Speed up access to cutting-edge cancer therapies

Patients don’t have luxury of time, Robin Markowitz says.

Strides in cancer research have increased survival rates and provided a better quality of life for patients under treatment. But getting the best treatment as soon as possible remains a matter of life and death for many Canadians.

Speed is of the essence. Patients live with the hope that a new treatment will be available by the time they need it.

Health Canada has recognized the need to keep up with the pace of innovations which have allowed new cancer drugs to be brought to market sooner, and has worked to speed up approval of promising new cancer therapies.

But that doesn’t mean these approved drugs are funded more quickly or at all. Few patients consider that the treatment recommended by their doctor, and often available in other countries, may not be approved for funding, essentially meaning that it will be inaccessible.

Since Canadian health care is under the jurisdiction of provinces and territories, it’s up to them to decide if a drug will be eligible for public reimbursement. For cancer drugs, provinces and territories broadly follow the recommendation of the pan-Canadian Oncology Drug Review (pCODR) when making final reimbursement and coverage decisions. A negative recommendation is tantamount to a stop sign in terms of patient access.

The pCODR typically relies on randomized control trials (RCT) to make its decisions. That’s the gold standard.

However, RCTs are not always feasible, appropriate or ethical for the evaluation of new therapeutic interventions. Non-comparative data is increasingly being used. The criteria pCODR uses for RCT data has not kept pace with the breakthroughs in cancer research. Their system needs to be updated.

For many new targeted therapies, the patient population is too small to conduct this type of trial. In other instances, the time required to conduct an RCT trial equates to years — precious years that cancer patients do not have. Once a drug therapy is deemed safe and efficacious, it may be fast-tracked, enabling patients to quickly have access. These fast-track drugs are often submitted to the regulatory body with non-comparative data.

But increasingly, pCODR has been rejecting these funding submissions. In a policy paper that Lymphoma Canada published earlier this year, we found that, beginning in 2015, the number of pCODR submissions supported by evidence from non-comparative data increased significantly. Yet the rate of negative recommendations also increased. Between January 2015 and the end of December 2017, 63 per cent of submissions received negative recommendations from pCODR. In the three prior years, the negative recommendation rate was 25 per cent.

This month, health researchers and health policy decision-makers who have the power to speed up access to new treatments and save the lives of Canadians are meeting in Halifax for a conference hosted by the Canadian Agency for Drugs and Technology in Health (CADTH) — the agency that’s home to pCODR. Lymphoma Canada and 12 other signatories across the cancer spectrum are calling on attendees to advocate for reforming pCODR’s approach.

There are two key ways pCODR can improve the process and the health of Canadians. Firstly, it can issue positive recommendations by providing for temporary funding while the drug manufacturer addresses the perceived uncertainty of the clinical value. Under such circumstances, procedures regarding evidence collection would need to be established.

Secondly, in addition to acquiring more robust trial data, real-world evidence can be collected to reduce the uncertainty. Collaboration among relevant stakeholders could be leveraged to ensure pCODR’s recommendations remain sound over time. Costs can be managed through innovative risk-sharing and other price-control and reduction agreements.

While we acknowledge that there are limitations to estimating the value of a new treatment using non-comparative clinical studies, this setting is increasingly becoming the final stage of clinical evaluation for new cancer drugs, especially for rare cancers, for tumours with distinct molecular profiles or where no standard of care exists.

Increased requirements for evidence of a drug ’s clinical and cost effectiveness prior to use may seem reasonable to reduce uncertainty.

However, patients cannot wait. Canadian governments and their policy-makers should not deny or delay funding of a drug when there is sufficient data available to discern its efficacy and safety in a vulnerable patient population.