Karen Facey

Rapid reviews versus full systematic reviews: An inventory of current methods and practice in health technology assessment

OBJECTIVES This review assessed current practice in the preparation of rapid reviews by health technology assessment (HTA) organizations, both internationally and in the Australian context, and evaluated the available peer-reviewed literature pertaining to the methodology used in the preparation of these reviews. METHODS A survey tool was developed and distributed to a total of fifty International Network of Agencies for Health Technology Assessment (INAHTA) members and other selected HTA organizations. Data on a broad range of themes related to the conduct of rapid reviews were collated, discussed narratively, and subjected to simple statistical analysis where appropriate. Systematic searches of the Cochrane Library, EMBASE, MEDLINE, and the Australian Medical Index were undertaken in March 2007 to identify literature pertaining to rapid review methodology. Comparative studies, guidelines, program evaluations, methods studies, commentaries, and surveys were considered for inclusion. RESULTS Twenty-three surveys were returned (46 percent), with eighteen agencies reporting on thirty-six rapid review products. Axiomatic trends were identified, but there was little cohesion between organizations regarding the contents, methods, and definition of a rapid review. The twelve studies identified by the systematic literature search did not specifically address the methodology underpinning rapid review; rather, many highlighted the complexity of the area. Authors suggested restricted research questions and truncated search strategies as methods to limit the time taken to complete a review. CONCLUSIONS Rather than developing a formalized methodology by which to conduct rapid reviews, agencies should work toward increasing the transparency of the methods used for each review. It is perhaps the appropriate use, not the appropriate methodology, of a rapid review that requires future consideration.

Rapid versus full systematic reviews: Validity in clinical practice?

Introduction:  Rapid reviews are being produced with greater frequency by health technology assessment (HTA) agencies in response to increased pressure from end‐user clinicians and policy‐makers for rapid, evidence‐based advice on health‐care technologies. This comparative study examines the differences in methodologies and essential conclusions between rapid and full reviews on the same topic, with the aim of determining the validity of rapid reviews in the clinical context and making recommendations for their future application.

Harmonization of evidence requirements for health technology assessment in reimbursement decision making

As more countries use HTA to inform decisions on the reimbursement of health technologies, harmonization of evidence requirements between jurisdictions has been proposed, mainly on the grounds of improved efficiency. Harmonization has the potential to avoid duplication of effort for both manufacturers and HTA bodies involved in preparing and reviewing HTA submissions for innovative technologies. However, it also carries risks of loss of local control over decisions, the application of general data standards which are not universally accepted and slowing the rate of development of innovation in the analytical disciplines supporting HTA. This study reviews the issues associated with harmonization taking into account the perspectives of the multiple stakeholders. This study draws on experiences from recent initiatives intended to promote the harmonization of HTA and experience from related fields, particularly regulatory approval of new medical technologies.

Virtual community consultation? Using the literature and weblogs to link community perspectives and health technology assessment

Background  Community views, expressed in social impact assessments and collected through community consultation, should play an important role in health technology assessment (HTA). Yet HTA methodologists have been slow to include outcomes of these forms of inquiry in analyses, in part because collecting community views is time‐consuming and resource intensive.

Patient-focused HTAs

HTA can be described as a research-based, practice-oriented assessment of relevant evidence and knowledge on the direct and intended effects of healthcare technologies, as well as the indirect and unintended consequences (4). It is also viewed as a multidisciplinary field of policy analysis that studies the medical, ethical, social, and economic implications of the development, diffusion, and use of a health technology (5).

IMPROVING THE EFFECTIVENESS AND EFFICIENCY OF EVIDENCE PRODUCTION FOR HEALTH TECHNOLOGY ASSESSMENT.

Objectives: Health Technology Assessment (HTA) needs to address the challenges posed by high cost, effective technologies, expedited regulatory approaches, and the opportunities provided by collaborative real-world evaluation of technologies. The Health Technology Assessment International (HTAi) Policy Forum met to consider these issues and the implications for evidence production to inform HTA. This paper shares their discussion to stimulate further debate. Methods: A background paper, presentations, group discussions, and stakeholder role play at the 2015 HTAi Policy Forum meeting informed this paper. Results: HTA has an important role to play in helping improve evidence production and ensuring that the health service is ready to adopt effective technologies. It needs to move from simply informing health system decisions to also working actively to align stakeholder expectations about realistic evidence requirements. Processes to support dialogue over the health technology life cycle need to be developed that are mindful of limited resources, operate across jurisdictions and learn from past processes. Collaborations between health technology developers and health systems in different countries should be encouraged to develop evidence that will inform decision making. New analytical techniques emerging for real-world data should be harnessed to support modeling for HTA. Conclusions: A paradigm shift (to “Health Innovation System 2.0”) is suggested where HTA adopts a more central, proactive role to support alignment within and amongst stakeholders over the whole life cycle of the technology. This could help ensure that evidence production is better aligned with patient and health system needs and so is more effective and efficient.

The Imperative for Patient-Centred Research to Develop Better Quality Services in Rare Diseases

The definition of a rare disease differs country by country, from an occurrence of\1 per 10,000 people in Taiwan and China to B5 per 10,000 people in Europe and Argentina. The issue everyone agrees with is that there are many, at least over 5,000 and perhaps even 6,000–8,000 different rare diseases. For many of these diseases, the prevalence is much lower than the defined threshold of rarity and they are genetic chronic diseases that impact daily life and quality of life, are life threatening and there is no approved disease-modifying or curative treatment. All of this means that patients can feel isolated and healthcare services often struggle to define and deliver good quality care, support and rehabilitation. Efforts have been made to improve this situation with special regulatory incentives and assistance for the development of treatments for rare diseases. The US Food and Drug Administration was the first to establish a special process for medicines for rare diseases and, in 2000, the European Medicines Agency established its orphan medicinal products process. However, despite this, in the decade from 2000, only 108 products for rare conditions were submitted to the European regulator and only 63 were authorised for treatment [1]. In 2009, the Council of the European Union recommended that Member States should take action on rare diseases and establish and implement strategies for rare diseases to ensure that patients with rare diseases have access to high-quality care including diagnostics, treatments and habilitation. The goal was to have these strategies in place by the end of 2013 and there has been a flurry of activity to see this achieved in Europe. Some countries have undertaken surveys to understand the issues faced by patients with rare diseases who are seeking to access services, and the following common themes arise: delay in diagnosis, lack of awareness of diseases in medical community, need to improve equity of access to services and effective treatments, fragmented non-specialist care and lack of research. In this special edition of The Patient, we seek to demonstrate that valuable informative research can be undertaken in rare diseases that can inform all these issues. A range of studies show how qualitative and humanistic research can be used to understand the most challenging aspects of living with a rare disease, patients’ unmet needs, patients attitudes to new treatments and how patients can influence the complex world of reimbursement. We have studies from researchers, clinicians and patients. They demonstrate how through collaboration they can learn from one another about research, living with an illness and decision making.

Putting Patients at the Centre of Healthcare: Progress and Challenges for Health Technology Assessments

Health technology assessments (HTAs) are meant to inform health policy by taking account of all the potential impacts of using a health technology. In the 1990s, HTAs included rigorous research to produce patient-based evidence, and some supported participation of patient representatives to help focus HTA research and determine value. In the 2000s, HTAs became more closely linked to reimbursement decisions, focusing on clinical and cost effectiveness. Patient involvement should be tailored to the specific needs of each HTA. As the timeframe for HTAs has reduced, research to produce patient-based evidence has been replaced by input from patient groups. This places a burden on individuals and organizations that needs to be critically reviewed. Therefore, it is imperative that we clarify when patient involvement is likely to add value and support patients to provide their unique knowledge in the most optimal way to influence HTA decision making. To reduce the burden on patient groups, more must be done to encourage research to produce patient-based evidence early in technology development. Like clinical research, a programme of research should be carefully planned, with appropriate methodological rigor for each study, and all research should be published. For this, the development of quality standards for research to produce patient-based evidence may be needed. Patient involvement has inherent value. It should be focused, systematic and transparent, and evolve according to the experiences of all stakeholders. All countries or collaboratives that undertake HTA should consider how they can elicit the needs, preferences and experiences of patients to support creation of patient-centered healthcare policy.