But does it work? Exploring the techno-scientific and socio-political dynamics of Health Technology Assessment and Pharmaceutical Regulation

Lead Research Organisation: University of Sussex
Department Name: Sch of Law, Politics and Sociology

Abstract

Appropriate use of medicines can make an important contribution to public health. Yet pharmaceutical drugs are expensive and costs are increasing at a time when national health care spending is constrained. Consequently, national governments and health care authorities must decide how best to allocate limited resources to achieve the greatest health benefits for society. This includes decisions on whether new drugs should be reimbursed. To inform this decision-making process, national governments are increasingly likely to establish organisations to undertake different forms of health technology assessment (HTA). The purpose of HTA bodies is to review and evaluate the available evidence, and to advise policy-makers on the clinical and cost-effectiveness of new medicines compared to available therapies. In the UK this function is undertaken by the National Institute for Health and Clinical Excellence (NICE).

Innovative pharmaceutical products can enter the market in the UK and other member states of the European Union only after they have been assessed by government scientists in national regulatory agencies or by the centralised European Medicines Agency (EMA). These organisations determine whether the drugs are safe and efficacious based on techno-scientific evidence, including data from clinical trials. The regulatory requirements of these agencies significantly determine the nature and quality of the evidence base for new drugs available at the time of a drug's launch onto the market. HTA bodies like NICE will then assess this evidence to determine whether new drugs offer sufficient therapeutic value relative to other treatments to justify reimbursement (that is, whether they are cost effective). Increasingly, stakeholders have claimed that the scientific evidence base for new drugs at time of market launch fails to provide robust data on clinical effectiveness and is consequently inadequate for HTA purposes. This in turn may lead to flawed decision-making, increasing the risk of patients being treated with sub-optimal drugs, scarce resources being misdirected, and missed opportunities for maximising health gains offered by other treatments.

The proposed research aims to address this major emerging challenge for pharmaceutical and public health policy and to inform current policy debates on these issues by providing a social scientific investigation of how techno-scientific regulatory standards governing the development and marketing authorisation of new drugs assist and/or limit the ability of HTA organisations to evaluate the clinical and cost-effectiveness of such drugs and their contribution to public health. Through empirical investigations of NICE technology appraisals (TAs) of drugs to treat obesity and type II diabetes the research aims: 1) to determine the nature and extent of evidence gaps and uncertainty in TAs for these drugs; (2) to investigate the ways in which HTA technical assessors and other interested stakeholders respond to scientific uncertainties, and to explore the socio-political dynamics of those responses; and (3) to map and assess the prospects of recent initiatives within the UK and EU to align HTA, drug development and regulatory assessment with patient and public health needs.

The research programme will be geographically located in the UK and the supranational EU, and will draw on documentary and interview research methods. In addition, training undertaken during the award in the methodologies of health economics, clinical epidemiology and health policy decision-making - and in particular training in specialist quantitative techniques - will enable the researcher to understand and engage effectively with the highly technical aspects of health technology appraisals.

Planned Impact

In a context of limited national budgets for health care expenditure, the question of how to optimise spending on pharmaceutical products for the benefit of public health is high on the policy agenda. The proposed research seeks to investigate whether, and to what extent, Health Technology Assessment (HTA) bodies are able to meet this goal given current weaknesses and gaps in the evidence base for new drugs. In addition, the research engages directly with the current policy context by investigating the extent to which various initiatives and proposals meet the needs of different stakeholder groups. Consequently, the research is highly relevant and timely, and has the potential to inform policy at a time when several private and public sector initiatives to increase collaboration between HTA bodies and the supranational European drug licensing agency are being pursued. The research will be of interest to and directly benefit the following groups of stakeholders:

UK Government and Public Sector bodies.
Findings from the proposed research of potential obstacles to robust HTA appraisal of new drugs have the potential to inform current policy debate about the future role of NICE, HTA and value-based pricing in the UK. It will therefore benefit policy-makers in the Department of Health, the NHS's National Institute for Health Research, the National Institute for Health and Clinical Excellence, and GPs as future commissioners of health care.

Supranational Institutions of the EU.
Research on the relationship between market authorisation and licensing, and on emerging EU-level policy initiatives is highly relevant to current policy debates and will benefit the EU Commission, which is actively supporting the development of HTA across Member States, and in particular the Directorate Generals for Health & Consumers and Enterprise & Industry which, through the Pharmaceutical Forum, have addressed reimbursement and relative effectiveness as priority issues. The proposed research is directly relevant to the question of what evidence is needed for HTA bodies to determine the relative effectiveness of new drug products and will therefore be of benefit to these EU bodies. The research will also be of interest to the European Medicines Agency (EMA) which has become involved in collaborative pilot projects with HTA bodies to provide joint advice to pharmaceutical companies developing new products in select therapeutic areas.

Private Sector.
The pharmaceutical industry is concerned that the spread of national and regional HTA bodies within Member States of the EU presents a "fourth hurdle" to market access and increases the evidential requirements (and thus the development costs) for new drugs. Some companies increasingly favour standardisation of the evidence required by HTA bodies and the EMA. The research is directly pertinent to this issue and will be of interest to industry generally, and in particular to The European Healthcare Innovative Leadership Network (EHILN) - an industry initiative to bring together representatives of pharmaceutical companies, patient groups, HTA bodies and drug licensing authorities to consider how companies can demonstrate the value of their drugs.

Third Sector Organisations.
Finally, the research will be of interest to patient groups within the UK and EU, who have expressed concerns about access to medicines and the quality of evidence to inform treatment decisions, and to consumer health organisations with an interest in the rational use of medicines such as Health Action International, Prescrire International and the Medicines in Europe Forum. The research will also be of interest to professional associations such as HTA International, EUnetHTA and the European Public Health Association and to health advisory/ research bodies and networks such as the James Lind Alliance and WHO Europe's European Observatory on Health Systems and Services.

Publications

10 25 50
publication icon
Davis C (2016) "Adaptive pathways" to drug authorisation: adapting to industry? in BMJ (Clinical research ed.)

Related Projects

Project Reference Relationship Related To Start End Award Value
ES/J006610/1 30/09/2012 31/12/2012 £187,217
ES/J006610/2 Transfer ES/J006610/1 01/01/2013 30/10/2015 £161,875
 
Description Citation in European Medicine's Agency report on its Adaptive Pathways Workshop
Geographic Reach Europe 
Policy Influence Type Citation in other policy documents
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URL http://www.ema.europa.eu/docs/en_GB/document_library/Report/2017/02/WC500222153.pdf
 
Description Citation in stakeholder policy submission to the 17th International Conference of Drug Regulatory Authorities
Geographic Reach Multiple continents/international 
Policy Influence Type Citation in other policy documents
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Description Letter to the Executive Director of the European Medicines Agency
Geographic Reach Europe 
Policy Influence Type Implementation circular/rapid advice/letter to e.g. Ministry of Health
Impact A letter of concern was sent to the Executive Director of the European Medicines Agency (EMA) regarding the EMA's proposals to implement a new adaptive apporoach to drug licensing. This letter arose from a workshop at which the principle investigator presented her research findings, and was based on discussions that had taken place during the workshop, including discussions of the PI's work.
URL https://epha.org/scientists-voice-concerns-about-adaptive-pathways/
 
Description Health Action International funding
Amount € 10,000 (EUR)
Organisation Health Action International 
Sector Charity/Non Profit
Country Netherlands
Start 01/2017 
End 03/2017
 
Description Canadian Institute of Health Research-National Health and Medical Research Council of Australia international comparative project on the impact of regulatory safety warnings 
Organisation University of British Columbia
Country Canada 
Sector Academic/University 
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Collaborator Contribution This study compares safety advisories on medicines in Canada, the United States, the European Union (United Kingdom and the Netherlands), and Australia from 2005 to 2016. The focus is on new safety concerns for which the decision to communicate, methods used, or key messages differ between countries. We will develop a database of advisories on serious safety concerns in the five countries from 2005 to 2016. At a population level, we examine whether recommended changes to prescribing, other health-related behaviours, and health outcomes have occurred to a greater extent in countries with safety advisories than in those without advisories, The study also includes policy analysis and interviews with clinicians and patient groups to better understand factors that enhance the effectiveness of safety advisories and how awareness and medicine use are affected. The aim of the study is to identify best practices in regulatory risk communication on medicines, in terms of effects on medicine prescribing and use, and ultimately on public health.
Impact Sociology Pharmacoepidemiology Health policy Health services Medicine
Start Year 2017
 
Description Canadian Institute of Health Research-National Health and Medical Research Council of Australia international comparative project on the impact of regulatory safety warnings 
Organisation University of Copenhagen
Country Denmark 
Sector Academic/University 
PI Contribution UK case study investigating how UK and EU medicines regulatory bodies have coordinated and governed the management and communication of drug safety risks for the SGLT2-inhibitor diabetes drugs
Collaborator Contribution This study compares safety advisories on medicines in Canada, the United States, the European Union (United Kingdom and the Netherlands), and Australia from 2005 to 2016. The focus is on new safety concerns for which the decision to communicate, methods used, or key messages differ between countries. We will develop a database of advisories on serious safety concerns in the five countries from 2005 to 2016. At a population level, we examine whether recommended changes to prescribing, other health-related behaviours, and health outcomes have occurred to a greater extent in countries with safety advisories than in those without advisories, The study also includes policy analysis and interviews with clinicians and patient groups to better understand factors that enhance the effectiveness of safety advisories and how awareness and medicine use are affected. The aim of the study is to identify best practices in regulatory risk communication on medicines, in terms of effects on medicine prescribing and use, and ultimately on public health.
Impact Sociology Pharmacoepidemiology Health policy Health services Medicine
Start Year 2017
 
Description Canadian Institute of Health Research-National Health and Medical Research Council of Australia international comparative project on the impact of regulatory safety warnings 
Organisation University of Sydney
Country Australia 
Sector Academic/University 
PI Contribution UK case study investigating how UK and EU medicines regulatory bodies have coordinated and governed the management and communication of drug safety risks for the SGLT2-inhibitor diabetes drugs
Collaborator Contribution This study compares safety advisories on medicines in Canada, the United States, the European Union (United Kingdom and the Netherlands), and Australia from 2005 to 2016. The focus is on new safety concerns for which the decision to communicate, methods used, or key messages differ between countries. We will develop a database of advisories on serious safety concerns in the five countries from 2005 to 2016. At a population level, we examine whether recommended changes to prescribing, other health-related behaviours, and health outcomes have occurred to a greater extent in countries with safety advisories than in those without advisories, The study also includes policy analysis and interviews with clinicians and patient groups to better understand factors that enhance the effectiveness of safety advisories and how awareness and medicine use are affected. The aim of the study is to identify best practices in regulatory risk communication on medicines, in terms of effects on medicine prescribing and use, and ultimately on public health.
Impact Sociology Pharmacoepidemiology Health policy Health services Medicine
Start Year 2017
 
Description Trans-Atlantic Network for Access to Safe and Effective Medicines 
Organisation European Consumers Association
Country Belgium 
Sector Charity/Non Profit 
PI Contribution I was involved in advising EPHA and first establishing this network within the EU, and then in developing the network into a Trans-Atlantic collaboration involving the Collaboration for Research Integrity and Transparency at Yale Law School and a number of US non-governmental organisations and scholars working on the topic of medicines R&D, regulation and access. I have participated in and spoken at all related workshops and conferences.
Collaborator Contribution All partners have participated in the related workshops and conferences. The European Public Health Association (EPHA) hosted the first brainstorming and strategy meeting in Brussels, 18&19 April 2016 EPHA jointly hosted, with the Polish Permanent Representation to the EU, a conference in Brussels 29-30th November 2016 on 'Healthy Innovation for All' Yale University will be hosting an upcoming workshop on 22nd - 23rd June 2017, which aims to develop an international collaboration on issues of common interest related to current trends in pharmaceutical regulation, development and pricing
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Start Year 2016
 
Description Trans-Atlantic Network for Access to Safe and Effective Medicines 
Organisation European Public Health Association (EUPHA)
Country European Union (EU) 
Sector Charity/Non Profit 
PI Contribution I was involved in advising EPHA and first establishing this network within the EU, and then in developing the network into a Trans-Atlantic collaboration involving the Collaboration for Research Integrity and Transparency at Yale Law School and a number of US non-governmental organisations and scholars working on the topic of medicines R&D, regulation and access. I have participated in and spoken at all related workshops and conferences.
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Start Year 2016
 
Description Trans-Atlantic Network for Access to Safe and Effective Medicines 
Organisation Health Action International
Country Netherlands 
Sector Charity/Non Profit 
PI Contribution I was involved in advising EPHA and first establishing this network within the EU, and then in developing the network into a Trans-Atlantic collaboration involving the Collaboration for Research Integrity and Transparency at Yale Law School and a number of US non-governmental organisations and scholars working on the topic of medicines R&D, regulation and access. I have participated in and spoken at all related workshops and conferences.
Collaborator Contribution All partners have participated in the related workshops and conferences. The European Public Health Association (EPHA) hosted the first brainstorming and strategy meeting in Brussels, 18&19 April 2016 EPHA jointly hosted, with the Polish Permanent Representation to the EU, a conference in Brussels 29-30th November 2016 on 'Healthy Innovation for All' Yale University will be hosting an upcoming workshop on 22nd - 23rd June 2017, which aims to develop an international collaboration on issues of common interest related to current trends in pharmaceutical regulation, development and pricing
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Description Trans-Atlantic Network for Access to Safe and Effective Medicines 
Organisation London School of Hygiene and Tropical Medicine (LSHTM)
Country United Kingdom 
Sector Academic/University 
PI Contribution I was involved in advising EPHA and first establishing this network within the EU, and then in developing the network into a Trans-Atlantic collaboration involving the Collaboration for Research Integrity and Transparency at Yale Law School and a number of US non-governmental organisations and scholars working on the topic of medicines R&D, regulation and access. I have participated in and spoken at all related workshops and conferences.
Collaborator Contribution All partners have participated in the related workshops and conferences. The European Public Health Association (EPHA) hosted the first brainstorming and strategy meeting in Brussels, 18&19 April 2016 EPHA jointly hosted, with the Polish Permanent Representation to the EU, a conference in Brussels 29-30th November 2016 on 'Healthy Innovation for All' Yale University will be hosting an upcoming workshop on 22nd - 23rd June 2017, which aims to develop an international collaboration on issues of common interest related to current trends in pharmaceutical regulation, development and pricing
Impact doi: https://doi.org/10.1136/bmj.i4437 - political sociology, epidemiology, medicine, public health
Start Year 2016
 
Description Trans-Atlantic Network for Access to Safe and Effective Medicines 
Organisation Prescrire International
Country France 
Sector Private 
PI Contribution I was involved in advising EPHA and first establishing this network within the EU, and then in developing the network into a Trans-Atlantic collaboration involving the Collaboration for Research Integrity and Transparency at Yale Law School and a number of US non-governmental organisations and scholars working on the topic of medicines R&D, regulation and access. I have participated in and spoken at all related workshops and conferences.
Collaborator Contribution All partners have participated in the related workshops and conferences. The European Public Health Association (EPHA) hosted the first brainstorming and strategy meeting in Brussels, 18&19 April 2016 EPHA jointly hosted, with the Polish Permanent Representation to the EU, a conference in Brussels 29-30th November 2016 on 'Healthy Innovation for All' Yale University will be hosting an upcoming workshop on 22nd - 23rd June 2017, which aims to develop an international collaboration on issues of common interest related to current trends in pharmaceutical regulation, development and pricing
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Start Year 2016
 
Description Trans-Atlantic Network for Access to Safe and Effective Medicines 
Organisation The Cochrane Collaboration
Country Global 
Sector Charity/Non Profit 
PI Contribution I was involved in advising EPHA and first establishing this network within the EU, and then in developing the network into a Trans-Atlantic collaboration involving the Collaboration for Research Integrity and Transparency at Yale Law School and a number of US non-governmental organisations and scholars working on the topic of medicines R&D, regulation and access. I have participated in and spoken at all related workshops and conferences.
Collaborator Contribution All partners have participated in the related workshops and conferences. The European Public Health Association (EPHA) hosted the first brainstorming and strategy meeting in Brussels, 18&19 April 2016 EPHA jointly hosted, with the Polish Permanent Representation to the EU, a conference in Brussels 29-30th November 2016 on 'Healthy Innovation for All' Yale University will be hosting an upcoming workshop on 22nd - 23rd June 2017, which aims to develop an international collaboration on issues of common interest related to current trends in pharmaceutical regulation, development and pricing
Impact doi: https://doi.org/10.1136/bmj.i4437 - political sociology, epidemiology, medicine, public health
Start Year 2016
 
Description Trans-Atlantic Network for Access to Safe and Effective Medicines 
Organisation Yale University
Country United States 
Sector Academic/University 
PI Contribution I was involved in advising EPHA and first establishing this network within the EU, and then in developing the network into a Trans-Atlantic collaboration involving the Collaboration for Research Integrity and Transparency at Yale Law School and a number of US non-governmental organisations and scholars working on the topic of medicines R&D, regulation and access. I have participated in and spoken at all related workshops and conferences.
Collaborator Contribution All partners have participated in the related workshops and conferences. The European Public Health Association (EPHA) hosted the first brainstorming and strategy meeting in Brussels, 18&19 April 2016 EPHA jointly hosted, with the Polish Permanent Representation to the EU, a conference in Brussels 29-30th November 2016 on 'Healthy Innovation for All' Yale University will be hosting an upcoming workshop on 22nd - 23rd June 2017, which aims to develop an international collaboration on issues of common interest related to current trends in pharmaceutical regulation, development and pricing
Impact doi: https://doi.org/10.1136/bmj.i4437 - political sociology, epidemiology, medicine, public health
Start Year 2016
 
Description Interview on BMJ Talk Medicine 16 August 2016 
Form Of Engagement Activity A broadcast e.g. TV/radio/film/podcast (other than news/press)
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact This was an interview with the British Medical Journal, for 'Talk Medicine', on the 16 August 2016, discussing an Analysis piece first authored by the PI, and published in the BMJ.
Year(s) Of Engagement Activity 2016
URL http://www.bmj.com/content/354/bmj.i4437
 
Description Participation in a European Medicines Agency-EU Commission workshop on Adapative Pathways Approaches to Medicines Aiuthorisation 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact This public workshop, which was jointly organised by the European Medicines Agency and the EU Commission, was held to gather the views and proposals of stakeholders on the adaptive pathways approach to medicines authorisation and potential implications for: public health, health technology assessment and access to medicines in Europe.
Year(s) Of Engagement Activity 2017
URL http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/events/2016/09/event_detail_001324...
 
Description Symposium on Access to Medicines In Europe 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Policymakers/politicians
Results and Impact Over 100 participants attended this two-day conference in Brussels on 29-30 November 2016, jointly organised by EPHA and the Polish Permanent Representation to the EU. Participants included, representatives from the EU Commission, industry, consumer and patient advocacy groups, experts in the field of pharmaceutical regulation and pricing and the media. The purpose of the symposium was to debate issues related to medicines development, regulation, pricing and equity of access in the European Union.
Year(s) Of Engagement Activity 2016
URL https://epha.org/healthy-innovation-for-all/
 
Description Symposium on Health Technology Assessment in the EU 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Policymakers/politicians
Results and Impact This was a symposium to discuss the future of health technology assessment in the EU, and in particular collaboration between the European Medicines Agency and national HTA bodies. It was attended by over 200 policy makers, regulators, health economists, consumer and patient groups, expert scientists and clinicians. The session in which I was involved discussed the merits of initiating and organizing an Early Dialogue between relevant regulatory bodies, HTA bodies and companies developing health products. Furthermore, the possible involvement of other relevant stakeholders in the process was also be addressed.
Year(s) Of Engagement Activity 2015
URL http://www.has-sante.fr/portail/jcms/c_2055829/en/forum-has-health-technology-assessment-sans-fronti...
 
Description Workshop and strategy meeting - The Push Towards Acclerated Market Approvals 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Third sector organisations
Results and Impact A closed brainstorming and strategy meeting with third sector organisations. As a consequence of this workshop, a letter signed by numerous experts in public health and evidence-based medicine,was sent to the Executive Director of the European Medicines Agency (EMA), expressing concern over, and the need for transparency in relation to EMA plans to implement a new approach to medicines authorisation called 'adaptive pathways'. This letter was published on the website of the EMA along with the EMA's response. partly as a consequence of this letter, a public workshop was held to discuss results of the EMA's Adaptive Pathways Pilot project.
Year(s) Of Engagement Activity 2016