Details
| Original language | English |
|---|---|
| Article number | 47 |
| Number of pages | 12 |
| Journal | Orphanet journal of rare diseases |
| Volume | 19 |
| Publication status | Published - 7 Feb 2024 |
Abstract
Health technology assessment (HTA) decisions for pharmaceuticals are complex and evolving. New rare disease treatments are often approved more quickly through accelerated approval schemes, creating more uncertainties about clinical evidence and budget impact at the time of market entry. The use of real-world evidence (RWE), including early coverage with evidence development, has been suggested as a means to support HTA decisions for rare disease treatments. However, the collection and use of RWE poses substantial challenges. These challenges are compounded when considered in the context of treatments for rare diseases. In this paper, we describe the methodological challenges to developing and using prospective and retrospective RWE for HTA decisions, for rare diseases in particular. We focus attention on key elements of study design and analyses, including patient selection and recruitment, appropriate adjustment for confounding and other sources of bias, outcome selection, and data quality monitoring. We conclude by offering suggestions to help address some of the most vexing challenges. The role of RWE in coverage and pricing determination will grow. It is, therefore, necessary for researchers, manufacturers, HTA agencies, and payers to ensure that rigorous and appropriate scientific principles are followed when using RWE as part of decision-making.
Keywords
- Cell and gene therapy, Coverage determination, Coverage with evidence development, Health technology assessment, Rare disease, Real world evidence
ASJC Scopus subject areas
- Medicine(all)
- Genetics(clinical)
- Medicine(all)
- Pharmacology (medical)
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In: Orphanet journal of rare diseases, Vol. 19, 47, 07.02.2024.
Research output: Contribution to journal › Article › Research › peer review
}
TY - JOUR
T1 - Real-world evidence for coverage determination of treatments for rare diseases
AU - Dayer, Victoria W.
AU - Drummond, Michael F.
AU - Dabbous, Omar
AU - Toumi, Mondher
AU - Neumann, Peter
AU - Tunis, Sean
AU - Teich, Nelson
AU - Saleh, Shadi
AU - Persson, Ulf
AU - von der Schulenburg, Johann Matthias Graf
AU - Malone, Daniel C.
AU - Salimullah, Tay
AU - Sullivan, Sean D.
N1 - Funding Information: The authors thank Catrena J. Sullivan for editorial assistance. Editorial support was also provided by Jennifer Weintraub of Kay Square Scientific, Newtown Square, PA, USA, and this support was funded by Novartis Gene Therapies, Inc.
PY - 2024/2/7
Y1 - 2024/2/7
N2 - Health technology assessment (HTA) decisions for pharmaceuticals are complex and evolving. New rare disease treatments are often approved more quickly through accelerated approval schemes, creating more uncertainties about clinical evidence and budget impact at the time of market entry. The use of real-world evidence (RWE), including early coverage with evidence development, has been suggested as a means to support HTA decisions for rare disease treatments. However, the collection and use of RWE poses substantial challenges. These challenges are compounded when considered in the context of treatments for rare diseases. In this paper, we describe the methodological challenges to developing and using prospective and retrospective RWE for HTA decisions, for rare diseases in particular. We focus attention on key elements of study design and analyses, including patient selection and recruitment, appropriate adjustment for confounding and other sources of bias, outcome selection, and data quality monitoring. We conclude by offering suggestions to help address some of the most vexing challenges. The role of RWE in coverage and pricing determination will grow. It is, therefore, necessary for researchers, manufacturers, HTA agencies, and payers to ensure that rigorous and appropriate scientific principles are followed when using RWE as part of decision-making.
AB - Health technology assessment (HTA) decisions for pharmaceuticals are complex and evolving. New rare disease treatments are often approved more quickly through accelerated approval schemes, creating more uncertainties about clinical evidence and budget impact at the time of market entry. The use of real-world evidence (RWE), including early coverage with evidence development, has been suggested as a means to support HTA decisions for rare disease treatments. However, the collection and use of RWE poses substantial challenges. These challenges are compounded when considered in the context of treatments for rare diseases. In this paper, we describe the methodological challenges to developing and using prospective and retrospective RWE for HTA decisions, for rare diseases in particular. We focus attention on key elements of study design and analyses, including patient selection and recruitment, appropriate adjustment for confounding and other sources of bias, outcome selection, and data quality monitoring. We conclude by offering suggestions to help address some of the most vexing challenges. The role of RWE in coverage and pricing determination will grow. It is, therefore, necessary for researchers, manufacturers, HTA agencies, and payers to ensure that rigorous and appropriate scientific principles are followed when using RWE as part of decision-making.
KW - Cell and gene therapy
KW - Coverage determination
KW - Coverage with evidence development
KW - Health technology assessment
KW - Rare disease
KW - Real world evidence
UR - http://www.scopus.com/inward/record.url?scp=85184739660&partnerID=8YFLogxK
U2 - 10.1186/s13023-024-03041-z
DO - 10.1186/s13023-024-03041-z
M3 - Article
C2 - 38326894
AN - SCOPUS:85184739660
VL - 19
JO - Orphanet journal of rare diseases
JF - Orphanet journal of rare diseases
SN - 1750-1172
M1 - 47
ER -