Nov 19 2012

Health Canada Orphan Drug Policy – A Long Overdue Need

Health CanadaCanada is one of the only first world countries without an orphan drug policy. The United States, European Union, Australia, Singapore, and Japan recognized a long time ago the importance of having established policies around the development of orphan drugs.

In a 1996 report from Health Canada’s Drugs Directorate that analyzed the need for an orphan drug policy in Canada, it was concluded that there was sufficient provision for drug availability for patients with orphan diseases through the special access program, patient enrollment in international clinical trials, importation of drugs for personal use, and the existing early approval mechanisms; by priority review and conditional approval of drugs.

In the ensuing years there have been multiple pleas to the regulatory agency to revisit the orphan drug policy issue.  Mountains of proof contradict the conclusions made in the 1996 report and in reality patients with orphan diseases in Canada represent grossly under-served populations. Since 2005, of the 63 orphan drugs approved in the United States of America, only 42 were approved in Canada . Of the 49 orphan drugs approved in the European Union, only 29 have been approved in Canada; quite clearly Canadian patients with orphan diseases do not have the same access as their international counterparts to the appropriate treatments.

At the DIA’s Annual Canadian meeting in November 2012 in Ottawa, patient advocacy groups recounted stories of making personal pleas to pharmaceutical companies to bring their drugs to Canada, to little avail. Canada’s lack of provision for orphan drug development provides no incentive for developers to come to Canada; the gauntlet of obtaining provincial reimbursement of drugs augments this unenthusiastic attitude.

However, this year has heralded a change. Canada recently joined Orphanet, the European based reference portal for rare diseases, and on 03 October 2012, the government of Canada announced a new regulatory roadmap around Orphan Drug development. Within this framework a new division of Orphan Drug Development will be established within the Health Products and Food Branch.  In order to increase the speed of implementing the framework, the Office of Legislative and Regulatory Modernization are leading the effort to develop the regulations simultaneously with guidance documents and an infrastructure of standard operating procedures and processes.

Health Canada is working in close collaboration with their counterparts in the EU and US to develop an orphan drug framework around the concept of life-cycle management, whereby data is collected on an ongoing basis to ensure that even with limited patient information, drugs can be approved and mechanisms implemented to ensure the continuation of data collection and analysis to confirm ongoing drug safety and efficacy.  Health Canada is also establishing efforts to share information with international authorities in order to develop policies and engage technical discussion.  In addition,  they are looking to determine whether it is feasible to develop a common application for the EU, Japan, and Canada.

Canada’s policy will define an orphan drug as one that treats life-threatening and debilitating conditions that occur in less than 5 in 10,000 people. The incentives proposed by Health Canada include priority review, waivers and remissions from filing fees, protocol advice and input from patients, experts, and advocacy groups, and intellectual property protection – although this will remain under Industry Canada jurisdiction. The objective of the policy is to reduce red tape, establish a streamlined process, and ultimately serve the massive unmet medical need of orphan diseases without compromising on the safety of the patient populations.

Overall, the move is nothing but a positive one in the eyes of the patients, drug developers, and health care providers. If a policy is in place, at least this declares to drug developers that Canada is open for business for orphan drugs. Canada has a long way to go, but being so far behind in entering the field of orphan drug development, many years of lessons learned by the international regulators can be applied while defining the upcoming policy and guidance.  Health Canada, the world awaits your policy…..the release date still remains elusive.

This post was written by Catherine Sheppard, Ph.D.  Catherine is the Assistant Director, Regulatory Strategy for Cato Research Canada.