Expensive Drugs for Rare Diseases in Canada: What Value and at What Cost?

Sandra Sirrs; Helen Anderson; Bashir Jiwani; Larry D Lynd; Eric Lun; Bob Nakagawa; Dean Regier; Shirin Rizzardo; Anne McFarlane

doi:10.12927/hcpap.2023.27000

Expensive Drugs for Rare Diseases in Canada: What Value and at What Cost?

Healthc Pap. 2023 Jan;21(1):10-26. doi: 10.12927/hcpap.2023.27000.

Authors

Sandra Sirrs¹, Helen Anderson², Bashir Jiwani³, Larry D Lynd⁴, Eric Lun⁵, Bob Nakagawa⁶, Dean Regier⁷, Shirin Rizzardo⁸, Anne McFarlane⁹

Affiliations

¹ Clinical Professor, Division of Endocrinology, Department of Medicine, University of British Columbia, Vancouver, BC.
² Director, Systemic Therapy, BC Cancer, Victoria, BC.
³ Lead Ethicist and Executive Director, Ethics and Diversity Services, Fraser Health Authority, Surrey, BC.
⁴ Associate Dean, Research, Professor and Director, Collaboration for Outcomes Research and Evaluation (CORE), Faculty of Pharmaceutical Sciences, University of British Columbia, Vancouver, BC.
⁵ Executive Director, BC Transplant, Provincial Specialized Programs and Clinical Policy, Provincial Health Services Authority, Vancouver, BC.
⁶ Former Assistant Deputy Minister - Pharmaceutical Services Division, BC Ministry of Health, Coquitlam, BC.
⁷ Clinical Scientist, Cancer Control Research, BC Cancer, Associate Professor, School of Population and Public Health, University of British Columbia, Vancouver, BC.
⁸ Senior Economic and Socioeconomic Analyst, Patented Medicine Prices Review Board, Vancouver, BC.
⁹ Chair, British Columbia Expensive Drugs for Rare Diseases Advisory Committee, Provincial Health Services Authority, Victoria, BC.

PMID: 36692912
DOI: 10.12927/hcpap.2023.27000

Abstract

There has been explosive growth in the market for expensive drugs for rare diseases (EDRDs). Traditional standards of evidence are not achievable for rare diseases, so lower standards are applied. The price of these drugs is extremely high. This combination of lower standards and higher prices make EDRDs attractive to manufacturers. Legislation designed to incentivize drug development for rare diseases contains loopholes that drive prices up worldwide. Canada compounds those problems with a complex network of agencies that impede communication between those providing market authorization and those purchasing drugs. Drug pricing is not related to metrics like investment or value, but rather willingness to pay. Without high-quality evidence to assess value, we inadvertently prioritize patients with rare diseases over those with common diseases, creating conflict among ethical principles such as social utility, justice and the rule of rescue. Lack of transparency over what is being funded and for whom makes it hard to mitigate challenges through effective policy development. We review the evidentiary, economic and ethical issues around EDRDs and ways to move forward, including enhanced transparency and the development of high-quality evidence to ensure that we do not pay for drugs that do not work.

Publication types

Review

MeSH terms

Canada
Cost Control
Drug Costs*
Humans
Insurance, Health, Reimbursement
Rare Diseases* / drug therapy