Concise review: The high cost of high tech medicine: Planning ahead for market access

Dawn Driscoll, Stephanie Farnia, Panos Kefalas, Richard Maziarz

Research output: Contribution to journalArticle

14 Scopus citations

Abstract

Cellular therapies and other regenerative medicines are emerging as potentially transformative additions to modern medicine, but likely at a staggering financial cost. Public health care systems’ budgets are already strained by growing and aging populations, and many private insurer’s budgets are equally stretched. The current systems that most payers employ to manage their cash flow are not structured to absorb a sudden onslaught of very expensive prescriptions for a large portion of their covered population. Despite this, developers of new regenerative medicines tend to focus on the demands of regulators, not payers, in order to be compliant throughout the clinical trials phases, and to develop a product that ultimately will be approvable. It is not advisable to assume that an approved product will automatically become a reimbursed product, as examples from current practice in hematopoietic stem cell transplantation in the U.S. demonstrate; similarly, in Europe numerous Advanced-therapy Medicinal Products achieved market authorization but failed to secure reimbursement (e.g., Glybera, Provenge, ChondroCelect, MACI). There are however strategies and approaches that developers can employ throughout clinical development, in order to generate clinical and health economic data which will be necessary to demonstrate the value proposition of the new product and help ensure market access for patients; furthermore, performance based managed entry agreements coupled with post-launch evidence generation can help overcome challenges around product uncertainty at launch and reduce market access delays.

Original languageEnglish (US)
Pages (from-to)1723-1729
Number of pages7
JournalStem cells translational medicine
Volume6
Issue number8
DOIs
StatePublished - Aug 1 2017

Keywords

  • Acute myelogenous leukemia
  • Autologous stem cell transplantation
  • Cellular therapy
  • Hematopoietic stem cell transplantation

ASJC Scopus subject areas

  • Developmental Biology
  • Cell Biology

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