A Systematic Review of the Effectiveness of Taxes in Preventing Obesity Trends

Abstract

A391review of Medline and EMBASE databases was conducted for the period 2000 -2013. The search sought to identify papers on the topic of assessment and reimbursement policy for rare diseases. Health policy studies, commentaries, and review articles were included. Clinical or economic studies of specific drugs or diseases were excluded. Information was extracted on assessment and reimbursement challenges and author recommendations for addressing these issues. Results: The literature review identified 726 papers; 49 met the inclusion criteria. The most frequently identified issues included multiplicity of orphan indications (34/49), high per-patient cost (32/49), and the difficulty in undertaking robust clinical and economic evaluations given limited evidence (32/49). Several authors commented on limitations of current health technology appraisal processes. The issue of equity and societal preference for funding rare diseases was highlighted in almost half of the papers (22/49). Lack of availability of alternative treatments was also considered an important factor. Suggestions for improvements to the assessment and reimbursement process included: greater use of registries (22/49), adjustment to preference weights used in cost effectiveness analysis (19/49) and conditional reimbursement and risksharing-schemes (12/49). Some authors advocated alternative pathways for assessing rare disease treatments including a specific approach utilising multi-criteria decision analysis. ConClusions: The debate on payer policy in rare diseases has grown in the last 5 years as concerns have increased about patient access to new medicines. While there is some consistency in the literature, there is as yet little consensus on how policy should be changed to address these issues.