Health Technology Assessment and Appraisal of Therapies for Rare Diseases

Abstract

Innovative rare disease therapies and health technology assessment (HTA) share a lot of similarities. Both represent cases of interaction of epidemiology and health economics. Both are relatively new topics in public health practice. And both pose a lot of challenges to rare disease stakeholders who are currently looking for tools to support the timely access to innovative treatments while putting budget spending in order. This is why optimisation of assessment and appraisal of new rare disease therapies is a fundamental issue in rare disease health policy. Rare disease patients and caregivers expect prolonged life expectancy and improved quality of life and they perceive innovative health technologies as a rightful way to achieve these objectives.Multi-criteria decision analysis (MCDA) provides a structured, transparent approach to identify preferred alternatives by means of combined calculation of relative importance of different criteria and performance of the alternatives on these criteria. The labyrinth of competing interests and numerous stakeholders involved is why innovative rare disease health technologies make an excellent case study of the integration between HTA and MCDA. This kind of formalisation of decision-making is perceived as fair and legitimate, leading to a balance and agreement. MCDA provides a stage for a debate of policy priorities, health system specifics and societal attitudes, while also addressing the impact of rarity on all criteria and considerations.

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

“…First of all, it is argued that one universally true and “constant” ICER threshold does not exist, and, in fact, is constantly changing. Moreover, most countries do not apply an explicit ICER threshold ( Iskrov et al, 2017 ; Eichler et al, 2004 ). Moreover, as we have mentioned before, it is difficult for OMPs (especially those indicated for ultra-rare diseases) to adhere to standard cost-effectiveness thresholds ( Schuller et al, 2015 ; Drummond et al, 2007 ).…”

Are Global Health Systems Ready for Transformative Therapies?

“…10 Although HTA agencies are beginning to acknowledge differences in technology types (eg, the Diagnostics Assessment Programme of the National Institute for Health and Care Excellence [NICE]), most have not developed explicit channels for addressing transformative or curative effect per se. [11][12][13] Even recent efforts to evolve next-generation value frameworks have not included explicit emphasis on transformative or curative effect. 14 Although it is clear that transformative medicine is not marginal, studies addressing transformative effect indicate that health stakeholders find it difficult to assign a single definition or threshold of transformative effect for creating health policies.…”

“…Technology-driven trends poised to influence the global health ecosystem. 6,8,13,24,40, Trend Examples…”

“…This has long been a challenge for new medical devices. 13,[173][174][175] The rapid expansion of rare disease therapies has also stimulated much debate and shift of focus toward solutions to manage aggregate cost impacts. [176][177][178] Similarly, cellular therapies involving multiple steps and technologies face similar challenges, as do smart and e-connective technologies not readily accommodated by today's systems.…”

“…Key factors that influence how new health technologies are addressed by health system decision makers [4][5][6][7][8][9][10][11][12][13][14][15][16][17]. …”