Objectives: Novel specialty biopharmaceuticals hold promise for patients living with complex and chronic conditions. However, high research and development costs, special handling, and other necessary enhancements to patient support programs all contribute to frequently higher prices for these products. This study sought to assess the value of specialty pharmaceuticals through an examination of the clinical, functional, and economic benefits of these treatments for the top 3 disease areas by pharmaceutical spend: rheumatoid arthritis (RA), multiple sclerosis (MS), and breast cancer (BC).
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“How do you feel when someone says that it only worth paying £36,000 for one quality adjusted health year (QALY) for someone with MS? If a treatment costs more than that then it is not worth paying for it. The surprise in the study below is how poor the cost effectiveness of MS DMTs are ($248,000 per QALY) compared to those in rheumatoid arthritis ($38,900 per QALY) and breast cancer ($51,900 per QALY). The authors use a systematic review that only looks at published articles and misses unpublished data. This technique often misses negative data and results in publication bias; as a result of this the gap between MS DMTs and those in RA and breast cancer may actually be worse than this.”
“Is this study relevant? I think it is out of data and is dominated by first-generation DMTs. Now that we treat-2-target of NEDA and have more effective treatments these figures are bound to improve. This study, however, does raise the topic of cost-effectiveness and the issue of rationing. MSers in the UK are already used to be told you can’t have access to drugs x, y and z or procedure a, b and c because they have not been assessed by NICE, or they have been assessed by NICE and shown not to be cost-effective. When MSers make plans to access there therapies so that the taxpayer does not have to pay for them, people criticise the clinicians who prescribe these products privately. They imply private prescribing is entrenching inequality in the healthcare system and that these clinicians should be kicked out of the NHS. What these people forget is that an elected democratic government has enacted legislation to allow this to occur and are pushing for it to happen. The solution to austerity Britain is to subsidise the NHS with private prescribing. Is there a solution to this modern dilemma? Higher-taxes? Ring-fenced NHS taxation? Falling healthcare standards? I welcome suggestions.”
Zalesak et al. The value of specialty pharmaceuticals – a systematic review. Am J Manag Care. 2014 Jun;20(6):461-72.
Study Design: Systematic literature review.
Methods: A systematic review of market research and cost-effectiveness articles was conducted for each disease area to assess clinical, functional, and economic outcomes associated with specialty medicine treatments versus the previous standard of care.
Results: All RA clinical (American College of Rheumatology) and functional (Health Assessment Questionnaire) outcome articles were classified as positive. The median cost-effectiveness ratio was $38,900 per quality-adjusted life year (QALY). All MS clinical outcome (relapse rate) articles were positive. The MS functional outcome (Expanded Disability Status Scale) findings were less conclusive. The median cost-effectiveness ratio was $248,000 per QALY. The majority of BC articles yielded statistically inconclusive results for survival. All functional outcome (Quality of Life Questionnaire- Core 30) articles were positive. The median cost-effectiveness ratio was $51,900 per QALY.
Conclusions: Novel specialty therapies hold promise for arresting disease progression and improving quality of life for the 3 conditions associated with the highest specialty pharmaceutical spend. These findings demonstrate a strong value proposition for specialty pharmaceuticals, and suggest even greater potential individual patient benefit with consideration of patient heterogeneity.