Change search
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association-8th-edition
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf
Balancing early access with uncertainties in evidence for drugs authorized by prospective case series - systematic review of reimbursement decisions
Univ Gothenburg, Sweden; Sahlgrens Univ Hosp, Sweden.
Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences. Linköping University, Center for Medical Image Science and Visualization (CMIV).
2018 (English)In: British Journal of Clinical Pharmacology, ISSN 0306-5251, E-ISSN 1365-2125, Vol. 84, no 6, p. 1146-1155Article, review/survey (Refereed) Published
Abstract [en]

AimsTo review clinical and cost-effectiveness evidence underlying reimbursement decisions relating to drugs whose authorization mainly is based on evidence from prospective case series. MethodsA systematic review of all new drugs evaluated in 2011-2016 within a health care profession-driven resource prioritization process, with a market approval based on prospective case series, and a reimbursement decision by the Swedish Dental and Pharmaceutical Benefits Agency (TLV). Public assessment reports from the European Medicines Agency, published pivotal studies, and TLV, Scottish Medicines Consortium and National Institute of Health and Care Excellence decisions and guidance documents were reviewed. ResultsSix drug cases were assessed (brentuximab vedotin, bosutinib, ponatinib, idelalisib, vismodegib, ceritinib). The validity of the pivotal studies was hampered by the use of surrogate primary outcomes and the absence of recruitment information. To quantify drug treatment effect sizes, the reimbursement agencies primarily used data from another source in indirect comparisons. TLV granted reimbursement in five cases, compared with five in five cases for Scottish Medicines Consortium and four in five cases for National Institute of Health and Care Excellence. Decision modifiers, contributing to granted reimbursement despite hugely uncertain cost-effectiveness ratios, were, for example, small population size, occasionally linked to budget impact, severity of disease, end of life and improved life expectancy. ConclusionFor drugs whose authorization is based on prospective case series, most applications for reimbursement within public health care are granted. The underlying evidence has limitations over and above the design per se, and decision modifiers are frequently referred to in the value-based pricing decision making.

Place, publisher, year, edition, pages
WILEY , 2018. Vol. 84, no 6, p. 1146-1155
Keywords [en]
cost-effectiveness; decision making; uncertainty; evidence-based health care; prospective case series
National Category
Gerontology, specialising in Medical and Health Sciences
Identifiers
URN: urn:nbn:se:liu:diva-149371DOI: 10.1111/bcp.13531ISI: 000434063100006PubMedID: 29381234OAI: oai:DiVA.org:liu-149371DiVA, id: diva2:1229749
Note

Funding Agencies|Swedish Dental and Pharmaceutical Benefits Agency (TLV); ALF grants [ALFGBG-428711, ALFGBG-716941]

Available from: 2018-07-02 Created: 2018-07-02 Last updated: 2018-08-03

Open Access in DiVA

fulltext(464 kB)2 downloads
File information
File name FULLTEXT02.pdfFile size 464 kBChecksum SHA-512
c05ec84dae6ea0f8a2f59e9cc38b2ea73dc92f562313c5f9ba904f9d29c94544b63e02627ce1382e251117d210e6b0a874d536a887443c5360f710770baa6a26
Type fulltextMimetype application/pdf

Other links

Publisher's full textPubMed

Search in DiVA

By author/editor
Henriksson, Martin
By organisation
Division of Health Care AnalysisFaculty of Medicine and Health SciencesCenter for Medical Image Science and Visualization (CMIV)
In the same journal
British Journal of Clinical Pharmacology
Gerontology, specialising in Medical and Health Sciences

Search outside of DiVA

GoogleGoogle Scholar
Total: 2 downloads
The number of downloads is the sum of all downloads of full texts. It may include eg previous versions that are now no longer available

doi
pubmed
urn-nbn

Altmetric score

doi
pubmed
urn-nbn
Total: 99 hits
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association-8th-edition
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf