New insights regarding challenges in rare disease diagnosis, drug approval, and patient access

Value in Health, the official journal of ISPOR (the professional society for health economics and outcomes research), announced today the publication of a series of articles offering important insight regarding the challenges in rare disease diagnosis, drug approval, and patient access. The special themed section appears in the May 2018 issue of Value in Health. Kati Copley-Merriman, MS, MBA, RTI Health Solutions, Ann Arbor, Michigan, USA is the section guest editor.

The themed section commences with an editorial in which Ms Copley-Merriman briefly outlines core rare disease challenges and introduces the 9 articles in the series:

  1. Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group, by Sandra Nestler-Parr, et al
  2. The Problem of Rarity: Estimation of Prevalence in Rare Disease, by Stéphane Auvin, et al
  3. Clinical Outcome Assessments: Use of Normative Data in a Pediatric Rare Disease, by Dawn Phillips and Beth Leiro
  4. Economic Modelling Considerations for Rare Diseases, by Isobel Pearson, et al
  5. Budgetary Impact and Cost Drivers of Drugs for Rare and Ultra-Rare Diseases, by Michael Schlander, Charalabos-Markos Dintsios, and Afschin Gandjour
  6. Can Severity Outweigh Smaller Numbers? A Deliberative Perspective from Canada, by Monica Magalhaes
  7. Societal Preferences for Funding Orphan Drugs in the United Kingdom: an Application of Person Trade Off and Discrete Choice Experiment Methods, by Siobhan Bourke, Catrin Plumpton, and Dyfrig Hughes
  8. Evaluating and Valuing Drugs for Rare Conditions: No Easy Answers, by Daniel Ollendorf, Richard Chapman, and Steven Pearson
  9. Patient Access to Medicines for Rare Diseases in European Countries, by Andreja Deticek, Igor Locatelli, and Mitja Kos

In her editorial, Ms Copley-Merriman noted the efforts of 2 working groups of the ISPOR Rare Disease Special Interest Group in developing a rare disease terms and definitions manuscript and broadly identifying challenges confronting those engaged in rare disease-focused research and development and technology assessment. As a follow-up to that work, Value in Health issued a special call for papers to attract submissions for this themed section dedicated to rare diseases.

"While the international community has taken steps to address the rare disease challenges outlined in these papers, it has done so with a high degree of variation," Copley-Merriman said. "The ISPOR Rare Disease Special Interest Group plans to continue its multistakeholder efforts towards providing recommendations to address rare disease challenges regarding evidence generation and health technology assessment."

As more rare disease diagnostics and treatments are developed, the challenges for demonstrating safety, efficacy, effectiveness and value, are becoming increasingly evident. The ISPOR Rare Disease Special Interest Group was formed to elucidate issues in the rare disease environment so that researchers, payers, patients, and life sciences companies can effectively address key challenges and more effectively establish the value of new and existing diagnostics and therapeutics.​

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