Evidence map›Paper›PMID 30229376›Full record

ArticleThe European journal of health economics : HEPAC : health economics in prevention and care2019

Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare.

Bengt Jönsson, Grace Hampson, Jonathan Michaels, Adrian Towse, J-Matthias Graf von der Schulenburg, Olivier Wong

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in The European journal of health economics : HEPAC : health economics in prevention and care, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 53 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
53citing papers in PubMed, 5 pooled it
31.1field-weighted citation impact, top 1% of its field
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

53 citing papers in PubMed, 5 syntheses or guidelines pooled it, 117 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Pooled it
  5. Pooled it
  6. Trial
  7. Article
  8. Review
  9. Review
  10. Article
  11. Article
  12. Article
  13. Article
  14. Article
  15. Article
  16. Article
  17. Review
  18. Article
  19. Article
  20. Review
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

6 authors at 4 institutions in 3 countries.

Bengt JönssonDepartment of Economics, Stockholm School of Economics, Stockholm, Sweden. Bengt.Jonsson@hhs.se.ORCID http://orcid.org/0000-0002-0051-7785
Grace HampsonOffice of Health Economics, London, UK.ORCID http://orcid.org/0000-0002-2019-1268
Jonathan MichaelsUniversity of Sheffield, Sheffield, UK.ORCID http://orcid.org/0000-0002-3422-7102
Adrian TowseOffice of Health Economics, London, UK.
J-Matthias Graf von der SchulenburgLeibniz University Hannover, Hannover, Germany.ORCID http://orcid.org/0000-0002-5322-9453
Olivier WongMedi-Qualite Omega, Paris, France.ORCID http://orcid.org/0000-0002-7344-7837
Office Of Health Economics · GBLeibniz University Hannover · DEStockholm School of Economics · SEUniversity of Sheffield · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundAdvanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them.

objectivesTo identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology-specifically economic evaluation methods-in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges.

methodsAn Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs.

resultsThree key topics were identified and prioritised for discussion-uncertainty, discounting, and health outcomes and value. The panel discussed that evidence challenges linked to increased uncertainty may be mitigated by collection of follow-on data, use of value of information analysis, and/or outcomes-based contracts. For discount rates, an international, multi-disciplinary forum should be established to consider the economic, social and ethical implications of the choice of rate. Finally, consideration of the feasibility of assessing the value of ATMPs beyond health gain may also be key for decision-making.

conclusionsATMPs face a challenge in demonstrating their value within current HTA frameworks. Consideration of current HTA principles and practices with regards to the specific characteristics of ATMPs and continued dialogue will be key to ensuring appropriate market access. CLASSIFICATION CODE: I.

Indexed as

Advisory CommitteesBiological TherapyCell- and Tissue-Based TherapyCost-Benefit AnalysisDecision MakingDelivery of Health CareEuropeGenetic TherapyHumansRegenerative MedicineTechnology Assessment, BiomedicalTreatment OutcomeAdvanced therapy medicinal productsCell therapyGene therapyHealth technology assessmentRegenerative medicineValue

Identifiers

PMID30229376
PMCPMC6438935
OpenAlexW2892087921

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.