Accountable and Reasonable Processes for Coverage Decision-Making for Rare Disease and Regenerative Therapies – Which Criteria Should be Considered and Why?

Abstract

reduced by at least $22 million under the new law. Reducing the corporate tax rate from 35% to 21% reduces the value of all tax credits, unless a business sustains a loss. Reducing the Orphan Drug Credit by half reduces the tax shelter provided by this specific credit, and reduces the incentive of companies to invest in orphan drugs. The corporate tax rate reduction should also result in an increased investment from companies, but this investment will be in all aspects of the company to maximize profit. It is likely that research investments would be made in other disease areas, which have larger markets and greater potential profits[1]-a large disease like diabetes has nearly 17 times the expected profits of a typical orphan disease. Conclusion: A profitable company may reduce its investments into orphan drug research to maximize profits due to the reduction in the orphan tax credit, despite the apparent advantages orphan drugs have, as orphan drug markets are much smaller. [1]Bio.org.

Priority-setting for hospital funding of high-cost innovative drugs and therapeutics: A qualitative institutional case study

“…A clear decision-making process with explicit principles and evaluation criteria can help manage the complexity of contentious funding decisions [4][5][6][7]. For example, health technology assessment (HTA) frameworks are increasingly used in the context of resource allocation for various health interventions, including D&Ts, within publicly funded health systems [1,3,[8][9][10]. These frameworks are employed in value-based assessments of health technologies-often premised on principles of clinical effectiveness, economic efficiency, and patient and/or societal values-to inform the allocation of limited public sector resources [3].…”

Institutional Priority-Setting for Novel Drugs and Therapeutics: A Qualitative Systematic Review

“…Established processes for health technology assessment (HTA), incorporating a range of frameworks and tools, are increasingly integrated into public health system approaches to resource allocation for health technologies, including drugs and therapeutics. 4,[7][8][9] For decision-makers at all levels of the healthcare system, a robust decision-making process with articulated principles, goals and criteria can help manage the complexity of funding decisions surrounding high-cost, innovative therapies. [10][11][12][13] While no formal, standardized threshold exists to distinguish between high-and low-cost drugs, definitions of highcost therapeutics have included those which are expensive, generate excessive costs, and represent a disproportionate cost relative to the total cost of the agent in terms of volume and duration.…”