This article is the second part of an 8-part series from the authors. The series, Waiting for new drugs for rare disorders in Canada, is an in-depth look at the disadvantages faced by Canadians with rare disorders in accessing needed, innovative drugs.
By Nigel Rawson and John Adams, July 19, 2023
The first article in our series focused on the lack of incentives to encourage developers to seek marketing approval for drugs for rare disorders in Canada. We now consider the second step in the process of getting a new drug to Canadians. This is the health technology assessment (HTA) performed by the Institut national d’excellence en santé et en services sociaux (INESSS) for Quebec and the Canadian Institute for Drugs and Technologies in Health (CADTH) for the rest of Canada.
HTAs try to evaluate the cost-effectiveness of new medicines – a noble concept in principle. They generally involve measuring a new drug’s cost and its health effect. Health effect is commonly estimated using something called quality-adjusted life-year (QALY), which is intended to measure the effect of the treatment on a patient’s health and wellbeing.
The metric used to assess whether a medicine is cost-effective is called the incremental cost-effectiveness ratio (ICER). ICER is the excess cost of a new drug over the cost of any existing treatment divided by the increase in QALYs achieved with the new drug over QALYs attained with existing treatment. The result is compared with a notional threshold dollar value to decide whether the medicine is cost-effective.
QALY is a generic measure of a patient’s health and wellbeing, which attempts to include both quality and quantity of life lived into a single value on a linear scale between 0 meaning death and 1 meaning full health. Several limitations exist when using QALYs and ICERs in HTAs. Three critical issues are:
- QALY is a simplistic and inadequate measure of health whereas, in reality, health is a complex, multi-faceted physical, psychological and social state. QALYs don’t capture the social value of a medicine, such as reduced caregiving needs or benefits to individuals and society in productivity from reduced absenteeism from work or school and inability to perform when at work or school.
- QALYs don’t account for severity of the disorder being treated. Sicker individuals frequently place more value on gains in health than less sick people. The value of health improvement for a person with a severe rare disorder causing much suffering and potentially premature death is particularly high.
- The threshold against which an ICER is assessed should be high for rare disorders. However, in Canada, a threshold of $50,000 per QALY is regularly applied by CADTH, regardless of whether a medicine is for a common or rare disorder. Organizations in other countries use multiple and higher thresholds. CADTH’s threshold has been in use for decades and never been adjusted for inflation, rarity or severity.
Other concerns exist about how HTAs are performed in Canada. First, they take time to complete, which delays access. CADTH’s website shows HTAs of 62 drugs for rare disorders given regulatory approval in Canada between 2015 and 2022 took an average of almost 8.5 months. For 6.5 percent of the drugs, the time required was a year or more.
Second, HTAs are commonly performed shortly after regulatory authorization when the only information usually available on a new drug’s benefits and safety comes from clinical trials. These trials are performed under carefully monitored conditions in which participants are selected because they have a narrowly-defined diagnosis and they take the medicine as prescribed, often under supervision. In real life, patients may not have precisely the same diagnosis and don’t have the same level of supervision, so they often fail to take the medicine exactly as prescribed. HTAs are, therefore, typically trying to predict what a new drug’s cost-effectiveness will be in everyday clinical practice based on highly-controlled environment of randomized trials.
Third, Canada’s HTA agencies rarely follow-up their first evaluation after a medicine has been used in medical practice for an extensive period when both clinical and real-world evidence of its use could be assessed. Canada has no process for conditional recommendations from HTA agencies for the use of new medicines while longer-term evidence of their benefits, risks and costs are collected. Some countries, like the United Kingdom, have this type of system for selected medicines, such as cancer drugs.
Last but definitely not least, INESSS and CADTH are managed and funded by the federal, provincial and territorial governments to which they report. Input from real patients is limited. Despite claims of independence, the truth is they are not independent of their funders and governors, leading to real or perceived conflicts of duties or interests.
Canada needs a new approach to the assessment of the cost-effectiveness of new drugs for rare disorders. One that allows real evaluations of benefits, risks and costs by a truly independent agency, not ones with conflicts of duty or interest providing long-lasting recommendations based on limited initial information on health benefits.
Nigel Rawson is a Senior Fellow with the Macdonald-Laurier Institute, an Affiliate Scholar with the Canadian Health Policy Institute and a Senior Fellow with the Fraser Institute. John Adams is cofounder and CEO of Canadian PKU and Allied Disorders Inc., a Senior Fellow with the Macdonald-Laurier Institute and volunteer board chair of Best Medicines Coalition.
The views expressed are the authors’ own and do not necessarily represent those of organizations with which they collaborate.