Evidence map›Paper›PMID 28749065›Full record

ReviewStem cells translational medicine2017

Concise Review: The High Cost of High Tech Medicine: Planning Ahead for Market Access.

Dawn Driscoll, Stephanie Farnia, Panos Kefalas, Richard T Maziarz

Abstract readReview
In one paragraph

Review in Stem cells translational medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.

0numbers the graph read from it
0cells of the map it votes in
25citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

25 citing papers in PubMed.

  1. Review
  2. Review
  3. Review
  4. Article
  5. Review
  6. Article
  7. Article
  8. Review
  9. Barriers to accessing cellular therapy for patients receiving care in community practices.Hematology. American Society of Hematology. Education Program · 2023
    Article
  10. Article
  11. Article
  12. Article
  13. Value-based pricing for advanced therapy medicinal products: emerging affordability solutions.The European journal of health economics : HEPAC : health economics in prevention and care · 2022
    Article
  14. Longevity leap: mind the healthspan gap.NPJ Regenerative medicine · 2021
    Review
  15. Regenerative medicine clinical readiness.Regenerative medicine · 2021
    Review
  16. Review
  17. Review
  18. Review
  19. Review
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Dawn DriscollDCi Biotech Inc. and DCi Biotech Pty Ltd., Perth, Australia.
Stephanie FarniaAmerican Society for Blood and Marrow Transplantation, Arlington Heights, Illinois, USA.
Panos KefalasCell and Gene Therapy Catapult Limited, London, United Kingdom.
Richard T MaziarzKnight Cancer Institute, Oregon Health and Science University, Portland, Oregon, USA.ORCID 0000-0002-8184-7099

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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. Stem Cells Translational Medicine 2017;6:1723-1729.

Indexed as

Cost-Benefit AnalysisEuropeHematopoietic Stem Cell TransplantationHumansTechnology, High-CostUnited StatesAcute myelogenous leukemiaAutologous stem cell transplantationCellular therapyHematopoietic stem cell transplantation

Identifiers

PMID28749065
PMCPMC5689744

What OpenQuestion holds

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LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.