifying HTA: patients’ role in new legislation
Earlier this year, the EU Regulation on Health Technology Assessment (HTA) – a deliverable of the EU Pharmaceutical Strategy – entered into force, marking an important step towards improving access to innovative technologies. However, this does not mean the legislation applies immediately – a three-year transitional period has been set up during which a new governance framework will be established to ensure effective application across the EU as of 2025. 
The new Regulation, which has been a key EURORDIS advocacy priority, does not come out of nowhere. It is built upon a system of voluntary cooperation between the Member States, which has taken shape through EU-funded initiatives – HTA Network and the EU-funded EUnetHTA Joint Actions. The legislation, therefore, formalises existing partnerships by introducing a permanent framework for joint work that will also cover joint scientific consultations, the identification of emerging health technologies, and voluntary cooperation, as well as work on joint clinical assessments.
Ahead of the first meeting on 21 April of the new EU HTA Coordination Group – a governing body whose composition is yet unknown – we are taking the opportunity to go back to basics and remind ourselves what HTA is, what the role of the patient community has been and why patients’ input is so critical for the new Regulation to succeed.
Remembering the basics: what is HTA?
In health care, the definition of health technologies is fairly broad, but it usually includes medicines, medical devices, diagnostic tools, and surgical procedures, as well as measures for disease prevention, screening, diagnosis, treatment, and rehabilitation.
Health Technology Assessment is the systematic evaluation of properties, effects, and impacts related to the use of health technologies. HTA Cooperation under the new Regulation, however, will only concern medicines and implantable and invasive devices. For patients, this means when medicine gets market authorisation by the EMA (European Medicines Agency), national HTA bodies (the payer) assess its therapeutic effect, potential side effects, influence on quality of life and means of administration.
Still to this day, when a new health technology comes in, each Member State (MS) has its own process to decide whether to reimburse it or not. EU citizens accept that different decisions are made in different countries, as different MS invest different budgets in healthcare, and health is not awarded the same budget priority in all MS. However, different agencies use different sources of data and different methods to assess the exact same technology and frequently reach different conclusions. This leads to confusion among the patient community and, in many cases, a lack of transparency in methods exacerbates this effect. Without close cooperation, no Member State can assess clinical aspects of all health technologies for which decisions need to be made each year, leaving millions of patients across Europe in medical jeopardy.
What’s happening now?
For almost 20 years the European Commission has supported Member States’ cooperation on health technology assessment (HTA). This consisted in assessing the effectiveness of new therapies once and jointly, by sharing expertise and avoiding duplication. It happened in the form of voluntary cooperation (and three Joint 
Actions), which since 2011 had its legal background in article 15 of the Patients’ Rights in Cross-border Healthcare EU Directive.
Now we are witnessing a new course with the adoption of the Regulation on European Cooperation on HTA, which creates a permanent framework for Member States to perform joint HTA at the European level, so as to get more consistent and robust information for their national health policies.
Long story short, the European Union has introduced a single European Union system for clinical HTAs, with mandatory cooperation between Member States on clinical HTA assessments. This aims to ensure greater transparency and patient engagement in the HTA process, improve the availability of health technologies with added value for patients across the European Union, due to more timely and evidence-based decision making, and reduce healthcare-related risks.
The provisions of the Regulation will become applicable for medicinal products with new active substances for oncological indications and advanced therapy medicinal products (ATMPs) from January 2025 and for all orphan drugs from 2028 onwards. Once fully implemented by 2030, all medicines registered by the European Medicines Agency will have to go through a joint clinical assessment. However, this will be limited to examining the clinical evidence only. Pricing and reimbursement (P&R) decisions, based on these joint assessments, would remain the responsibility of national-level governments.
It’s also worth noting that as of 2022 EMA offers joint scientific consultations for medicine developers, regulators, HTA bodies and other stakeholders to discuss the (sponsor’s) development plans and help generate optimal and robust evidence that satisfies the needs of both regulators and HTA bodies. And while these consultations do not have a legally binding effect, they facilitate early dialogue which enables patient access to important new medicines and benefits overall public health.
Patients’ role in shaping the new legislation
Since the early 2000s, EURORDIS has contributed to the effort of establishing appropriate cooperation between patients and health institutions, at both European and national levels. It has particularly promoted and supported the involvement of patients as members of the scientific committees at the European Medicines Agency and as experts in any regulatory procedures for medicines development and approval. To succeed in this ambition, EURORDIS since 2008 has organised training for patient advocates – the EURORDIS Summer School – with a focus on Research & Development, Regulatory and HTA.
Additionally, over three Joint Actions, from 2010 to 2021, EURORDIS has been actively participating in the work of EUnetHTA as a partner in the SEED project (Shaping European Early Dialogues for health technologies), and as a member of the EUnetHTA Stakeholder Forum (in its different configurations) and of the EUnetHTA Task Group for Patient involvement (2018-2020).
EURORDIS has been an active EUnetHTA contributor and has actively participated in policy discussions among Member States representatives within the HTA Network – chaired by the European Commission – over a harmonised and permanent framework for a European Cooperation on HTA.
Patient engagement under the new Regulation
With the approval of Regulation 2282/2021 (HTA Regulation), Member States will have a permanent framework and common rules to perform HTA jointly at the European level. The HTA Regulation also establishes quality standards for the joint work: among them, it requires the systematic and timely participation of patient experts in the procedures of the new Cooperation, especially in the main activities, such as Joint Scientific Consultations and Joint Clinical Assessments.
Furthermore, when patients are duly involved, they are also witnesses to the manner HTA is conducted. Part of the implementation effort should, therefore, consist in preparing the conditions for a timely and appropriate involvement of patients in the joint work. Training will be key, and so will be clear guidance on how to involve patients in European procedures, beyond a simple juxtaposition of existing national rules. This will help patient organisations respond to the upcoming requests for the identification of patient experts and build their competencies to provide relevant input in various procedures.
All this puts the patient community in an ambitious and onerous position. Especially in rare diseases, where patient inputs are a not-to-be-missed element of HTA, and the timely identification of the right patients to be involved in joint HTA procedures will be one of the keys to the success of the HTA Cooperation.
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Don’t miss out on a chance to hear from our Information & Access to Therapies Director, François Houÿez, and learn how patients could best prepare for their involvement in future European HTA activities at the Conference on “Health Technology Assessment – What’s next”. This event, organised by the European Commission, will take place on 22nd June 2022 in Brussels, but remote participation is possible. Registration closes on Friday 17 June: https://htaconference.eu/registration
Stanislav Ostapenko, Communications Senior Manager, EURORDIS