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Impact of rarity on Canadian oncology health technology assessment and funding

Published online by Cambridge University Press:  11 August 2020

James Keech Open the ORCID record for James Keech [Opens in a new window] ,
Wei Fang Dai ,
Maureen Trudeau ,
Rebecca E. Mercer ,
Rohini Naipaul ,
Frances C. Wright ,
Sarah E. Ferguson ,
Gail Darling ,
Scott Gavura  and
Andrea Eisen
...Show all authors
James Keech
Affiliation:
Cancer Care Ontario & Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada
Wei Fang Dai
Affiliation:
Cancer Care Ontario & Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada
Maureen Trudeau
Affiliation:
Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada
Rebecca E. Mercer
Affiliation:
Cancer Care Ontario & Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada
Rohini Naipaul
Affiliation:
Cancer Care Ontario, Toronto, Ontario, Canada
Frances C. Wright
Affiliation:
Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada
Sarah E. Ferguson
Affiliation:
University Health Network, Toronto, Ontario, Canada
Gail Darling
Affiliation:
University Health Network, Toronto, Ontario, Canada
Scott Gavura
Affiliation:
Cancer Care Ontario & Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada
Andrea Eisen
Affiliation:
Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada
C. Tom Kouroukis
Affiliation:
Juravinski Cancer Centre, Hamilton Health Sciences Centre, Hamilton, Ontario, Canada
Jaclyn Beca
Affiliation:
Cancer Care Ontario & Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada
Kelvin K.W. Chan*
Affiliation:
Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada
*
Author for correspondence: Kelvin Chan, E-mail: kelvin.chan@sunnybrook.ca
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Abstract

Objectives

The pan-Canadian Oncology Drug Review (pCODR) evaluates new cancer drugs for public funding recommendations. While pCODR's deliberative framework evaluates overall clinical benefit and includes considerations for exceptional circumstances, rarity of indication is not explicitly addressed. Given the high unmet need that typically accompanies these indications, we explored the impact of rarity on oncology HTA recommendations and funding decisions.

Methods

We examined pCODR submissions with final recommendations from 2012 to 2017. Incidence rates were calculated using pCODR recommendation reports and statistics from the Canadian Cancer Society. Indications were classified as rare if the incidence rate was lower than 1/100,000 diagnoses, a definition referenced by the Canadian Agency for Drugs and Technologies in Health. Each pCODR final report was examined for the funding recommendation/justification, level of supporting evidence (presence of a randomized control trial [RCT]), and time to funding (if applicable).

Results

Of the ninety-six pCODR reviews examined, 16.6 percent were classified as rare indications per above criteria. While the frequency of positive funding recommendations were similar between rare and nonrare indication (78.6 vs. 75 percent), rare indications were less likely to be presented with evidence from RCT (50 vs. 90 percent). The average time to funding did not differ significantly across provinces.

Conclusion

Rare indications appear to be associated with weaker clinical evidence. There appears to be no association between rarity, positive funding recommendations, and time to funding. Further work will evaluate factors associated with positive recommendations and the real-world utilization of funded treatments for rare indications.

Keywords

Health technology assessment (HTA)RarityDrug fundingUnmet need
Type
Assessment
Information
International Journal of Technology Assessment in Health Care , Volume 36 , Issue 4 , August 2020 , pp. 404 - 409
Copyright
Copyright © The Author(s), 2020. Published by Cambridge University Press

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Footnotes

*

J Keech and W F Dai contributed equally to this work.

†

J Beca and K Chan are co-senior authors.

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