A beginner’s guide to better understand curative and transformative therapies

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 current state of curative and transformative therapies. The special themed section appears in the June 2019 issue of Value in Health. Guest editors for the themed section are Don Husereau, BSc Pharm, MSc, University of Ottawa, Ottawa, ON, Canada, and Shelby D. Reed, RPh, PhD, Duke University, Durham, NC, USA.

The themed section commences with an editorial in which Husereau and Reed provide historical perspective and introduce the 8 themed articles in the series:

  1. Estimating the Clinical Pipeline of Cell and Gene Therapies and Their Potential Economic Impact on the US Healthcare System, by Casey Quinn, et al
  2. Are Global Health Systems Ready for Transformative Therapies?, by Eric Faulkner, et al
  3. Are Payers Ready, Willing, and Able to Provide Access to New Durable Gene Therapies?, by Jane F. Barlow, Mo Yang, and J. Russell Teagarden
  4. Defining and Managing High-Priced Cures: Healthcare Payers' Opinions, by Kai Yeung, et al
  5. New Cost-Effectiveness Methods to Determine Value-Based Prices for Potential Cures: What Are the Options?, by Steven D. Pearson, Daniel A. Ollendorf, and Richard H. Chapman
  6. Analytic Considerations in Applying a General Economic Evaluation Reference Case to Gene Therapy, by Michael F. Drummond, et al
  7. How Does Treating Chronic Hepatitis C Affect Individuals in Need of Organ Transplants in the United Kingdom?, by Anupam Bapu Jena, et al
  8. Uncertainty and Cures: Discontinuation, Irreversibility, and Outcomes-Based Payments: What Is Different About a One-Off Treatment?, by Adrian Towse and Elisabeth Fenwick

In their editorial, Husereau and Reed note several crosscutting themes that will need to be addressed by those in health technology assessment and outcomes research, including payers' concerns about duration of effect, perceptions of unaffordability, social values, and the desirability and feasibility of performance-based risk-sharing agreements.

"At the advent of this new era, we are all beginners," write the editors. "It is our hope that these articles provide ISPOR members and others with an up-to-date understanding of what we are about to face: what the potential impact of curative therapies might be, whether HTA bodies and payers are ready for them, what aspects of evaluation may need to be modified or expanded, and how we might pay for them."

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