Edition of 03 August 2020

The IMPACT HTA (Improved Methods and ACtionable Tools for enhancing HTA) Project was awarded a three-year grant by the European Commission in its Horizon 2020 program, ending in December 2020. It addresses several important policy-relevant gaps in the conduct of economic evaluations to inform HTA using international, interdisciplinary collaboration. One of the Work Packages (WP10) aims to develop guidance on novel approaches to appraising treatments for rare diseases to support robust, accountable decision-making across Europe on high-cost products that have a limited evidence base with high uncertainty and intense stakeholder scrutiny.

One of the main outputs of this stream of work, led by Bocconi University, is a detailed survey, started in 2018, of decision-makers across European member states, EEA countries, Canada, New Zealand and Australia, to identify which countries have different processes for appraisal or pricing and reimbursement of treatments for rare diseases. This survey has been augmented by desk research to identify additional guidance and develop detailed vignettes for each country that summarise their pricing and reimbursement processes. These vignettes have been validated by country experts. They are important resources for understanding the situation in each country, and for exchange of experiences and lessons learned.

In the context of a collaboration with Orphanet to improve the visibility of these resources, a link to the country vignettes now appear in the 'additional information' block of each disease, orphan drug and expert resource page. Orphanet will also, work to include a page to host these reports to ensure their longevity at the end of the IMPACT-HTA project. 

This work has also been presented in a recently published article in the Orphanet Journal of Rare Diseases entitled "Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches".The paper acknowledges that conventional appraisal approaches may not be suitable for assessing the value of treatments for rare diseases. Through an analysis of the country-level data collected, a number of special processes for the appraisal of rare disease therapies were identified that differ from standard processes.

The paper characterises the features identified within these special processes that allowed countries to better deal with some of the specificities of rare disease treatments in a more structured and consistent manner. These features enable different standards to demonstrate the value of rare disease treatments, support the interpretation of limited clinical evidence, help overcome the issues of lack of cost-effectiveness, and aim to accelerate the HTA process to guarantee more timely access to treatments for patients.

The authors express the need for transparency in national appraisal processes in the future as more therapies for rare diseases come to market, to ensure fair decision-making, and highlight that a common appraisal framework for rare disease therapies across Europe (and beyond) would be a positive evolution in this field.