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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.

References

Komastubara, K, Carvajal, R. The promise and challenges of rare cancer research. Lancet Oncol. 2016;17:136–7.CrossRefGoogle Scholar
Boyd, N, Dancey, JE, Gilks, CB, Huntsman, DG. Rare cancers: A sea of opportunity. Lancet Oncol. 2016;17:e52–61. Available from: http://dx.doi.org/10.1016/S1470-2045(15)00386-1CrossRefGoogle Scholar
Jeffcott, G. Rethinking the reimbursement review of orphan medicines in Canada. Prov Reimburse Advis. 2019;22:10–7.Google Scholar
Nestler-Parr, S, Korchagina, D, Toumi, M, Pashos, CL, Blanchette, C, Molsen, E, et al. Challenges in research and health technology assessment of. Value Health. 2018;21:493–500. Available from: https://doi.org/10.1016/j.jval.2018.03.004CrossRefGoogle ScholarPubMed
Guindo, LA, Wagner, M, Baltussen, R, Rindress, D, van Til, J, Kind, P, et al. From efficacy to equity: Literature review of decision criteria for resource allocation and healthcare decision making. Cost Eff Resour Alloc. 2012;10:9. Available from: http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=3495194&tool=pmcentrez&rendertype=abstractCrossRefGoogle Scholar
Wagner, M, Samaha, D, Casciano, R, Brougham, M, Abrishami, P, Petrie, C, et al. Moving towards accountability for reasonableness – A systematic exploration of the features of legitimate healthcare coverage decision-making processes using rare diseases and regenerative therapies as a case study. Int J Health Policy Manag. 2019;8:424–43. Available from: https://doi.org/10.15171/ijhpm.2019.24CrossRefGoogle ScholarPubMed
Sandman, L, Gustavsson, E. The (Ir)relevance of group size in health care priority setting: A reply to Juth. Health Care Anal. 2017;25:21–33.CrossRefGoogle ScholarPubMed
Baltussen, R, Marsh, K, Thokala, P, Diaby, V, Castro, H, Cleemput, I, et al. Multicriteria decision analysis to support health technology assessment agencies: Benefits, limitations, and the way forward. Value Health. 2019;22:1283–8. Available from: https://doi.org/10.1016/j.jval.2019.06.014CrossRefGoogle ScholarPubMed
Baran-Kooiker, A, Czech, M, Kooiker, C. Multi-Criteria decision analysis (MCDA) models in health technology assessment of orphan drugs—a systematic literature review. Next steps in methodology development? Front Public Health. 2018;6(October):287.CrossRefGoogle ScholarPubMed
CADTH. Drugs for rare diseases: a review of national and international health technology assessment agencies and public payers’ decision-making processes (Environmental scan; no. 77). 2018.Google Scholar
Hughes, DA, Tunnage, B, Yeo, ST. Drugs for exceptionally rare diseases: Do they deserve special status for funding? QJM – Mon J Assoc Physicians. 2005;98:829–36.CrossRefGoogle ScholarPubMed
McCormick, JI, Berescu, LD, Tadros, N. Common drug review recommendations for orphan drugs in Canada: Basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand. Orphanet J Rare Dis. 2018;13:1–12.CrossRefGoogle ScholarPubMed
Adkins, EM, Nicholson, L, Floyd, D, Ratcliffe, M, Chevrou-Severac, H. Oncology drugs for orphan indications: How are HTA processes evolving for this specific drug category? Clin Outcomes Res. 2017;9:327–42.CrossRefGoogle ScholarPubMed
CADTH, pCODR. pCODR Expert Review Committee Deliberative Framework [Internet]. 2016 [cited 2019 Aug 8]. Available from: https://www.cadth.ca/sites/default/files/pcodr/ThepCODRExpertReviewCommittee%28pERC%29/pcodr_perc_deliberative_frame.pdfGoogle Scholar
CADTH. Recommendation Framework for CADTH Common Drug Review and pan-Canadian Oncology Drug Review Programs: Guidance for CADTH's Drug Expert Committees [Internet]. 2016 [cited 2019 Aug 13]. Available from: https://www.cadth.ca/media/cdr/templates/pre-sub-phase/CDR_pCODR_recommendations_framework.pdfGoogle Scholar
Schey, C, Milanova, T, Hutchings, A. Estimating the budget impact of orphan medicines in Europe: 2010–2020. Orphanet J Rare Dis. 2011;6:62. Available from: http://ww.ojrd.com/content/6/1/62CrossRefGoogle ScholarPubMed
Health Technology Assessment and Optimal Use: Medical Devices; Diagnostic Tests; Medical, Surgical, and Dental Procedures [Internet]. CADTH. 2015 [cited 2018 May 17]. p. 1–4. Available from: https://www.cadth.ca/sites/default/files/pdf/HTA_OU_Topic_ID_and_Prioritization_Process.pdfGoogle Scholar
Rawson, NSB. Health technology assessment of new drugs for rare disorders in Canada: Impact of disease prevalence and cost. Orphanet J Rare Dis. 2017;12:1–5.CrossRefGoogle ScholarPubMed
Clarke, JTR, Coyle, D, Evans, G, Martin, J, Winquist, E. Toward a functional definition of a “rare disease” for regulatory authorities and funding agencies. Value Health. 2014;17:757–61. Available from: http://dx.doi.org/10.1016/j.jval.2014.08.2672CrossRefGoogle Scholar
Canadian Cancer Society, Statistics Canada, Public Health Agency of Canada. Canadian Cancer Statistics [Internet]. 2018. Available from: https://www.cancer.ca/~/media/cancer.ca/CW/cancer information/cancer 101/Canadian cancer statistics/Canadian-Cancer-Statistics-2018-EN.pdf?la=enGoogle Scholar
Billingham, L, Malottki, K, Steven, N. Research methods to change clinical practice for patients with rare cancers. Lancet Oncol. 2016;17:e70–80. Available from: http://dx.doi.org/10.1016/S1470-2045(15)00396-4CrossRefGoogle ScholarPubMed
Graf von der Schulenburg, JM, Pauer, F. Rare cancers—rarity as a cost and value argument. J Cancer Policy. 2017;11:54–9. Available from: http://dx.doi.org/10.1016/j.jcpo.2016.09.004CrossRefGoogle Scholar
PCODR. Find a review (pCODR) [Internet]. 2018 [cited 2018 May 31]. Available from: https://www.cadth.ca/pcodr/find-a-reviewGoogle Scholar
Gaasterland, CMW, Van Der Weide, MCJ, Du Prie-Olthof, MJ, Donk, M, Kaatee, MM, Kaczmarek, R, et al. The patient's view on rare disease trial design—A qualitative study. Orphanet J Rare Dis. 2019;14:1–9.CrossRefGoogle ScholarPubMed
Prasad, V, Oseran, A. Do we need randomised trials for rare cancers? Eur J Cancer. 2015;51:1355–7. Available from: http://dx.doi.org/10.1016/j.ejca.2015.04.015CrossRefGoogle ScholarPubMed
Luth, W, Ali-Khan, S, Bubela, T, Saltonstall, PL, McMillan, HJ, Campbell, C, et al. Towards a new way of evaluating orphan drugs at CADTH. Orphanet J Rare Dis. 2016;13:E1274–5. Available from: http://www.canadianinstitute.com/content/download-content/marketing-materials/350X16/Orphan-Drugs-at-CADTH_McKesson.pdf%0Ahttp://www.tandfonline.com/doi/full/10.1517/21678707.2014.971012%0Ahttp://paceomics.org/wp-content/uploads/2015/10/Canadas-Orphan-DruGoogle Scholar
Husereau, D. How do we value a cure? Expert Rev Pharmacoecon Outcomes Res. 2015;15:551–5.CrossRefGoogle ScholarPubMed
Julkowska, D, Austin, CP, Cutillo, CM, Gancberg, D, Hager, C, Halftermeyer, J, et al. The importance of international collaboration for rare diseases research: A European perspective. Gene Ther. 2017;24:562–71. Available from: http://dx.doi.org/10.1038/gt.2017.29CrossRefGoogle ScholarPubMed
Wood, M. A Report of the Ontario Citizens’ Council: Drugs for Rare Diseases. 2018.Google Scholar
Richter, T, Nestler-Parr, S, Babela, R, Khan, ZM, Tesoro, T, Molsen, E, et al. Rare disease terminology and definitions-A systematic global review: Report of the ISPOR rare disease special interest group. Value Health. 2015;18:906–14. Available from: http://dx.doi.org/10.1016/j.jval.2015.05.008CrossRefGoogle ScholarPubMed
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