Caring for Canada’s Most Vulnerable Citizens through a National Plan for Rare Disorders

Every year, hundreds of Canadians with severe or lifethreatening disorders do not get access to the medicines they need. What do these patients have in common? They all have rare disorders. It’s almost unbelievable but true. With a public health system that is the envy of much of the world, Canada sadly lags behind almost all other developed nations, including the U.S., the EU, Japan and Australia, in our care and treatment for rare disorders. We’ve made progress over the past few years but it’s time for Canada to stop making excuses and bring in a comprehensive plan that will give Canadians with rare disorders the same treatment as
those with common ones.

A rare disorder is defined as a disorder that affects less than one in 2,000 Canadians. Many are severe, progressive, and/or life threatening conditions, with high mortality rates and devastating impacts on patients, families and society. Although individually rare, collectively, they affect one in 12 persons or 2.8 million Canadians. There are no effective treatments for many of these disorders. However, for many others, if infants or children are diagnosed and treated early enough, they can avoid physical
disability or cognitive delays and live nearly normal lives.

Rare disorders present unique public health challenges. Few physicians are familiar with diagnosing and treating these conditions, which means most patients are undiagnosed, misdiagnosed, or delayed in diagnosis, so they do not get timely
access to treatments even when therapies exist.

There are relatively few therapeutic options – between 7,000 and 8,000 rare disorders have been identified, but there are only 400 effective drug treatments. This situation is expected to change dramatically with hundreds of new therapies in development
and coming to market over the next decade. But without change in our rare disease environment, many of these therapies will not be accessible for Canadian patients. Currently, Canadians tend not to be included in clinical trials and companies are reluctant to bring their drugs to Canada. Approximately half of the rare disease drugs available to patients in the U.S. or Europe have been approved in Canada and only half of these are funded through public drug plans.

Currently, Canada’s regulatory and reimbursement systems present significant challenges to researchers and developers of drugs for rare diseases, also known as orphan drugs. On the federal regulatory side, the review process is about to change.

In October 2012, Health Canada announced their intention to implement an Orphan Drug Regulatory Framework, a review process for orphan drugs that is similar to those of the U.S. and the European Union. Health Canada has indicated that this framework will include tools to improve the quality of knowledge used by provincial and territorial decision-makers and healthcare professionals, provide the opportunity for patient input, encourage transparency and sharing information, and support international collaboration. By harmonizing Canada’s orphan drug regulatory process with those of the U.S. and the EU,
companies will be able to set up clinical trials and to apply to Canada for market approval at about the same time as they do in these other jurisdictions, ultimately reducing delays and increasing the number of new drugs available to Canadians with
rare diseases.

Unfortunately, a federal regulatory framework will not assure equal access across all provinces. Under Canada’s federated model, screening, diagnosis, care and treatment are the responsibility of individual provinces. With an aging population, there is an inevitable increase in both incidence and prevalence of chronic disease, which translates into greater demand for healthcare and greater use of medications. Provinces challenged by healthcare costs that are increasing, on average by seven percent each year, and drug costs that are proportionately rising even faster, have placed more controls on drug prices and restricted access to new drugs.

To those ends, all provinces (except Quebec) have agreed to collaborate on a Common Drug Review (CDR) to assess the comparative effectiveness and cost-effectiveness of new drugs and the pan-Canadian Oncology Drug Review (pCODR) for oncology drugs. Both will soon by operated under one umbrella, the Canadian Agency for Drugs and Technologies in Health (CADTH). Their mandate is to make recommendations to the public drug plans on which drugs to reimburse, under what conditions and, often, at what price. Intended to reduce duplication and improve consistency across formulary listings, the common evaluation methods used by the processes have disadvantaged certain types of drugs resulting in more negative recommendations. These include drugs for small patient populations, first-in-class drugs without comparators, early-market entry drugs for severe or life-threatening conditions, or drugs that primarily impact quality of life, such as pain management or psychological conditions. What is needed is an orphan drug access framework designed specifically to provide access to drugs for
unmet needs as soon as they are approved by Health Canada. Known as managed access schemes, risk-sharing schemes, or  evidence building schemes, they provide appropriate patients reimbursed access to drugs with a monitoring plan that accumulates evidence of safety, effectiveness, and cost-effectiveness over time.  Such schemes are already being used in Canada  and most other countries, and are much more appropriate to rare disease drugs than the CDR or pCODR.

A new step in the process is the pan-Canadian Pricing Alliance (PCPA), which negotiates a single price and listing conditions for a new drug, on behalf of all participating provinces. The final decision about reimbursement, however, is still made by each provincial or territorial government, ostensibly on the basis of budget impact and other factors. Because the latter two steps
are not transparent – the basis for the negotiated price and the reimbursement decision are not publicly available – it is not clear how different jurisdictions arrive at their respective decisions. It remains to be seen whether the PCPA will reduce delays and  improve consistency of access to rare disease drugs.

Finally, many drugs for rare diseases fall outside of the established regulatory and reimbursement pathways, so patient access is evaluated on a case-by-case basis. These include drugs that are not approved in Canada, are used off-label for a rare condition, or are not listed on the drug formulary. Access may be requested through Health Canada’s Special Access Programme only for the named patient and the patient may additionally need to apply to the provincial compassionate or exceptional access programme. These processes are inefficient, costly and time-consuming, for both the patient and the health care system. Designed for urgent, one-off situations, they are not viable mechanisms to ensure Canadians with rare disorders have sustainable access to appropriate therapy in a monitored and timely fashion.

As importantly, getting drug reimbursement and access right for rare diseases will have big payoffs as we move into the era of personalized medicine for more common conditions. Our knowledge about genetic bases for individual differences in large population disorders such as breast or prostate cancer, diabetes, circulatory conditions, and psychiatric disorders such as schizophrenia will result in identifi cation of subgroups defi ned by genotype. What we are learning and pioneering in drug development and access for rare diseases will someday benefit all patients.

Over the past few years, Canada has made remarkable progress in the care and treatment of patients with rare diseases but, frankly, we still lag far behind most developed countries. The implementation of the Orphan Drug Regulatory Framework will have significant impact, but it still lacks some of the provisions that will ensure pharmaceutical and biotech companies bring research and clinical trials to Canada, including a period of market exclusivity for innovative drugs and tax incentives. Moreover, in order to realize the benefits of a regulatory framework, we need to follow the lead of the 27 EU countries in bringing in a national plan for rare diseases.

The Canadian Organization for Rare Disorders (CORD) in collaboration with the Canadian Institutes for Health Research, Genome Canada, BIOTECanada, and Rx&D are developing the framework for a Canadian Strategy for Rare Diseases that will address the definition of rare diseases, research, diagnosis, care and support, and access to treatment. This strategy allows us to consider rare disorders in the context of a broader public health approach and builds upon existing infrastructure, expertise, and programmes. Although Canada has come late to the game, there is an opportunity for us build on existing international experience and knowledge.

Canadians with rare diseases have a right to the same health care as those with more common conditions. A Canadian strategy for rare diseases will help ensure the most effective and cost-effective way to providing that care.

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