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

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.

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.