The Exceptional Patient Measure: A Safety Valve Allowing Quebecers in Need to Access Innovative Drugs

Economic Note calling for the maintaining of this mechanism that serves to limit the health risks of the very slow and bureaucratic drug approval process

Related Content

Potential change to Quebec’s drug insurance plan a worry (Montreal Gazette, December 19, 2024)

This Economic Note was prepared by Emmanuelle B. Faubert, Economist at the MEI. The MEI’s Health Policy Series aims to examine the extent to which freedom of choice and entrepreneurship lead to improvements in the quality and efficiency of health care services for all patients.

Although Quebecers generally do not have to pay directly to be treated in the public health care system, that does not mean the system is free. Indeed, the population funds it through taxes. In 2024-2025, health care will represent 41.9% of the province’s portfolio spending.(1) Moreover, Quebecers also end up paying with their time: between long ER waits, difficulties accessing specialized care, and approval delays for new drugs, they are regularly faced with one form or another of healthcare rationing.

In this context, in order to limit the harmful effects of long delays in obtaining access to innovative drugs, which can sometimes extend several years beyond their entry onto the global market,(2) the Quebec government set up an “exceptional patient” mechanism in 2005.(3) However, recent legislative changes could threaten the existence of this crucial measure for the well-being of many patients.

Mitigating the Rationing of Pharmaceutical Innovations

The exceptional patient measure allows patients with no other options, upon a doctor’s recommendation, to submit a request for access to drugs approved by Health Canada that do not appear on the list of the Régie de l’assurance maladie du Québec (RAMQ), and to which Quebec patients therefore do not normally have access.

This measure applies to two types of drugs:

• those awaiting approval by the Institut national d’excellence en santé et en services sociaux (INESSS), the provincial body responsible for adding drugs to the list of medications covered by Quebec’s public plan;
• those not added to the RAMQ list following an unfavourable assessment by the INESSS.(4)

For it to be possible to offer a drug to Quebec patients in a health facility or pharmacy, it must first be approved by Health Canada, which assesses its therapeutic efficacy and safety.(5) It must then be approved by the INESSS,(6) which assesses the “therapeutic value” of the drug based on the available evidence. This consists of assessing its innovative character compared to existing drugs, followed by a cost-benefit analysis.(7)

This analysis conducted for the RAMQ, and so for the entire population, therefore supplants the doctor’s analysis, even though the latter is better placed to judge the individual benefits of the drug for a specific patient.(8) The Department of Health and Social Services will base itself on the recommendations of the INESSS to determine if a drug will or will not be covered by the public plan.(9)

The exceptional patient measure allows patients with no other options to submit a request for access to drugs approved by Health Canada.

It is only after these steps have been completed that a drug can be administered in Quebec hospitals and clinics, and that health insurance will start to cover its costs.(10) Quebec is not the only province whose drug approval system works this way. On average, the delay between approval of a drug by Health Canada and coverage of its cost by provincial health authorities is nearly two years (study results vary between 537 and 678 days on average), which represents a form of rationing of access to new drugs.(11)

But what happens when the drugs already covered by the RAMQ are unsuitable for a patient’s health problem, and an unlisted drug exists that could do the job? The rationale behind the exceptional patient measure is precisely to address this kind of situation, and many patients benefit from it. In 2022, 35,836 people made use of this mechanism,(12) with a total of 341,972 such requests accepted(13) over a period of 10 years (see Figure 1).

For a patient to be able to benefit from the exceptional patient mechanism, several conditions need to be met. The attending physician must present a file explaining why the proposed drug is appropriate and no other pharmacological treatment already available on the list of medications is an option.(14) The file must then be approved by the RAMQ for patients covered by the public plan. For those covered by private insurance, the file is examined by the insurer, on the basis of similar criteria and consultations with doctors and pharmacists.(15)

Approval of the file allows the patient to access the drug in question for a year. After this period, if the drug still has not been added to the RAMQ list of medication and is still considered essential by the attending physician, the exceptional patient request must be renewed to continue to apply. For patients suffering from rare diseases, for instance, the exceptional patient mechanism allows them to have access to pharmaceutical innovations when waiting would have a considerably detrimental effect on their health.

The delay between approval of a drug by Health Canada and coverage of its cost by provincial health authorities is nearly two years.

Unfortunately for the tens of thousands of patients a year who, thanks to this mechanism, are able to benefit from treatments normally not covered by the RAMQ, their access could soon be at risk.

Exceptional Patient Benefits Threatened

In 2023, the National Assembly adopted Bill 15, An Act to make the health and social services system more effective.(16) This bill modifies the specific medical necessity measure, similar in principle to the exceptional patient measure but applicable to hospitalized patients specifically. The law stipulates that even if the attending physician can demonstrate a need to access a drug that is not on the RAMQ list, the hospital pharmacology committee responsible for assessing the request will not be able to authorize its coverage if the INESSS has negatively assessed its therapeutic value, unless delaying the taking of the said medication would lead to a rapid and irreversible deterioration of the patient’s condition, including in emergency cases.(17)

The bill only affects hospitalized patients, and does not modify the exceptional patient measure. However, it is possible that these changes will be extended to exceptional patients in 2025 (see Figure 2). This approach would eliminate this safety valve, under the pretext of reducing the RAMQ’s expenditures.

Recall that in September 2024, the Quebec Premier asked his ministers to “tighten their belts” and to respect the budgets they were allocated.(18) In the November 2024 economic update, it was revealed that spending on health and social services had exceeded the budget set in March 2024 by $3.4 billion.(19) While there has been no official public announcement on this topic, groups like the Alliance des patients pour la santé, as well as RAREi, the Canadian forum for rare disease innovators, fear that such changes will be put in place in the near future.(20)

When a drug is assessed to determine if it will be covered by the RAMQ, the INESSS asks two questions: will this new drug provide benefits to the population that other drugs already covered do not provide, and is this treatment worth the cost?

Should the exceptional patient measure become conditional on approval by the INESSS, it would lose a large part of its rationale.

While this approach aims to determine if a new treatment should be covered by the province’s public insurance plan, it cannot determine what is the best treatment for each and every patient. Doctors, however, are in a position to assess this, and the exceptional patient measure serves to limit the health risks of the bureaucratic INESSS approach.

Should the exceptional patient measure become conditional on approval by the INESSS, it would lose a large part of its rationale, depriving patients of a safety valve that allows them to access needed drugs on an exceptional basis.

Finally, the exceptional patient mechanism is not good just for patients, but also for the rest of the healthcare system. Obtaining quick access to innovative treatments improves patients’ prognosis, and so helps keep their health from deteriorating and prevents complications that could result from the RAMQ’s long delays in treating them. It can also help avoid other, potentially more expensive treatments in the health system, such as surgery and hospitalization.

Conclusion

In the current context of the rationing of new drugs, it is essential to maintain the exceptional patient mechanism safety valve. Policymakers need to be fully aware of the importance of this measure, both for patients and for the Quebec healthcare system as a whole, notably in terms of costs avoided thanks to reduced health risks and improved disease treatment.

It would also be useful to reform the very slow and bureaucratic drug approval process, which is largely responsible for the need to resort to measures like the exceptional patient mechanism. By accelerating the addition of drugs onto the RAMQ list (including the INESSS approval process and negotiations with manufacturers), many patients would not need to use the exceptional patient mechanism, as the drugs they need would already be covered.(21)

The exceptional patient mechanism is an essential measure that allows tens of thousands of Quebecers to access drugs not on the RAMQ medication list. Access to this safety valve must be preserved to mitigate the rationing of pharmaceutical innovations that characterizes the Quebec healthcare system.