CORD submission to PMPRB Scoping Paper consultation on new Guidelines

This week CORD submitted its feedback to the PMPRB’s new Scoping Paper on upcoming guidelines. We are encouraged that the PMPRB appears to be returning to its core mandate and is undertaking more meaningful engagement with patients and other stakeholders.

Moving forward, as the PMPRB reflects on its guidelines approach, CORD recommends focusing on these two key questions:

1. How will the PMPRB changes impact the implementation of Canada’s rare disease drug strategy? 
2. How will the new rules affect Canada’s access to new medicines?

Read our full submission to learn more: https://www.raredisorders.ca/content/uploads/CORD-submission-to-PMPRB-Scoping-Paper-consultation-FINAL-Dec-20-2023.pdf

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December 20, 2023

Patented Medicine Prices Review Board (PMPRB)
333 Laurier Avenue West, Suite 1400
Ottawa ON, K1P 1C1

Subject: PMPRB Scoping Paper consultation (2023)

Dear PMPRB Board Members,

The Canadian Organization for Rare Disorders (CORD) is the national alliance of over 100 rare disease patient organizations and representing approximately 3 million Canadians affected by rare diseases. 

On behalf of our patients and families, I would you like to commend you for taking a refreshed perspective on the guidelines development process. We are especially heartened that the PMPRB appears to be returning to its core mandate, which hopefully will help turn the page on what has been a very traumatic chapter for CORD and the rare disease community.

Based on the media hype and disinformation campaigns, one would be excused in imagining that the hospitals and pharmacies are overflowing with expensive rare disease therapies. Nothing could be further from the truth, with less than 5% of rare diseases having an approved treatment and many of these delayed in their entry to Canada, and some, not at all.

However, our community wants to move forward constructively and not dwell on the collective stress and apprehension caused by the proposed changes over the past seven years. On behalf of Canadian rare disease families, of which my family is one, I urge you to keep us forefront in your minds as you deliberate on your new guidelines. Here are examples of why appropriate pricing regulations are essential to rare disease families.

• A young woman diagnosed with fibrodysplasia ossificans progressiva (FOP), a rare musculoskeletal condition whereby, after birth and progressively through life, muscles and tendons are gradually transformed into bone (similar to the condition affecting Celine Dion). A treatment developed in Quebec more than eight years ago has now been approved by Health Canada and recommended by CADTH. It is awaiting consideration for pricing negotiations but the process could be further delayed by the fact that drug was just approved by the FDA this past August so there are limited international benchmarks. 
• A 10-year-old boy with Neurofibromatosis type 1 (NF1), has undergone numerous surgeries to remove tumours throughout his body, and now has new growths near his spine. An oral therapy that has been shown to shrink the tumours, thus reducing pain and avoiding surgery, was approved in 2020 and 2021 in the USA and EU, respectively. Submission to Health Canada was delayed several years until pricing and reimbursement had been secured elsewhere. Several children could have avoided surgery with more timely access.

A 21-year-old student with homozygous familial hypercholesterolemia (HOFH), a genetic condition characterized by very high LDL-C levels, travels four hours every two weeks to receive plasma transfusions. However, these are not very effective and she has experienced several cardiovascular events. A new drug infused monthly is effective in reducing LDL-C by nearly 50%. Its submission in Canada was delayed for about two years until after EU and USA had negotiated pricing and reimbursement. She suffered her last attack during that delay.

These are only a few examples of the many serious impacts on rare disease patients, with many companies choosing to delay or even forego bringing life-altering and life-saving therapies to Canada, citing the unreasonable, un-negotiable and unprecedented changes in how PMPRB operates, which would have put our pricing ceilings far below other OECD countries. Rare disease patients are still suffering from delayed and no entry of therapies during these past years, many of which are the first for that condition.

To illustrate these challenges, we have updated and attached a list of medicines approved by the United States Food & Drug Agency versus submissions to Health Canada, finding that since the first formal Patented Medicines Regulations changes were published on August 22, 2019, only 102 out of the 197 medicines already approved in the US have even been submitted for review by Health Canada as of December 6, 2023. This means that Canadians have access to only about half of the medicines available just south of the border, including treatments for many cancers, Alzheimer’s disease, diabetes, and other serious diseases. These are deliberate choices made by companies driven in part by the uncertainty surrounding compliant pricing and the potential impacts on pricing in other markets.

As patients, we wholeheartedly endorse the essence of the PMPRB’s mandate, which is to prevent excessive prices for patented medicines and report on pricing and research trends in Canada. Sadly, the previous reforms strayed far from this objective. The focus on driving prices down without adequately considering the impact on patient access contradicts the foundation upon which this organization was built.

In light of these issues, CORD takes some comfort that the PMPRB has begun to engage in more meaningful consultations, and this starts with considering the real impact of pricing policies on patients. This can only be achieved through a thorough review of operational changes with practical case studies. Collaboration with patients, clinicians, payers, and stakeholders is crucial in formulating pricing guidelines that serve everyone’s interests. 

A measure of success or failure for the PMPRB needs to be an understanding of how its regulatory role – reviewing prices for potential excessiveness – is either enabling or obstructing the opportunity to access rare disease research and medicines in Canada. 

As the PMPRB begins to reflect on its guidelines approach, we encourage you to consider the following key questions:

1. How will the PMPRB changes enable the implementation of Canada’s rare disease drug strategy?

• How would updated price ceilings affect the capacity for health systems and payers to negotiate dynamic agreements that provide access to medicines and the full range of care required for precision medicines, including cell and gene therapies? In Canada, our payers are getting creative and negotiating managed access programs and outcomes-based agreements. How can the PMPRB support these efforts, which are the hallmark of access for rare diseases in other countries and, increasingly, in Canada?

• How do you plan to benchmark or monitor for excessive prices when a medicine is only available in a jurisdiction that isn’t within the PMPRB11, for example, the US, where most medicines are made available first?

CORD has been active in the development and implementation of rare disease strategies and we are ready to bring our collective ingenuity to bear on these and other questions as you move forward. We would welcome the opportunity to take part in any guidelines steering groups, as we have done in the past.

Thank you for the opportunity to provide input.

Sincerely,

Durhane Wong-Rieger, PhD
President & CEO
durhane@raredisorders.ca