As a result of their deliberations committees may make recommendations which they include in their reports for the consideration of the House of Commons or the Government. Recommendations related to this study are listed below.

Health Canada’s Market Authorization of Drugs for Rare Diseases

Recommendation 1

That the Government of Canada, in collaboration with the provinces and territories, develop a coordinated process for the market authorization and reimbursement of drugs for rare diseases.

Recommendation 2

That the Government of Canada work to ensure greater transparency and information sharing throughout the life cycle of drugs for rare diseases to ensure timely access for key decision-makers, including health care providers, health technology assessors and patients.

Recommendation 3

That the Government of Canada in collaboration with the provinces and territories develop a national, independent, expert review panel to provide recommendations and guidance on the regulatory review, pricing and reimbursement of drugs for rare diseases in Canada, including instructions on how to streamline these processes; and report publicly on its findings.

Recommendation 4

That Health Canada and the Canadian Agency for Drugs and Technologies in Health undertake their respective scientific evidence review processes of drugs for rare diseases in tandem as a standard practice.

Recommendation 5

That Health Canada, in collaboration with the Canadian Agency for Drugs and Technologies in Health, provide guidance and advice to drug manufacturers in the design of clinical trials to ensure that they meet the requirements of both market authorization and reimbursement processes in Canada.

Recommendation 6

That Health Canada consider removing regulatory requirements for drug manufacturers to seek additional approval for an open-label extension for drugs at the completion of a clinical trial to ensure that patients have uninterrupted access to these drugs if no safety concerns are present, in line with regulatory practices in the United States.

Recommendation 7

That Health Canada consider reducing regulatory submission fees for manufacturers of drugs for rare diseases seeking to obtain market authorization for the drugs in Canada.

Recommendation 8

That Health Canada be more proactive in its communications with physicians and patients regarding the specific medical need criteria required for obtaining access to drugs through the Special Access Programme.

Recommendation 9

That the Government of Canada remove the requirement to reapply to the Special Access Programme every three to six months when accessing a drug for a permanent, stable condition. Once initially approved, Canadians' approvals should remain in place until a doctor rescinds the approval or the patient's condition changes significantly.

Recommendation 10

That Health Canada ensure that drug manufacturers meet their regulatory obligations when Notice of Compliance with conditions are granted for drugs where limited evidence is available regarding their quality, safety and efficacy.

Drug Prices

Recommendation 11

That the Government of Canada move forward with implementing proposed changes to the Patented Medicines Regulations to address high drug prices in Canada.

Recommendation 12

That the Government of Canada consider establishing separate requirements for determining price ceilings for drugs for rare diseases under the Patented Medicines Regulations to reflect the small market for these drugs in Canada.

Recommendation 13

That the Patented Medicine Prices Review Board be required to consider the advice and recommendations of the proposed independent advisory committee on drugs for rare diseases in setting the price ceilings for drugs for rare diseases.

Recommendation 14

That the Government of Canada introduce additional regulatory requirements under section 88(1) (c) of the Patent Act that require manufacturers of patented pharmaceuticals to provide information to the Patented Medicine Prices Review Board regarding their research and development costs for a drug once they have obtained market authorization from Health Canada.

Recommendation 15

That the Government of Canada undertake a review of the entire pharmaceutical research and manufacturing process to better understand where government regulations and laws are having the unintended consequences of raising final drug costs for patients. This review should include an examination of whether drug costs could be reduced through open science.

Reimbursement of Drugs for Rare Diseases

Recommendation 16

That the Government of Canada, in collaboration with the provinces, territories and drug manufacturers, establish a jointly funded compassionate care program that covers the costs of drugs for rare diseases while they are under review for market authorization and cost reimbursement.

Recommendation 17

That the reimbursement of drugs for rare diseases be included as part of a national pharmacare program established by the Government of Canada, in collaboration with the provinces and territories, through amendments to the Canada Health Act, as recommended by the House of Commons Standing Committee on Health in its report entitled Pharmacare Now: Prescription Medicine Coverage For All Canadians.

Recommendation 18

That the Office of the Auditor General conduct an audit of Health Canada to determine whether it has been effective in managing its funding agreement with the Canadian Agency for Drugs and Technologies in Health, including determining whether Health Canada is effectively ensuring that the Agency is fulfilling its mandate in accordance with agreed terms and conditions of the agreement with Health Canada.

Research

Recommendation 19

That the Government of Canada provide funding through the Canadian Institutes of Health Research for research into the diagnosis of patients with rare diseases and the collection of real-world evidence regarding the effectiveness of treatments for these conditions.