A pilot study to identify areas for further improvements in patient and public involvement in health technology assessments for medicines.

BACKGROUND: Patient and public participation in health technology assessment (HTA) of medicines has been cited as an important component of the decision-making structure; however, how to actually achieve meaningful involvement is less understood. OBJECTIVES: Our objectives were to conduct a pilot study to form the basis of future research and to gain insight into how to practically and meaningfully advance patient and public input in HTAs for medicines. METHODS: Semi-structured interviews (n = 13) with informants in Australia (n = 7), Canada (n = 3), and the UK (n = 3) were conducted across agencies and experts (n = 9), as well as patient and advocacy groups (n = 4). RESULTS: This pilot study identified through structured interviews three areas for further consideration. Advancement area 1 indicates that industry could help bring the patient perspective into the HTA process through incorporating patient experiences early in the drug development process and by including qualitative research on patient experiences in HTA dossiers. Advancement area 2 involves recognizing and supporting the role of patient advocacy groups, and making use of their access to the genuine patient perspective and experience of living with the condition in question. Finally, advancement area 3 is the continuous development of HTA systems and processes to better facilitate involvement, increasing transparency and feedback, exploring new options for reaching patients, and focusing on creating an active and informed health consumer. CONCLUSIONS: The HTA process is becoming increasingly transparent to patients and the public; however, more effort is required to fully engage patients in the decision-making processes for medicine HTAs. This pilot study identified three key areas for further advancement in this field, and recognized a need for further research in the areas of measuring the impact of patient engagement on decision making in medicine HTAs, as well as the best methods to better prepare patient advocacy groups through HTA education and training. These research recommendations will form the basis of a future study with a larger, more comprehensive sample.

Coverage with Evidence Development: applications and issues.

OBJECTIVES: The aim of this study was to describe the current issues surrounding Coverage with Evidence Development (CED). CED is characterized by restricted coverage for a new technology in parallel with targeted research when the stated goal of the research or data collection is to provide definitive evidence for the clinical or cost-effectiveness impact of the new technology. METHODS: Presented here is information summarized and interpreted from presentations and discussions at the 2008 Health Technology Assessment International (HTAi) meeting and additional information from the medical literature. This study describes the differences between CED and other conditional coverage agreements, provides a brief history of CED, describes real-world examples of CED, describes the areas of consensus between the stakeholders, discusses the areas for future negotiation between stakeholders, and proposes criteria to assist stakeholders in determining when CED could be appropriate. RESULTS: Payers could interpret the evidence obtained from a CED program either positively or negatively, and a range of possible changes to the reimbursement status of the new technology may result. Striking an appropriate balance between the demands for prompt access to new technology and acknowledging that some degree of uncertainty will always exist is a critical challenge to the uptake of this innovative form of conditional coverage. CONCLUSIONS: When used selectively for innovative procedures, pharmaceuticals, or devices in the appropriate disease areas, CED may provide patients access to promising medicines or technologies while data to minimize uncertainty are collected.

Estimating the costs and benefits of new drug therapies: atypical antipsychotic drugs for schizophrenia.

This article presents an analytic tool populated with data from published studies to illustrate the likely impacts of a switch from typical to atypical antipsychotic drugs over a 3-year period on individual, family, and societal outcomes for a U.S. schizophrenia population. Data taken from clinical trials and observational data studies are used. Changes in annual health care costs, schizophrenia symptom days, extrapyramidal symptom (EPS) days, suicide rates, and employment days are estimated. For each 1,000 people treated with typical drugs, the base case scenario gives estimates of annual medical care costs of dollar 28.9 million with 80,253 moderate/severe schizophrenia symptom days and 92,006 EPS days. In the base case scenario, after switching to atypical drugs, schizophrenia symptom days are estimated to decrease up to 33 percent, EPS days up to 50 percent, and total medical care costs up to 19 percent over the 3-year period. Suicide rates fall and employment rates increase. The direction of the impacts remain the same for a wide range of input parameter values used in sensitivity analyses. Thus, switching to atypical drugs will likely reduce total medical care costs and decrease other disease burdens for people with schizophrenia, their families, and society.