Expert insight:

Outlook for HTA cooperation in Europe


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By Neil Grubert, market access specialist, analyst and author.
 

Health technology assessment—a focus of increasing attention for the EU

Health technology assessment (HTA) is an area of growing interest to the EU. As long ago as 2004, the European Commission (EC) and Council of the European Union stated that “HTA has become a political priority and there is an urgent need for establishing a sustainable European network on HTA.”1

In 2006, the EU member states, Norway and Switzerland created the European network for Health Technology Assessment (EUnetHTA), an alliance of 80 national and regional HTA authorities and other healthcare agencies. EUnetHTA's declared mission is “to support the collaboration between European HTA organisations that brings added value to healthcare systems at the European, national and regional level.”2 To date, EUnetHTA has focused largely on encouraging early dialogue between drug manufacturers and HTA bodies, promoting parallel scientific advice from the European Medicines Agency (EMA) and HTA agencies, developing an HTA core model, and carrying out pilot projects on rapid relative effectiveness assessments (REAs).

In 2011, the European Parliament and Council of the European Union established the HTA Network to provide strategic direction on cross-border HTA collaboration in Europe. The network comprises national ministries of health. Participation is voluntary, but all 28 EU member states have signed up for membership. In addition, Iceland and Norway are observer members.
 

EUnetHTA—a cautious first step towards collaboration

EUnetHTA has overseen three sequential Joint Action (JA) programmes. JA1 had a budget of €6 million and ran from 2010 to 2012; it focused on scientific/technical cooperation on methodologies and tools. JA2 had a budget of €9.5 million and ran from 2012 to 2015; the focus was on continued development of cooperation and piloting joint assessments. The budget for JA3, scheduled to run from 2016 to 2020, has been increased to €20 million; the programme aims to conduct 80 joint assessment reports and 35 early dialogues, improve the uptake of joint work at national/regional level, and develop/revise guidelines, models, methodologies and other HTA tools.

To date, however, the practical impact of joint working on HTA has been limited. The EC sees four main shortcomings to EU cooperation on HTA:

  • The uptake of joint work at national/regional level has been modest, leading to considerable duplication of effort by HTA bodies and manufacturers.
  • Administrative capacity and procedural frameworks vary substantially among the more than 50 national and regional HTA bodies active across the EU. Member states also differ in the status they grant to the output from HTA agencies (e.g., recommendations vs legally binding judgements).
  • National HTA methodologies also vary significantly—data requirements for dossiers (Table 1), designated comparators and the way of expressing added therapeutic value (e.g., a scale versus a simple “yes/no” assessment) are just some of the variables. As a consequence of these variations, the outcomes of evaluations are often very different from one country to another.
  • The current model for cross-border HTA cooperation is not financially sustainable in the long term.

Table 1: Data requested/accepted by selected European HTA agencies and EUnetHTA for antidiabetic drugs
eu_hta_table1.png
Source: Adapted from Inception Impact Assessment: Strengthening of the EU cooperation on Health Technology Assessment (HTA).3
 

For its part, the life sciences industry has given a mixed response to EU HTA collaboration to date. Pharmaceutical companies have generally derived benefit from both multi-HTA early dialogues and parallel scientific advice. They have particularly appreciated the opportunity to explore issues such as patient population, choice of comparator, clinical trial design, endpoints, statistical analysis (e.g., patient stratification) and health economic data. However, drug manufacturers note that regulators are essentially interested in a medicine in isolation (i.e., its safety, efficacy, and quality), whereas HTA agencies have to consider a drug’s overall impact on their healthcare systems. Furthermore, pharmaceutical companies are frustrated at the limited reuse of joint work by national/regional HTA agencies, a situation that can actually increase the expense and workload for manufacturers. One study estimates the average cost of each national HTA review at €30,000 for agencies and €100,000 for manufacturers (not including the cost of collecting additional data to meet varying national data requirements).4
 

Strengthening EU cooperation on health technology assessment

Mindful of the shortcomings of HTA collaboration to date, in September 2016, the EC unveiled a new initiative known as “Strengthening of the EU cooperation on Health Technology Assessment (HTA).” This project has three general objectives:

  • Enable member states to strengthen their cooperation on HTA in a sustainable manner.
  • Improve the functioning of the internal market for health technologies.
  • Contribute to a high level of human health protection.

In addition, the EC has set four more-specific objectives for the new HTA initiative:

  • Reduce duplication of effort for both HTA agencies and manufacturers.
  • Promote convergence in HTA procedures and methodologies.
  • Improve the uptake of joint work in member states.
  • Ensure the long-term sustainability of EU HTA cooperation.

In pursuit of these objectives, the EC has proposed five options for the future evolution of HTA cooperation in the EU. The proposals range from maintaining the status quo, in which case HTA cooperation would come to an end in 2020, to cooperation on the production of joint full HTA reports, with compulsory uptake of joint outputs by member states. Table 2 summarises the key features of each option.
 

Table 2: Options for future evolution of EU HTA cooperation
eu_hta_table2.png
Source: Adapted from Inception Impact Assessment: Strengthening of the EU cooperation on Health Technology Assessment (HTA).3
 

Public consultation on the HTA initiative

To ascertain the views of interested parties on the proposed HTA initiative, the EC conducted an online public consultation from 21st October 2016 to 31st January 2017. The consultation received 249 responses—63 (25%) from individuals/citizens and 186 (75%) from public administrations, economic stakeholders, associations or organisations, including 97 (39%) from industry or service providers.

The consultation found that a clear majority of administration representatives, patients/consumers and healthcare providers considered EU cooperation on HTA to be useful, and none of the respondents from these groups felt that such collaboration was not useful. By comparison, the pharmaceutical and medical technology industries were notably less enthusiastic: only 4% of respondents from big pharmaceutical companies and 5% from medical technology companies thought HTA cooperation had been useful, although clear majorities felt that working together had been somewhat useful. Interestingly, 28% of respondents from medical technology companies and 22% of representatives of small and medium-sized companies held the view that HTA cooperation was not useful (Table 3).

Interestingly, 28% of respondents from medical technology companies and 22% of representatives of small and medium-sized companies held the view that HTA cooperation was not useful (Table 3).
 

Table 3: Stakeholders’ views on the usefulness of EU cooperation on health technology assessment
eu_hta_table3.png
Source: Adapted from Strengthening of the EU Cooperation on Health Technology Assessment (HTA): Online Public Consultation Report.5
 

The consultation also asked respondents to comment on the degree to which they thought certain types of joint HTA activities would meet their needs. Overall, pharmaceutical industry participants were very positive about most activities. Almost all of the respondents from big pharmaceutical companies who answered this question indicated that joint tools/templates/databases, guidelines, early dialogues and joint relative effectiveness assessment would meet their needs very much. However, they felt very differently about joint full HTA: almost all believed such collaboration would not meet their needs (Table 4). This response reflects the industry’s conviction that non-clinical HTA domains—economic, ethical, organisational, legal and social factors—remain a national preserve, and therefore an unsuitable subject for cross-border collaboration.
 

Table 4: Pharmaceutical industry views on the degree to which selected joint HTA activities would meet their needs
eu_hta_table4_0.png
Source: Adapted from Strengthening of the EU Cooperation on Health Technology Assessment (HTA): Online Public Consultation Report.5
 

Rapid progress on joint scientific advice and commitment to improve processes and member states’ uptake of joint assessments

On 13th June 2017, the EUnetHTA Directorate, supported by the EC Directorate General for Health and Food Safety (DG SANTE), held a meeting of high-level HTA stakeholders, including senior representatives of HTA agencies and leading drug manufacturers. Participants agreed to form the EUnetHTA Early Dialogues Working Party (EDWP) to work with the EMA to establish a single European joint scientific advice process involving regulatory and HTA authorities. They also agreed on the need for greater adoption of joint assessments at the national level, particularly by author countries. A follow-up meeting is expected in 2018. A statement issued by EUnetHTA6 identified three key objectives for future interactions:

  • “Discuss adaptations to processes within national HTA agencies and within companies that are needed to ensure uptake of European reports.
  • Ensure the lessons from ongoing EUnetHTA assessments feed into improved European processes moving forward, in particular in light of a post 2020 scenario and a possible Commission initiative.
  • Broaden the discussion to all relevant stakeholders to ensure success of European cooperation post-2020.”

EUnetHTA and the EMA wasted no time in acting on the decision to establish a single European joint scientific advice process. On 4th July 2017, they announced the creation of the EDWP as a single gateway for requests for parallel consultations with the EMA and HTA agencies; the first requests are due to be processed in September 2017. Consultations will be possible not only for initial evidence generation, but also for post-authorisation data collection. Patient representatives will routinely be involved in parallel consultations.

In a press release issued by the EMA7, Guido Rasi, the agency’s Executive Director, noted that “enabling patients’ access to medicines is no longer a job for regulators alone. Today, we need to work with all decision-makers in healthcare to make sure that medicines that can make a real difference to people’s lives can actually reach them. Our work with EUnetHTA aims to align our respective requirements as much as possible so that developers can generate one set of data that allows the assessment of both the benefits and risks of a medicine and its added value.” Wim Goettsch, the Director of JA3, predicted that EUnetHTA’s “strengthened involvement in early dialogues and our collaboration with EMA will support the generation of evidence that better meets the needs of HTA bodies and will support informed decisions at national level."
 

Proposed new HTA regulation

On 31st January 2018, the EC published a proposal for a new regulation on HTA8. The proposed system is based on option 4 in the Inception Impact Assessment: Strengthening of the EU cooperation on Health Technology Assessment (HTA)3 (see Table 3). EU cooperation on HTA would be built on four main pillars:

  1. Joint clinical assessments, primarily of health technologies that are deemed to be the most innovative and to have the greatest potential to add value to the healthcare systems of EU member states.
  2. Joint scientific consultations that allow manufacturers to seek early guidance on the evidence that will be required for HTA submissions.
  3. Horizon scanning to identify the most promising health technologies for patients and health systems early and ensure that these technologies are included in HTA collaboration.
  4. Voluntary cooperation in areas that are not subject to mandatory cooperation, such as economic evaluation.

Joint clinical assessments of medicines will be limited to drugs that undergo the EU's central marketing authorisation procedure, including both new active substances and established products for which the manufacturer is seeking a label extension.

The EC's objective is to increase the predictability of HTA procedures across the EU and to avoid duplication and inconsistencies. To that end, member states would not generally be permitted to repeat joint clinical assessments, though they would be free to conduct non-clinical HTA assessments (e.g., of economic, ethical, organisational, legal and social factors). In exceptional circumstances, however, a safeguard clause would allow member states to revert to performing their own clinical assessments, subject to prior authorisation by the EC. In all cases, member states would retain their current freedom to draw their own conclusions regarding the value of health technologies and to make pricing and reimbursement decisions.

The proposed new rules would take effect three years after they entered into force, that is, 20 days after they were adopted by both the European Parliament and the Council. Thereafter, an additional  three-year transitional period would allow member states time to adjust to the new system. The EC envisages that the number of joint clinical assessments performed would increase from 10-15 in the first year of implementation to approximately 65 by the end of the transitional period. Once the transitional period had ended, all drugs and the medical devices included in the scope of the proposal would be subject to joint clinical assessment.

The life sciences industry has broadly welcomed the proposals on HTA cooperation. In a press release on the subject, for example, the European Federation of Pharmaceutical Industries and Associations (EFPIA), expressed optimism that “joint clinical assessments will facilitate faster access, avoid duplication at national level and deliver greater consistency, clarity and predictability for everyone involved in the process. Joint scientific consultations or ‘early dialogue’ will help to ensure that medicine development programmes meet the data and evidence requirements of both future regulatory and clinical benefit assessments. The inclusion of horizon scanning will facilitate better planning and management of the introduction of new health technologies that have a significant impact on patients, on healthcare systems and on society.”9

It remains to be seen if the European Parliament and Council will adopt the EC’s proposal. Assuming they do, manufacturers will want assurances that future change will be widely implemented and will genuinely help to reduce costs and avoid duplication of effort. It should not be forgotten that drugs that are approved by methods other than the centralised marketing authorisation procedure will not be included in the joint HTA system. Moreover, even if member states do make use of joint clinical assessments for centrally authorised medicines, they will still be free to make additional HTA demands on manufacturers for these products, such as requirements for health economic evaluations. Furthermore, pharmaceutical companies will ultimately remain at the mercy of national or regional authorities that determine the value of health technologies and set pricing and reimbursement terms. HTA cooperation may save all stakeholders some time, money and effort, but it will not of itself lead to more uniform market access across the EU.

Last update: March 2018

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1 High Level Group on Health Services and Medical Care. Accessed at www.goo.gl/X7hvdB.

2 EUnetHTA Mission, Vision and Values. Accessed at www.eunethta.eu/about-us/mission-vision-values.

3 Inception Impact Assessment: Strengthening of the EU cooperation on Health Technology Assessment (HTA). Accessed at www.ec.europa.eu/smart-regulation/roadmaps/docs.

4 European Cooperation on Health Technology Assessment, Ecorys, January 2013. Accessed at ec.europa.eu/health

5 Strengthening of the EU Cooperation on Health Technology Assessment (HTA): Online Public Consultation Report. Accessed at ec.europa.eu/health.

6 Meeting with Heads of Agencies/High-Level Representatives and Heads of European Pharmaceutical Companies. Accessed at www.eunethta.eu/news/meeting-heads-agencieshigh-level-representatives-and-heads-european-pharmaceutical-companies.

7 EMA and EUnetHTA step up interaction to align data requirements. Accessed at www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events

8 Proposal for a Regulation of the European Parliament of the Council on health technology assessment and amending Directive 2011/24/EU. Accessed at ec.europa.eu/health.

9 EFPIA views Commission’s proposals on Health Technology Assessment (HTA) as positive step. Accessed at https://www.efpia.eu/news-events/the-efpia-view/