Who will speak for the unmet needs of the sick?

Private sector support for patient groups is an ethical necessity in the absence of public funding.

Some academic authors have criticized pharmaceutical companies for funding patient advocacy groups because they see it as a conflict of interest. Media commentators also tend to take a negative view of industry funding for such non-profit organizations. Although it cannot be proved that funding from pharmaceutical companies influences the policy positions of patient groups regarding whether drugs should receive insurance coverage, the implication, with varying levels of subtleness, is that this is the case.

Authors of these articles and media reports are unlikely to have a child or partner suffering from a major debilitating or life-threatening condition for which the only effective therapy is an innovative costly drug. Otherwise, they might be less judgemental and investigate whether the private health insurance to which they and their employers contribute provides coverage for the required medicine. Since most universities and media corporations contribute to a comprehensive private insurance plan, they might be lucky. If not, they could look to their provincial public drug plan, but the likelihood of gaining reimbursed coverage would be substantially lower, because Canada has a two-tier drug insurance system.

Individuals with unmet needs often turn to a patient support group or create one. These groups are not new. The Infantile Paralysis Fellowship (now the British Polio Fellowship) was founded 80 years ago when poliomyelitis infection was relatively common. The Fellowship’s objectives included connecting victims to develop their interests and abilities, finding training for occupations so that members could support themselves, alleviating sufferers’ loneliness, linking those requiring advice and assistance with sources of help, arousing the social conscience on problems encountered by the paralyzed, and to influence policy and legislation.

Patient groups continue to have similar objectives, providing education, peer-to-peer support and encouragement, even hand-holding, during difficult times. In the present Canadian health care environment, these groups are essential and have proliferated. Support groups undertake advocacy activities to ensure better access to care and treatment that their members need but the health care system is not adequately providing. Patients with rare disorders frequently have a greater requirement for support due to their small numbers and isolation.

All patient groups need funding to be able to provide the support their members require. Only large groups representing common diseases that resonate with the public are likely to be sufficiently successful at awareness and donation campaigns to be able to sustain their activities from these sources alone. Most patient groups must look elsewhere for funding. Governments will not provide funding, particularly if it is likely to be used to lobby for access to expensive care or treatments.

Patient groups representing individuals with rare disorders suffer from a distinct disadvantage in that the public is usually unaware of the disorder and, unless directly confronted by individuals suffering from it, generally does not care. It is, therefore, no surprise if a patient advocacy group connects with the manufacturer of the drug that it wants governments to cover.

No comprehensive national strategy exists in Canada for making costly drugs available to patients with unmet needs in an affordable manner through private or public insurance plans. Just the opposite, numerous barriers exist to restrict access to expensive medicines, especially drugs for rare disorders. Only 73% of these drugs approved in the United States or Europe came to Canada in 2002-2016 and 25% of those that did took longer than 2.5 years to be approved. After regulatory approval, patients can wait years before costly drugs are adequately covered by public drug plans.

Five major barriers make Canada a less attractive jurisdiction in which to market drugs:

1. Canada is a second-tier country behind the United States and the European Union when pharmaceutical manufacturers seek regulatory approval for their products, resulting in a lag in access to innovation.
2. Current health technology assessment methods do not work for costly medicines, especially those for rare disorders.
3. The pan-Canadian Pharmaceutical Alliance can delay access to new medicines by being slow or inflexible in price negotiations, or just refusing to negotiate.
4. The Patented Medicine Prices Review Board can also impact access. The federal government has proposed changes that would not only require pharmaceutical manufacturers to reduce prices based on an international comparison with countries with lower drug prices but also include health technology assessment using a low cost-effectiveness threshold value and comparisons with an arbitrary low annual sales ceiling.
5. Individual provincial and federal government drug plans can ignore a positive health technology assessment reimbursement recommendation and/or a pan-Canadian Pharmaceutical Alliance price agreement and not cover a drug.

The 2019 federal Budget proposed a new Canada Drug Agency to evaluate drug effectiveness and negotiate drug prices for all drug plans, private and public. This was based on the interim report from the Advisory Council on the Implementation of National Pharmacare, which proposed a national drug agency that would amalgamate some functions of Health Canada, the health technology assessment agencies, the pan-Canadian Pharmaceutical Alliance and perhaps the Patented Medicine Prices Review Board. However, this will not eliminate barriers to access. In fact, a super-agency has the potential to be a huge barrier – bigger is not always better. Any additional deterrents to manufacturers launching new costly drugs are highly likely to lead to Canada falling from being a second-tier to a third-tier country. This would lengthen wait times for patient access to medicines.

Patients want precision medicines and welcome news of a disease being moved from untreatable to treatable or from treatable to curable. As the number of drugs targeting disorders previously untreatable increases, an urgent need continues for the development of processes to make these medicines available to patients in a non-discriminatory and transparent way. Without policies to provide fair, just and equitable access to new costly drugs and where public drug plans use every reason not to fund them, no one should be surprised that patient advocacy has become a major endeavour.

The connection between patient group and manufacturer is, of course, symbiotic; manufacturers want to sell their medicines and patients want access to them. However, the relationship should be a collaborative one with potential conflicts of interest being managed by strictly enforced and publicly available policies. For instance, Innovative Medicines Canada has a code of ethical practices requiring patient support programs to have a primary objective of improving health outcomes, not simply to influence reimbursement recommendations.

Patient groups should also have their own publicly accessible codes of ethical practice with regard to accepting money from pharmaceutical companies. The Best Medicines Coalition of 27 national and regional patient associations has a code of conduct regarding funding to ensure that relationships are ethical and transparent. Nevertheless, many academics do not believe that patients and manufacturers can collaborate ethically to promote public or private insurance coverage for expensive drugs, few of which, in their opinion, are worth their cost.

We should not forget that academics can also have conflicts of interest. Professors in Canada are employed by universities that are mainly dependent upon governments for their budgets including research. Academics may also undertake advisory or investigative activities on behalf of a government. If they do not wish to incur the displeasure of their ultimate employer (the government) or their government research funder, they will be careful about what they study and how they report any results that are contrary to government interests, because they do not want to bite the hand that feeds them. Commentators at government-funded media organizations can also be conflicted.

Instead of censuring patient support groups representing individuals with unmet or poorly met health needs, academic and media critics should be asking:

• In a wealthy country like Canada, why are advocacy groups a necessity in trying to obtain financial coverage for drugs that could significantly reduce the suffering or extend the lives of patients with diseases for which no real alternative treatments exist?
• When will governments introduce fair, accountable and transparent processes to make new drugs for rare disorders available in public drug plans without the necessity for patient support group advocacy?
• When the federal government has a mantra of “affordability, accessibility and appropriate use of prescription drugs,” why does cost-containment always take priority?
• Why do billions of taxpayers’ dollars continue to be squandered by the federal and provincial governments supporting activities that have poor financial oversight leading to wasteful spending, while patients in need of life-saving and life-changing drugs go untreated?

In the spirit of disclosure, no funding was received for this article. Our potential conflicts of interest over the past three years are: NSBR has received funding for consulting, research and publication from pharmaceutical companies and their associations, the Fraser Institute, the Canadian Health Policy Institute, Ward Health, and Canadian PKU and Allied Disorders Inc. JA has received funding for consulting, publication, research or conference travel and registration from Aptatek BioSciences, Best Medicines Coalition, Better Pharmacare Coalition of BC, BioMarin Pharmaceutical, Canadian Agency for Drugs and Technology in Health, Canadian PKU and Allied Disorders Inc, Global Association for PKU, and the Ontario College of Physiotherapists (on behalf of several regulatory colleges).

Nigel Rawson (Ph.D., M.Sc.)

Nigel Rawson is a pharmacoepidemiologist, pharmaceutical policy researcher, and President of Eastlake Research Group in Oakville, Ontario. Educated in the United Kingdom, he holds an M.Sc. in statistics and a Ph.D. in pharmacoepidemiology. Dr. Rawson has performed epidemiologic studies of the use of drugs and their outcomes for over 35 years and published more than 120 book chapters and articles in peer-reviewed journals. He is also the author of the monograph “Drug Safety: Problems, Pitfalls and Solutions in Identifying and Evaluating Risk” published by Friesenpress. He held academic research positions in the United Kingdom until the end of 1989 and subsequently held professorships at the University of Saskatchewan and Memorial University of Newfoundland in Canada. His research activities focused on population-based studies of the use and safety of drugs using administrative healthcare utilization data and the evaluation of issues impacting access to new drugs. Dr. Rawson has also been a senior researcher in an independent research centre in one of the United States’ largest health insurers, where he collaborated with the Food and Drug Administration on drug safety studies, and GlaxoSmithKline’s only epidemiologist in Canada providing advice and analysis for the company’s current and developing medicines and vaccines. Dr. Rawson established Eastlake Research Group in 2012 with a mission to create data-driven responses to pharmaceutical and health policy issues.

John Adams

Mr. Adams is Board Chair of the Best Medicines Coalition, Co-Chair of the Disability Tax Fairness Alliance, Co-founder, President & CEO of Canadian PKU and Allied Disorders non-profit and Co-Founder and Trustee of the Global Association for PKU. He is a former Chair of the Canadian Organization for Rare Disorders. Mr. Adams is a graduate of York University; worked as a reporter for The Globe and Mail; chief of staff to a Cabinet Minister in Ontario; elected three times to Toronto City Council, serving as Budget Chief and Acting Mayor; and served as a trustee of Canada’s largest hospital, the University Health Network.