The approval process for new drugs

When you’re suffering from a fatal (orphan) disease for which there is no cure, such as Duchenne muscular dystrophy, each year, month, day, hour and second that separates you from a potential treatment is VITAL.

Nowadays, in an era when science and technology are advancing at the speed of light, new forms of treatment for Duchenne muscular dystrophy, about to be approved to represent real hope. However, these new forms of treatment must go through an approval process that takes between 2 and four years before becoming available to patients.

When rare diseases are involved, patients should be involved in this process so that it occurs as quickly as possible and that the medication is reimbursed by all provinces across Canada.

Here is a brief overview of the process.

PHASE 1 – OBTAINING THE PHARMACEUTICAL COMPANY MARKETING NUMBER

  • TIMELINE: 1 – 2 YEARS

  • OPTION FOR THE PATIENT:

The patient can access treatment before the end of the process through a special access program, but the treatment is not reimbursed.
See:
http://www.hc-sc.gc.ca/dhp-mps/acces/drugs-drogues/index-eng.php

In Canada, Health Canada is responsible for authorizing the use of a drug. What follows is the process that pharmaceutical companies sponsoring the development of a new drug must adhere to:

Step 1> The developer of the drug submits an application to Health Canada in order to obtain permission to conduct clinical trials in Canada.

Step 2> If the review of clinical trials shows that the drug has a potential therapeutic value that surpasses the risks associated with taking the drug, the pharmaceutical company may choose to submit a New Drug Submission to the HPFB (Health Products and Food Branch) of Health Canada.

Step 3> Submission of a “New drug submission” to the Branch, who performs a thorough review of the information submitted.

Step 4> If findings reveal that the benefits outweigh the risks, the Branch issues a Notice of Compliance and a Drug Identification Number (DIN) which allows the company to market the drug in Canada.

 

PHASE 2 – PROCESS LEADING TO THE REIMBURSEMENT OF TREATMENT

(After obtaining the “DIN” marketing number)

Let’s now look at how things work in the provinces and territories. First, it’s worth mentioning that each province and territory in Canada has a drug reimbursement system of its own.

Process for QUÉBEC

TIMELINE: ONE YEAR (approximately)

OPTION FOR PATIENTS:

One can make an exception request that provides for reimbursement of treatment on an individual basis.

In Québec, this process is governed by the INESSS (National Institute of excellence in health and social services).

Step 5> The pharmaceutical company files an application with the Institute (INESSS). Applications for registration of drugs are analyzed by INESSS professionals and by the Standing Scientific Committee. This application can be filed only after obtaining the (DIN).

Step 6> INESSS publishes a Notice to the Minister, that contains recommendations relating to the products evaluated by its department, and explains the reasons for product acceptance or rejection, in accordance with governing legislation.

Step 7> The RAMQ updates the list of insured drugs. The last update took place on February 8, 2016.

END OF PROCESS: TREATMENT NOW AVAILABLE TO PATIENTS

 

PHASE 2 – PAN-CANADIAN PROCESS

In the other provinces and territories, the process is governed by the Common Drug Review Program (CDR) and the Pan-Canadian Drug Purchasing Alliance – PCDPA

TIMELINE: 1 YEAR + … (slightly longer than in Québec)

Step 5> Submission to the CDR. Submissions are necessary for public reimbursement in Canada with any new drug. The CDR submissions affect all provincial reimbursement programs (except Quebec).

Step 6> The CDR conducts a rigorous and objective evaluation of the clinical and economic evidence, and makes recommendations to federal, provincial and territorial government public drug insurance plans, except Québec which administers its own review process (INESSS).

Step 7> After the drug review, the CDEC (Canadian Drug Expert Committee) may recommend the inclusion of a drug in the participating drug plans.

Step 8> The participating drug plans then make a final decision on the refund policy for this drug: each province and territory (except Quebec) chooses to reimburse patients or not for the drug.

END OF PROCESS