Understanding the Role and Evidence Expectations of Health Technology Assessment and Coverage/Payer Bodies: What Are They Looking for, and How and Why Does This Differ From What Regulators Require?

Pharmaceutical companies have a good understanding of the needs and requirements of regulatory bodies, but the evidence expectations of health technology assessment (HTA) and coverage/payer bodies are less well understood and addressed. This paper seeks to improve this understanding by providing an overview of the expectations of HTA and coverage/payer bodies, explaining how and why these differ from those of regulators, and describing the extent and limitations of work on harmonization. The article goes on to describe ways in which HTA and coverage/payer bodies' expectations can be addressed, and to encourage industry to interact with HTA and coverage/payer bodies to increase mutual understanding and hence promote more efficient development of and access to innovative medicines.

At the center of health care policy making: the use of health technology assessment at NICE.

The UK's National Institute for Health and Clinical Excellence (NICE), a world leader in health technology assessment (HTA), sits at the interface of a policy environment where everything is urgent and consensus between policy makers and stakeholders is sometimes difficult to attain. The majority of stakeholder challenges to NICE's use of HTA concern the interpretation of evidence and the methodological rules applied by the appraisal committees. We discuss the most significant issues: choice of comparators; evidence synthesis and indirect comparison; parameter selection, especially for the valuation of quality of life; extrapolation beyond clinical trial data; and the level of the cost-effectiveness threshold.

The National Institute for Health and Clinical Excellence and its role in assessing the value of new cancer treatments in England and Wales.

The boundaries of medical science in the treatment of cancer are constantly extending. Developments of existing treatments, innovative approaches, new discoveries, and more targeted therapeutic options are translating into practice. With advances come increasing costs, often of a magnitude that stretches finite financial resources. When decisions about funding are made on behalf of a population, standardized processes and methods are needed in order to produce robust guidance in a fair, consistent, and transparent way. The challenges of making these difficult decisions are brought into particularly stark relief when potentially life-extending treatments for patients with a short life expectancy are appraised. The National Institute for Health and Clinical Excellence (NICE) produces guidance on the clinical- and cost-effectiveness of medicines compared with current standard practice. Approximately 40% of the technologies appraised by NICE are indicated for cancer, and the majority of these are pharmaceuticals, mostly biological agents. This article provides an overview of the current role of NICE in making new technologies for cancer available in England and Wales. This includes a summary of experiences with end-of-life treatments and the supplementary advice regarding such treatments that was issued by NICE to its decision-making committees in 2009.

10 years of NICE: still growing and still controversial.

The National Institute for Health and Clinical Excellence (NICE) will have existed for 10 years on April 1, 2009. Over the past decade, the institute's methodological approach to the development of guidance and assessment of the value of health-care interventions has received international interest and acclaim. Furthermore, individual decisions, in particular those made on new cancer drugs, have generated enormous controversy. An early example was the appraisal of irinotecan and oxaliplatin for colorectal cancer in 2002. In 2003, NICE described the rationale behind its decision making. The 10th anniversary of the institute provides an opportunity to review some of the key issues affecting cancer appraisals and to explain the development of other NICE guidance programmes relevant to the provision of cancer services.

Cardiac resynchronisation therapy for the treatment of heart failure: NICE technology appraisal guidance.

This NICE technology appraisal guidance on cardiac resynchronisation therapy provides additional treatment options for some of the groups of people covered in the earlier guidance on implantable cardioverter defibrillators.

Improving patient access to novel medical technologies in Europe.

The European Society of Cardiology (ESC) organized a one-day workshop with clinicians, health economic experts, and health technology appraisal experts to discuss the equity of patient access to novel medical technologies in Europe. Two index technologies were considered: implantable cardioverter defibrillators (ICDs) and drug-eluting stents (DES). The use of ICDs range from 35 implants/million population in Portugal to 166 implants/million population in Germany, whereas for implants of DES (as percentage of total stents) it is lowest in Germany at 14% and high in Portugal at 65%. These differences can in part be explained by a lack of structured implementation of guidelines, the direct cost in relation to the overall healthcare budget, and to differences in procedures and models applied by Health Technology Assessment (HTA) agencies in Europe. The workshop participants concluded that physicians need to be involved in a more structured way in HTA and need to become better acquainted with its methods and terminology. Clinical guidelines should be systematically translated, explained, disseminated, updated, and adopted by cardiologists in Europe. Clinically appropriate, consistent and transparent health economic models need to be developed and high-quality international outcome and cost data should be used. A process for funding of a technology should be developed after a positive recommendation from HTA agencies. Both the ESC and the national cardiac societies should build-up health economic expertise and engage more actively in discussions with stakeholders involved in the provision of healthcare.

The cancer technology appraisal programme of the UK's National Institute for Clinical Excellence.

The National Institute for Clinical Excellence (NICE) was established to ensure faster and more equitable uptake of new technologies by the NHS, through the provision of guidance on clinical and cost-effectiveness. The treatment of cancer is one of the UK government's priority areas and a range of guidance products have been developed by NICE to support implementation of national plans for managing patients with cancer in England and Wales. In its first 3 years, NICE's main activity was the "Technology Appraisals Programme" and it has created considerable interest and some controversy. 15 (out of a total of 56) technology appraisals related to oncology have been completed and another four are in preparation. The open, transparent, and inclusive approach NICE has adopted in reaching its decisions highlights the difficult ethical issues that need to be addressed in seeking to balance individual desires with public-health requirements. In this review we describe the process of appraising technologies, and address the recent criticism of the appraisal programme with regard to treatment of patients with cancer.