How do Cancer Drugs get to Market?

Our goal is to make sure that women with metastatic breast cancer have access to drugs that can give them more time and improve quality of life. Put another, simpler, way: we want patients to have access to the right drug at the right time — and for this to happen regardless of where they live. But one of the challenges to this goal is that Canada’s drug approval process is long and complicated. Here’s how it works:

Step One: Development

This can take years and significant financial investments. In oncology, it starts with the discovery of a chemical compound with anti-cancer properties. As doctors and researchers have embraced precision medicine, one new way to find these compounds is to put them up against different cancer-causing genetic mutations to see if they’ll have an effect. But even when a compound shows promise, it takes a long time to turn it into a drug. And once that happens, there are several rounds of clinical trials to demonstrate safety and effectiveness:

  • Preclinical trials happen in the lab, not on people. This is when researchers try to determine effectiveness; if a compound shows promise, it moves onto the next stage.
  • Phase I happens in a small group of patients. This stage is about safety, first and foremost. Researchers are trying to determine the right way to administer a drug (tablet vs. injection), how much patients should take at each dose, how often they should take it and what side effects they might experience.
  • Phase II also happens in a small group, and tests whether a drug works against a specific type of cancer when following the dosage and schedule Phase I determined was safe.
  • Phase III compares a new drug to the best existing treatment to see if it is more, less or similarly effective. This is usually a very large study with lots of people enrolled. It considers quality of life, survival and side effects.
  • Phase IV is about looking for long-term effects—if a drug makes it past Phase III, researchers continue following the participants from that trial to look for risks and benefits.

Yale Cancer Center in New Haven, Connecticut produces a weekly public radio show that answers audience questions about cancer treatment, research and prevention, and in 2008, they tackled this exact question with Dr. Mario Sznol, Professor of Medicine in Medical Oncology at Yale University.

“Just taking a chemical entity and making a drug, in other words putting it into a vial and making sure that it is safe, being able to manufacture the drug, and then testing it in the clinic, can take years and years,” he said.

“For example, it can take a year just to find out the right dose and schedule for a drug, and it can take three to five years to do the final clinical trial that determines whether a drug works or not in patients. If you are thinking about the initial discovery of the drug in the test tube to final data in a randomized clinical trial that shows that it works, that could be a process of seven to 10 years.”

Step Two: Approval

Once a drug has made it through Phase III clinical trials, the pharmaceutical company submits it to Health Canada for approval. The application includes all the information that exists about the drug: the results of preclinical and clinical trials, how it is produced, information about side effects, even what the packaging and labelling looks like. Health Canada has one year to review each application. Then, if Health Canada believes that “the benefits outweigh the risks and the risks can be mitigated” the drug is issued a Notice of Compliance (NOC), as well as a Drug Identification Number (DIN).

But when Health Canada signs off on a drug, it doesn’t mean that therapy will immediately become publically available to patients — that is actually decided and administered by each province.

Step Three: Cost-Effectiveness

Before the provinces and territories (except Quebec) decide whether the drug is eligible for public funding it waits for a recommendation from the Canadian Agency for Drug and Health Technology (CADTH), which evaluates the clinical, economic and patient evidence for oncology drugs. (This was formally known as the pan-Canadian Drug Review (pCODR), but now CADTH reviews all drugs under one system.)

CADTH doesn’t just look at the information the manufacturer has submitted. It also solicits input from the patient groups and health care professionals representing those who would be using this drug, and looks at how it stacks ups in terms of effectiveness, cost, therapeutic benefit and implementation compared to existing drugs.

The timeline for performing a CADTH review varies, but on average, it takes five to eight months. Once the review is done, the organization makes a formal recommendation regarding funding, which provinces take into account when making their final decision on whether to fund a drug.

Step Four: Price Negotiations

If CADTH makes a positive recommendation, the pan-Canadian Pharmaceutical Alliance (pCPA), which includes representatives from each province, territory and the federal government, steps in to decide whether to negotiate price with the manufacturer. The pCPA was formed in 2010 specifically to get better drug prices for Canadians; before, provinces and territories negotiated separately and since larger jurisdictions had more negotiating power, they often paid less for drugs than smaller jurisdictions.

When a new drug comes down the pipeline, each provincial rep looks at the CADTH recommendation and decides whether they want to negotiate as a group for the drug. If so, one province or territory takes the lead, going back and forth with the manufacturer on pricing. Once they reach an agreement, they sign a letter of intent, which they share with the rest of the provinces and territories in that negotiation to use when they individually fund the drug locally.

Step Five: Available in Market (Eventually)

Once pCPA reaches an agreement with a drug manufacturer, the provinces and territories are supposed to list the drug on their provincial formulary. But, there’s no timeline on when this actually happens, and no accountability for delays. And each province takes a different approach — in Ontario, for example, the Ontario Drug Benefit Program Executive Officer makes the final decision. This means, in practice, patients often still have to wait to access these much-needed medications.

The Next Step: CDIAC

The Cancer Drug Implementation Advisory Committee (CDIAC), which reports into the Canadian Association of Provincial Cancer Agencies (CAPCA). CAPCA is a national government agency that works to progress the quality of cancer care across Canada by improving access to information, such as cancer research and surveillance data. In the context of oncology drugs, this means CDIAC helps decide which medications should be considered for public funding. Patients groups are ostensibly part of this decision-making process, but  we’re not sure how that will look in practice. In fact, there’s still a lot of confusion about what CAPCA does, what parts of the process it touches, what its decisions are based on, and how it will affect timelines.

The government is changing who decides whether patients can access cancer drugs and cancer patients are losing their voice in the process, feeling left in the dark. There are too many uncertainties for women with metastatic breast cancer and this should not be one of them.

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