Nine years of comparative effectiveness research education and training: initiative supported by the PhRMA Foundation.

The term comparative effectiveness research (CER) took center stage with passage of the American Recovery and Reinvestment Act (2009). The companion US$1.1 billion in funding prompted the launch of initiatives to train the scientific workforce capable of conducting and using CER. Passage of the Patient Protection and Affordable Care Act (2010) focused these initiatives on patients, coining the term 'patient-centered outcomes research' (PCOR). Educational and training initiatives were soon launched. This report describes the initiative of the Pharmaceutical Research and Manufacturers Association of America (PhRMA) Foundation. Through provision of grant funding to six academic Centers of Excellence, to spearheading and sponsoring three national conferences, the PhRMA Foundation has made significant contributions to creation of the scientific workforce that conducts and uses CER/PCOR.

Proposed drug-drug cost effectiveness methodology.

The advent of prospective reimbursement in hospitals has forced hospital administrators, pharmacy directors, and pharmacy and therapeutic committees to carefully compare the cost and benefits of similar drugs. Accomplishing this task is difficult. This paper reviews the literature on drug-drug cost effectiveness procedures and outlines a methodology that might be used to contrast and compare two drugs with similar therapeutic outcomes. The data to conduct this analysis can be obtained from published articles on clinical studies and company sources and entered into a microcomputer electronic spreadsheet. The study discusses the implications and use of cost effectiveness analysis to evaluate drugs by hospital formulary committees.

Paying for health care in retirement: workers' knowledge of benefits and expenses.

Anecdotal evidence of retirees returning to the workforce to obtain health coverage has appeared against a backdrop of rising health insurance premiums and cutbacks in employer health benefit offerings to both current and future retirees. We present findings from a survey of workers ages forty-five to sixty-four concerning their attitudes toward and plans for health care coverage and expenses during retirement. We find a mismatch between workers' expectations about the benefits that are likely to be available to them and their planning as to how they will pay for health care in retirement.

The cost of new drug discovery and development.

Extract: The development of a new drug requires a major investment of capital, human resources, and technological expertise. It also requires strict adherence to regulations on testing and manufacturing standards before a new drug can be used in the general population. All these requirements contribute to the cost increases for a new chemical entities (NCE, i.e., new drug candidate) research and development (R&D). The central question raised by this trend is who will pay for new pharmaceutical R&D? With this question in mind, this article has three objectives: 1) to describe how the environment for pharmaceutical R&D has changed over time and the effect of these changes on the R&D process, 2) to summarize available information on the cost of drug discovery and development for NCEs, and 3) to consider the societal value of new drugs. The focus is on the United States, as the largest pharmaceutical market, and for which the relevant literature is most comprehensive, but many of the issues discussed are similarly important in the other major markets.

Regulatory issues for health-related quality of life--PhRMA Health Outcomes Committee workshop, 1999.

INTRODUCTION: Health-related quality-of-life (HRQL) can be defined as the impact of disease and treatment across the physical, psychological, social and somatic domains of functioning and well-being. Health-related quality-of-life measures are included in clinical trials of drug treatment to assess the impact of therapy on the patient's functioning. HRQL guidance could allow for use of this data in drug labeling and promotion. OBJECTIVES: The aim of our study was to provide recommendations with respect to regulatory issues important to the development of guidelines for HRQL research. METHODS: The HRQL workshop was planned jointly by members of the Pharmaceutical Research and Manufacturers of America Health Outcomes Committee and the Division of Drug Marketing, Advertising, and Communications of the Food and Drug Administration. The workshop was limited to six regulatory issues related to HRQL research in clinical trials of pharmaceutical therapies. These six issues were: instrument selection and validation, study design, data analysis, HRQL and safety, clinical meaning, and promotional use. Before the meeting, a consensus was reached that HRQL does not measure, nor should it be used to measure, safety. Therefore, five work groups discussed HRQL issues and made recommendations. RESULTS: Overall, the workshop recommended that HRQL measures be treated as any other clinical end point. The workshop recognized that research in HRQL methods is ongoing and that any guidance should be flexible to allow for changes in this developing research area. CONCLUSIONS: HRQL provides a patient perspective on the impact of disease and therapy on patients' daily life and functioning. Including HRQL information in promotion could be beneficial to decision making on the use of therapies. HRQL is a measure of effectiveness, not safety, and should be treated as any other clinical end point.