Drugs for Rare Diseases Receive Overdue Policy Attention in Canada

espite significant efforts over the past decade–and the fact that the world’s major regulatory bodies long ago developed tailored regulatory frameworks for orphan and/or drugs for rare diseases, or DRDs–Canada remains one of the few developed countries without a specific orphan drug regulatory framework.

Background

Health Canada published an Initial Draft Discussion Document for A Canadian Orphan Drug Regulatory Framework in December 2012, intended to encourage dialogue on the development of a specific pathway for DRDs. Unfortunately, after years of consultations Health Canada did not end up formally adopting the framework, although it did launch an effort to bring clarity to its regulatory approach to DRDs in 2018.

In the meantime, provincial governments stalled on creating any specific pathways for funding of DRDs, waiting for the Health Canada framework to be finalized. The Ontario Ministry of Health had established its own framework for DRDs in December 2007, while in 2016 the national health technology assessment (HTA) body, Canadian Agency for Drugs and Technologies in Health (CADTH), declined to create specific processes for DRDs, despite strong advocacy efforts on this issue.

Unfortunately, this left patients at the mercy of a highly inconsistent and inequitable patchwork of approaches to the evaluation and funding of DRDs across the country. These challenges in patient access were clearly articulated in the Rare Disease Strategy released by the Canadian Organization for Rare Disorders (CORD) in May 2015.

Renewed Provincial-Territorial Efforts

The Expensive Drugs for Rare Diseases (EDRD, also referred to as DRDs, Working Group [WG]) was established in 2014 to explore opportunities to improve the management of rare disease drug therapies with evidence-based approaches focusing on four core areas: evidence, pricing, access, and communications.

To address some of the identified challenges, this WG launched in October 2018 consultations on a proposal for a supplemental drug review process for complex/specialized drugs (including DRDs) that builds upon the existing national and jurisdictional review processes. The main objective of this proposal is “to implement a proactive, consistent, fair and transparent process to assess complex/specialized drugs for the purpose of making responsive funding decisions.”

Most importantly, the proposal begins to acknowledge the need for different considerations in the assessment of DRDs, noting that they have a broader impact and thus a greater value than what is captured in clinical trials or other current methods of assessing these products.

Specifically, the proposal includes modifications to the current national review process–including improved use of real world evidence (RWE) to inform regulatory/HTA evaluations and funding decisions and the use of centralized panels of experts for consistent implementation decisions. It also offers the opportunity for eligible drugs to be submitted concurrently to Health Canada, CADTH, PMPRB (the Patented Medicine Prices Review Board), and pCPA (the pan-Canadian Pharmaceutical Alliance) to help reduce overall submission review time.

Input from stakeholder consultations will be used to refine the proposal, with potential implementation of a supplemental process in Spring/Summer 2019.

Pressure on the Federal Government

Public drug plans, collectively as pCPA and individually as jurisdictions, have the role and responsibility to support the public by making appropriate drug funding decision that are evidence-informed, cost-effective, and affordable. The pCPA itself undertakes pricing negotiations for brand name and generic drugs on behalf of these publicly funded drug programs. In December 2018, the pCPA submitted a brief on behalf of the public drug plans to the House of Commons’ Standing Committee on Barriers to Access Treatment and Drugs for Canadians Affected by Rare Diseases and Disorders.

According to the pCPA brief, when it comes to DRDs, public drug plans in Canada and elsewhere around the world are experiencing significant challenges related to: (1) evidence limitations, often inadequate evidence around drug efficacy, safety, and cost-effectiveness to support drug coverage decisions; (2) extremely high drug prices and growing costs threatening drug program affordability, sustainability, and, therefore, patient access; and, (3) gaps in national alignment and coordination of processes related to DRDs.

To address these challenges, they proposed a major role for the federal government in the context of market access to DRDs; that is, federal funding support for these drugs, implementation of pricing controls via the proposed PMPRB modernization changes, and collaboration with provincial/territorial public drug plans and the pCPA to better align, collaborate, and coordinate approaches to address DRD evidence and pricing issues.

The Standing Committee has not yet completed its report on DRD barrier issues, so it is not clear how the pCPA brief will influence their recommendations. There has been no Health Canada response to the points raised by the pCPA to date.

Implications and Conclusions

• After many years of discussion, the Canadian environment now seems primed to take action on creating a specific HTA and funding pathway for DRDs.
• The role of RWE in the proposed HTA framework aligns with broader discussion of integrating RWE into decision-making throughout various parts of the Canadian pharmaceutical system.
• It is clear that the provincial/territorial and pCPA initiatives noted above will put a great deal of pressure on Health Canada to take a leadership role in DRD policy, and to develop new approaches for managing the complexity of issues associated with DRDs.