EURORDIS have launched a statement calling on European institutions to adopt appropriate measures to fully incorporate meaningful involvement of patients in EU cooperation on health technology assessment.

Patients and their organisations welcome efforts set out in the EU proposal for a new legislation on health technology assessment (HTA) to adopt high quality methods and standards when assessing health technologies they use for their own treatment.

However, patients and their organisations are convinced that changes need to be made to the legislation before it is adopted to guarantee an adequate involvement of patients in all HTA activities (joint consultations, early dialogues, scoping and assessments), which is essential to help HTA assessors determine the relative efficacy and safety of health technologies.

1. A MATTER OF QUALITY

Via a direct dialogue with patients, HTA assessors can better understand what important questions need to be answered: for complex diseases in particular, assessors need to hear from patients which relevant symptoms need to be improved and what impact they expect from the technology on their daily life. To fully interpret results of clinical trials and assess whether they are clinically meaningful, patients who are the end-user of the technology need to provide their views. Failure to consult patients adequately on this impact reduces the assessors’ ability to determine the therapeutic value of the technology.

The contribution of patients enables HTA bodies and experts to carry out a higher quality of work. In a domain where high uncertainties on the clinical benefit of new or older technologies exist, only direct discussions with clinicians and patients can clarify the incertitude.

2. ADEQUATE INVOLVEMENT

In contrast with the approach adopted by the European Parliament on that point, patients and their organisations consider that submitting comments in writing on complex documents with information of varying importance after all discussions have taken place, is not an adequate involvement. HTA relies on collective thinking, where different experts discuss different information, confront different interpretations, simultaneously and according to the same methods.

By ‘adequate involvement’, we mean participation in the discussions that take place prior to and while a medicine or technology is going through the HTA assessment, for example face-to-face meetings (joint consultations), focus groups with assessors for the scoping phase, telephone interviews with assessors, patients’ preferences elicitations techniques, and expert or citizen panels.

EUnetHTA, the European Network of HTA Agencies, is currently piloting various methods to adequately involve patients in HTA. These methods vary in their modalities, costs, and the information they might provide.

More research is needed to determine which methods to use to involve patients in the European Cooperation on HTA.

At the national level, more HTA agencies are using different methods to involve patients:

• G-BA, the German HTA authority, made the participation of patients in HTA a condition for a quality assessment: in accordance with the regulations set forth in the German Social Code, Book Five (SGB V), leading nationwide advocacy groups that represent patient interests are entitled to take part indiscussions or submit requests, and their opinion have to be included in the decision.
• In Austria, LBI-HTA organises focus groups with patients. The groups meet for 2 hours with HTAassessors and this proves to be much more efficient that sending comments in writing.
• Other Member States (Ireland, Spain, Sweden, United Kingdom) adopted guidance on patient involvement in HTA.

3. LEGITIMACY

The participation of patients in HTA committees or procedures is key for civil society to witness how HTA is conducted. This is a transparency requirement – patients and citizens have the right to verify that assessments are based on science and medical evidence.

Of all interested parties, patients have the most at risk: our health is at stake. Engaging patients in the Coordination Group and in the scientific subgroups of the European HTA Cooperation is a necessary measure to enhance mutual trust.

Patient involvement ensures that the dignity and legitimacy of people living with a disease is recognised.

Patients represented by the below groups are calling to participate in all forums where decisions that affect their lives are made, with equal credibility as other experts.

The role of patients is clearly stated in most EU legislations on pharmaceuticals. At the European Medicines Agency (EMA), the European legislator decided patients are members with full rights in the Committee for Orphan Medicinal Products (Regulation EC 141/2000 Art.4), in the Paediatric Committee (Regulation EC 1901/2006 Art.4), in the Committee for Advanced Therapies (Regulation EC 1394/2007 Art.21), in the Pharmacovigilance Committee (Regulation (EU) No 1235/2010 art.61), and in the Management Board (Regulation EC 726/2004 Art.65).

To our opinion, Recital 18 of Regulation EC 726/2004 is valid for all EU health agencies and institutions: The structure and operation of the various bodies making up the Agency should be designed in such a way as to take into account the need constantly to renew scientific expertise, the need for cooperation between Community and national bodies, the need for adequate involvement

of civil society, and the future enlargement of the European Union. The various bodies of the Agency should establish and develop appropriate contacts with the parties concerned, in particular representatives of patients and health-care professionals.

Why should patients be full members in regulatory structures, but only be permitted to submit comment in writing in HTA procedures?

4. NOTHING ABOUT US, WITHOUT US

It is not without reason that in 1983, AIDS advocates formulated the principles of Treatment Advocacy, which applies to all patient advocacy movements, whichever their type, status, or history, as follow:

• Be involved at every level of decision-making; and
• Be included in all forums with equal credibility as other participants, to share their own experiencesand knowledge.
  These principles are used to be summarised in the motto “Nothing about us, without us”.At present, patients have their word to say on all aspects that affect the development and the evaluation of treatments, by:
  • Being members of ethics committees for clinical trials;
  • Working with industry to improve the quality of clinical trials, discussing the design, the doses, the outcomes, and the protocol in all its aspects;
  • Being full members of scientific committees at the European Medicines Agency;
  • Being involved at national level by HTA agencies in the most modern Member States, from the selection and the assessment of evidence to the appraisal; and
  • Contributing in pharmacovigilance by reporting on side effects and testifying about them.