But does it work? Exploring the techno-scientific and socio-political dynamics of Health Technology Assessment and Pharmaceutical Regulation
Lead Research Organisation:
King's College London
Department Name: Social Science, Health and Medicine
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.
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.
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.
Organisations
- King's College London (Fellow, Lead Research Organisation)
- European Consumers Association (Collaboration)
- The Cochrane Collaboration (Collaboration)
- University of Copenhagen (Collaboration)
- University of Sydney (Collaboration)
- London School of Hygiene and Tropical Medicine (LSHTM) (Collaboration)
- Prescrire International (Collaboration)
- Yale University (Collaboration)
- European Public Health Association (EUPHA) (Collaboration)
- UNIVERSITY OF BRITISH COLUMBIA (Collaboration)
- Health Action International (Collaboration)
People |
ORCID iD |
| Courtney Davis (Principal Investigator / Fellow) |
Publications
Davis C
(2016)
"Adaptive pathways" to drug authorisation: adapting to industry?
in BMJ (Clinical research ed.)
| Description | SPECIALIST TRAINING AND RESEARCH UNDERTAKEN Specialist training in the methods of health economic evaluation undertaken at the Centre for Health Economics in the University of York, and through participation in two short courses run by ISPOR, has enabled me to significantly extend my understanding of and expertise in methods of health technology assessment (HTA) and equipped me with the analytical tools necessary to undertake in-depth research and case study analyses of NICE appraisals of new drugs to treat type 2 diabetes (T2D) and evaluate wider policy initiatives at national and supranational levels. The research included extensive documentary data collection and analysis, including all publicly available documents for 14 NICE appraisals (3 Clinical Guidelines and 11 Technology Appraisals); interviews with 15 key experts and stakeholders; and participant observation of 11 separate meetings (2 NICE Appraisal Committee meetings; 4 forum meetings to discuss overall policy related to NICE; 2 NICE Annual Conferences; 1 EMA-HTA workshop; 1 European-level HTA workshop; and 1 ISPOR Conference). KEY FINDINGS A flood of new T2D drugs onto the market in recent years means that physicians and healthcare systems must choose between multiple treatments that can be used in varying combinations with older drugs. NICE undertakes technology appraisals (TAs) to evaluate the cost-effectiveness of new diabetes drugs relative to existing treatments with the overarching goal of improving health outcomes across the NHS by producing guidance that encourages an efficient use of NHS resources. Findings from the current project suggest that this goal has been undermined with respect to NICE TAs and clinical guidance for T2D treatments. First, intermingled forms of institutional and professional bias and clinician pressure have resulted in successive clinical guidelines recommending use of drugs that were not found to be cost-effective in health technology assessments. Second, critical uncertainties in the evidence base for new drugs - particularly relating to surrogate measures of drug benefit, lack of long-term safety data and lack of data for distinct patient groups - persist. This suggests that worldwide adoption of HTA processes has not incentivized pharmaceutical companies to generate more robust pre-marketing evidence for T2D drugs. Continued acceptance of surrogate measures of drug benefit has also resulted in health economic modelling that may both over- and under-estimate the long-term clinical benefits of individual T2D drugs in specific patient populations. Whilst there have been tremendous methodological advances in the quality and sophistication of cost-effectiveness assessment, complex methods cannot always compensate for 'evidence deficits', and in fact the greater the uncertainties and complexities of treatment, the greater the number of assumptions that must be made in model construction. And despite the development of increasingly sophisticated methods to characterise uncertainty, in practice companies have adopted a narrow approach to exploring the impact of alternative assumptions in TAs for new diabetes drugs. Methodological and model complexity, combined with political pressure to speed up the NICE TA process, may have had the undesirable consequence of undermining transparency and the capacity for independent scrutiny of industry submissions. This is important because overly optimistic assumptions about treatment effects and QALY values, and an unbalanced and selective choice of model inputs can systematically bias cost-effectiveness models and lead NICE to adopt new medicines that may not be cost-effective. In the face of immensely complex health economic evaluations, Committee members participating in NICE technology appraisals and guideline development are increasingly dependent on the expertise of a small handful of health economists sitting on the Committee and on reports undertaken by the Technology Assessment Report (TAR) groups, which are independent academic groups commissioned by NICE to produce either full health technology assessments (HTAs) or to review the HTAs produced by industry. However, introduction of the Single Technology Appraisal process in 2006, which aimed to accelerate NICE decision-making by requiring a single HTA produced by the company, further compromises Committee members' ability to independently assess company submissions. In the face of time pressures and the absence of a full and independent HTA, it has become difficult for Committee members and TAR groups to identify which assumptions are driving model outcomes, to explore alternative scenarios or generate alternative cost-effectiveness estimates. This problem is exacerbated by the 'black box' nature of some commercial economic models. Finally, increasing industry responsibility for and control of the production of HTAs has led to some asymmetry in the development of health economic modelling for T2D drugs. For example, the quality-of-life impacts of hypoglycaemia and weight gain/loss became a key focus in NICE appraisals only after companies sought to use these treatment effects to identify some incremental advantage for their drug over comparators in cost-effectiveness modelling. Conversely, negative findings from trial data (eg with respect to quality of life impacts) may be ignored, some suspected or know safety problems are not included in the health economic modelling, and inadequate attention has been paid in industry submissions (and hence NICE appraisals) to emerging clinical trial evidence that undermines some of the structural assumptions of existing models. NEW COLLABORATIONS Findings from this research project are being taken forward and will feed into a series of early stage collaborations, including further research involving colleagues from the LSE and UCL, and initiatives by health consumer and patient advocates, and EU and North American academics and practitioners to engage with pharmaceutical policy-makers at both national and international levels. |
| Exploitation Route | It has been estimated that updated NICE diabetes guidelines could lead to an increase of NHS spending from £841 per patient per year, to £4,312 per patient per year. This research suggests there is a risk this may not represent a cost-effective use of NHS resources, and this has significant implications for wider policy and the conduct of technology appraisals undertaken by NICE. Policy makers should reconsider their support for various proposals within the UK and the wider EU to implement accelerated and adaptive pathways for drug development and marketing since such proposals will further weaken the evidence base for new drugs at market launch. There is also a need for broader democratic debate concerning the potential societal costs and benefits of these strategies, including who should pay for further evidence generation. This research suggests that with respect to the adoption of new T2D drugs by the NHS, public health would be better served if regulators, HTA and reimbursement bodies required clearer characterisation of safety and robust evidence of treatment impacts on clinical outcomes that are meaningful to patients prior to drug marketing and reimbursement. In the meantime, NICE appraisals should more openly address emerging clinical evidence and ongoing scientific controversies, and include more robust exploration of the uncertainties to which these give rise. Health economists and other stakeholders need to consider how emerging data relating to the benefits and harms of treatment can be incorporated into existing health economic models, and how both clinical evidence and economic modelling might better capture the variable cost-effectiveness of specific treatment strategies in different patient populations. Policy makers should strengthen independent scrutiny of company submissions by allowing TAR groups to undertake full HTAs, even in the context of single technology appraisals. Understanding of the impact of uncertainty on health technology assessment and funding decisions would be enhanced by further research to explore whether the issues arising in relation to NICE technology appraisals of T2D drugs also impact on other disease areas, and to investigate whether and how uncertainties have been addressed by other national HTA bodies. |
| Sectors | Healthcare,Pharmaceuticals and Medical Biotechnology |
| Description | The evidence base for new drugs is largely shaped by the scientific and technical standards established by national and supranational medicines regulatory agencies, such as the European Medicines Agency (EMA). When new drugs are approved on the basis of limited or preliminary data this in turn challenges the capacity of health technology assessment and reimbursement bodies to determine the value of a new drug to patients and healthcare systems. Findings from the current project suggest that this is also true of current regulatory standards for new drugs to treat type 2 diabetes (T2D), and that in the absence of clinical trial data on new drugs' impact on mortality and morbidity compared to existing treatments, there is a risk that health economic modelling may both over- and underestimate the long-term relative efficacy and harms of individual T2D drugs in specific patients populations. The recent market appearance of multiple new drugs to treat T2D and the prevalence of the condition in the UK population have significant budget - and related opportunity cost - implications for the NHS. It has been estimated that updated NICE diabetes guidelines could lead to an increase of NHS spending from £841 to £4,312 per patient per year. In the absence of clinical trial evidence evaluating patient relevant outcomes, there is significant uncertainty that this represents a cost-effective use of limited NHS resources. Between March 2014 and August 2016, the EMA ran a pilot project to assess a new initiative called 'adaptive pathways, which will allow new drugs to be launched on the market faster on the basis of fewer data, with uncontrolled observational studies increasingly relied on to provide evidence of drug benefits and harms. Clearly this initiative may have a further negative impact on the capacity of HTA bodies to evaluate the cost-effectiveness for publicly-funded heath care systems of new drugs. Dissemination of findings from the research project at conferences and workshops has impacted the way in which civil society organisations have responded to the EMA's proposals, and resulted in collaborations between the grant holder, other academics and practitioners working in the fields of HTA, epidemiology and public health, and third sector groups. In collaboration with Health Action International (HAI), a non-governmental organisation dedicated to strengthening medicines policy to improve health, selected findings from the project were presented to a conference organised by the French Haute Autorite de Sante and the European Commission in Paris, October 2015, to discuss recent initiatives at the EU level to provide opportunities for greater formal collaboration and consultation between HTA bodies and the EMA. Selected findings from the project were also disseminated at a closed workshop in Brussels in April 2016. The workshop was attended by scientists, regulators, patient and consumer health advocates, medical journalists, not-for-profit drug development partnerships and health insurers, and findings from the project have informed the responses of a number of civil society organisations to the EMA's proposals - including HAI, the European Public Health Association (EPHA), the Medicines In Europe Forum, and BEUC (the European Consumer Organisation). Evidence presented at the workshop also provided the basis for a BMJ analysis article, co-authored with other academics from the workshop and on which I was lead author. A further outcome of the workshop was that a letter of concern was sent to the European Medicines Agency and the European Commission, co-signed by a number of prominent scientists. |
| First Year Of Impact | 2015 |
| Sector | Communities and Social Services/Policy,Healthcare,Pharmaceuticals and Medical Biotechnology |
| Impact Types | Societal,Policy & public services |
| Description | Citation in European Medicine's Agency report on its Adaptive Pathways Workshop |
| Geographic Reach | Europe |
| Policy Influence Type | Citation in other policy documents |
| Impact | Our analysis, and expression of the need for the European Medicines Agency (EMA) to consult with a wider range of stakeholders with respect to it proposals to implement a new 'adaptive' approach to medicines authorisation, contributed the the EMA and EU Commission's decision to host a workshop in November 2016 to increase transparency and engagement with stakeholders. |
| 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 |
| Impact | Our analyses of some of the potential consequences for public health of current trends in pharmaceutical regulation within the supranational EU has influenced the policy demands of coalition of five patient groups and non-government organisations, with citation of our work in a document submitted to the 17th International Conference of Drug Regulatory Authorities (ICDRA), Cape Town, South Africa in November 2016. |
| URL | http://www.tac.org.za/news/civil-society-appeal-medicines-regulators-and-government-occasion-17th-in... |
| 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 |
| 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 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 |
| 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 | 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. |
| 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 | 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 |
| 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 | 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 |
| 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 | 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 |