The European Medicines Agency has been working closely with health-technology-assessment (HTA) bodies since 2008. HTA bodies provide recommendations on the medicines and other health interventions that can be paid for or reimbursed by the healthcare system in a particular Member State. Recently, they have been gaining a greater influence on the access of novel medicines to patients, mainly due to increased pressure on healthcare budgets.
HTA bodies carry out their own assessments of medicines once they have received a marketing authorisation. In contrast to the benefit-risk assessment carried out by regulators, HTA bodies compare the relative effectiveness of medicines, in order to assess their usefulness to the healthcare system in their territory. Some HTA bodies also take the financial cost of medicines into account, in accordance with national legislation.
The Agency recognises that some new medicines that receive marketing authorisation fail to be reimbursed or used as expected. Cooperation between medicines regulators and HTA bodies is significant for patients, since it has the potential to increase access to these medicines and reduce the development costs for sponsors, by enabling the medicine development programme to be aimed at generating data relevant for both regulatory and HTA needs.
Interaction with European HTA bodies
Since 2010, the Agency has been working closely with the European Network for Health Technology Assessment (EUnetHTA), a network of government-appointed organisations from European Union Member States, the European Economic Area and accession countries and a large number of relevant regional agencies and non-for-profit organisations that produce or contribute to HTA in Europe.
The initial focus of the collaboration was a project looking into how the information on the benefits and risks of a medicine contained in European public assessment reports (EPAR) could better address the needs of HTA bodies. This started following recommendations by European policymakers to improve the way data published by regulators contribute to relative-effectiveness assessments by HTA bodies.
The project has resulted in a series of improvements to the EPAR template. The Agency and EUnetHTA are in the process of publishing the outcome of this project in more detail.
In addition, dialogue between the Agency and EUnetHTA has led to other areas of interaction. They are working together on topics including:
- giving advice on development plans for medicines from both regulators and HTA bodies;
- providing mutual input on methodological and disease-specific guidelines, evidence requirements and publication of data relevant for orphan-designated medicines;
- collaboration on study registries.
The Agency and EUnetHTA also agreed the development of a joint three-year work programme in May 2013 outlining key areas of collaboration.
Full details of the interaction between the Agency and EUnetHTA are available in the meeting minutes:
- Minutes of the European Medicines Agency and EUnetHTA meeting - May 2014
- Minutes of the European Medicines Agency and EUnetHTA meeting - December 2013
- Minutes of the European Medicines Agency and EUnetHTA meeting - May 2013
- Minutes of the European Medicines Agency and EUnetHTA meeting - November 2012
- Minutes of the European Medicines Agency and EUnetHTA meeting - February 2012
- Minutes of the European Medicines Agency and EUnetHTA meeting - March 2011
- Minutes of the European Medicines Agency and EUnetHTA meeting - June 2010
- Minutes of the European Medicines Agency and EUnetHTA meeting - February 2010
Joint scientific advice with HTA bodies
The Agency offers scientific advice and protocol assistance in parallel with HTA bodies. The aim of this is to allow medicine developers to gain feedback from from regulators and HTA bodies at the same time, early in the development of a medicine. This helps them to establish the evidence that both parties will need to determine a medicine's benefit-risk balance and value.
A pilot for parallel scientific advice was launched in July 2010. Around 25 procedures had been finalised or were ongoing as of November 2013, covering indications such as diabetes, heart failure, lung cancer, breast cancer, pancreatic cancer, melanoma, mesothelioma, asthma, rheumatoid arthritis, multiresistant infections, food allergies, diabetic gastroparesis, Alzheimer’s disease, depression, osteoporosis and three rare conditions.
The following healthcare-guidance and HTA organisations took part in different procedures in the pilot in various ways:
- Agenzia Italiana del Farmaco;
- College voor Zorgverzekeringen;
- Gemeinsamer Bundesausschuss;
- Haute Autorité de Santé;
- L'Agència d'Avaluació i Qualitat Sanitàries de Catalunya;
- National Institute for Health and Care Excellence;
- Tandvårds- och Läkemedelsförmånsverket;
- Vertragspartner Medikamente.
A joint EMA-HTA workshop on parallel scientific advice was held on 26 November 2013. Following the workshop, and based on the experience gained by all stakeholders, draft best practice guidance for EMA-HTA parallel scientific advice was developed and published for public consultation in May 2014:
- Draft best practice guidance for pilot European Medicines Agency health technology assessment parallel scientific advice procedures
The guidance details the timelines and actions whereby applicants can seek simultaneous feedback from regulators and HTA bodies on their development plans. The final guidance will take into account feedback from all stakeholders.
For more information, see scientific advice and protocol assistance.
- European Network for Health Technology Assessment (EUnetHTA)
- Roadmap to 2015
- Scientific advice and protocol assistance
- Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers
- Understanding the role and evidence expectations of health technology assessment and coverage/payer bodies. What are they looking for, and how and why does this differ from what regulators require?
- Regulators, payers, and prescribers: can we fill the gaps?
- EMA releases best practice guidance on parallel scientific advice with health technology assessment bodies (08/05/2014)
- European Medicines Agency / health-technology-assessment-body workshop on parallel scientific advice in drug development (26/11/2013)
- Early dialogue between regulators and health technology assessment bodies key to medicines development (28/11/2013)
- Draft best practice guidance for pilot European Medicines Agency health technology assessment parallel scientific advice procedures (08/05/2014)
- Report from the European Medicines Agency / health-technology-assessment-body workshop on parallel scientific advice in drug development on 26 November 2013 (08/05/2014)
- European Medicines Agency and EUnetHTA three-year work plan (19/11/2013)
- Call for expression of interest to the attention of healthcare product (pharmaceuticals or medical devices) developers (06/12/2013)