HTA challenges for appraising rare disease interventions viewed through the lens of an institutional multidimensional value framework

ABSTRACT

Introduction

Evaluating rare disease interventions poses challenges for HTA agencies, including uncertainties and ethical issues and tensions. INESSS has recently adopted a Statement of Principles and Ethical Foundations which proposes a multidimensional approach to value appraisal as well as five principles to frame the evaluation process.

Areas covered

Our aim was to identify and analyze HTA challenges for appraising interventions for rare diseases, using the Statement’s approach to value appraisal as an analytical framework, and outline how the Statement’s principles can help address these challenges. Challenges, covering a diversity of aspects, were identified by leveraging institutional experience in diverse domains of expertise and consolidated through narrative literature review. Challenges were categorized by value dimension (clinical, populational, economic, organizational, and sociocultural), which allowed to pinpoint how each challenge affects the ability to appraise the value of an intervention. Key ethical tensions across dimensions were also identified. Specific approaches to addressing these challenges – related to knowledge mobilization and integration, deliberation, and recommendation-making – were outlined on the basis of the principles promulgated in the Statement.

Expert opinion

A multidimensional approach can be fruitful for analyzing challenges for appraising the value of rare disease interventions and help guide approaches to tackle them.

KEYWORDS:

• Challenge
• HTA
• multidimensional
• rare diseases
• value appraisal
• value framework

Article highlights

• INESSS has adopted a Statement of Principles and Ethical Foundations that seeks to ascertain in how far an intervention creates value in the clinical, populational, economic, organisational, and sociocultural dimensions, thus enabling an overall value appraisal.

• In this work, a variety of challenges were identified and classified by ethics-derived goals (value dimension) to pinpoint in what way each affects the ability to appraise the value of interventions for rare diseases.

• We also outlined how the principles promulgated in the Statement can help address these challenges.

• This includes a rigorous and comprehensive knowledge mobilization and integration process that gathers all pertinent data by value dimension, highlights uncertainties and synthesizes the data in a clear and transparent manner to support deliberation.

• This also includes a deliberative process designed to develop fair, reasonable and value-adding recommendations by being organized around the value dimensions, which allows making ethical dilemmas explicit, and being oriented towards the common good.

Acknowledgments

The authors thank their colleagues, Geneviève Bigras, Anne-Marie Lemieux, Sylvie Bouchard and Virginie Landreville at the INESSS’ Direction de l’évaluation des médicaments et des technologies à des fins de remboursement, for their critical reading and commenting of the manuscript.

Declaration of interest

The authors are employed by INESSS. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

One peer reviewer declares: employee of PRECISIONheor, an HEOR consultancy that provides services to life sciences companies; holds equity in the parent company Precision Medicine Group. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Additional information

Funding

The work was self-funded by INESSS.