In May 2017, Health Canada approved Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis (PBC).

More specifically, the drug was approved in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

PBC is a rare, autoimmune liver disease that puts patients at risk for life-threatening complications, mostly older women. For the past 20 years, UDCA was a patient’s only treatment option and in many patients UDCA is not effective or only partially effective. Hence, the need for more treatment options.

So the news that Health Canada had approved the drug was great news to the PBC community but they were also aware that getting Health Canada to approve the drug was only the first hurdle to getting the drug into the hands of the patients.

In July 2017,  the Canadian Agency for Drugs and Technologies in Health (CADTH)  recommended that Ocaliva (obeticholic acid) be made available but they added the following conditions to the recommendations:

  • Patients should be under the care of a specialist with experience in the diagnosis and management of PBC.
  • Reduction in price of at least 60%.

So, now it is up to each provincial drug plan determine if they will cover the drug (and at what price).

The CADTH recommendations were based on clinical trial data showing the drug to be effective as well as patient input from the Canadian Liver Foundation and the Canadian PBC Society.

Recently, we talked with Gail Wright about the Canadian PBC Society’s efforts to let all the regulatory bodies across the country be aware of patients’ perspective on having a second treatment option for PBC.

“Even before it was approved by Health Canada, we were out talking to as many people as we could get appointments in the various ministries of Health,” noted Wright.  And what PBC Canada focused on in those discussions was not the drug but the burden that PBC places on the patient and their family and that more treatment options are needed. “We didn’t want to talk about a particular therapy because we are not qualified to talk about the clinical trial. The doctors and pharmaceutical company can do that. What patient groups should want to focus on is expressing the unmet needs of patients and the impact of not meeting those needs. And that is what we did.”

Wright added that the only real discussion about the drug under review is that they told the various ministries of health that about 40% of PBC patients do not respond to current therapy (UDCA) and without a proper response to therapy, the end results is the need for a liver transplant or death.

Wright noted that their more informal approach to presenting their case to the various ministries is representative of the patient population they serve – patients with a progressive, chronic condition that is in need of a better treatment option. “We don’t have that urgency like so many of our other rare disease partners where a person will die if they do not get a drug and they are lobbying all the time. Our meetings are a bit different. It’s very disease dependent.”

As to how to contact those people at the various health ministries, Wright said, it is not that hard and this week, CADTH made it a bit easier with their list of the contact information for each province.

Wright said, “You can send them an email and ask for an appointment and once you do that they’re just regular nice people doing a job. If you get half an hour with them, then you can explain the disease, what treatments are available, what the unmet need is, and ask them to keep the patients in mind when making a decision. Then six months later, maybe send them an email as a reminder. It’s very informal; you don’t need any formal presentations or text or slides. These people already get the science from Health Canada, the doctors, and the companies. They don’t need that from the patient. You want to keep it real.”

Wright also noted that each presentation will be a bit different based on the province. “I was in British Columbia and I told them that First Nations coastal people have a high prevalence of PBC so that gives them something to relate to, said Wright, adding, “and when I was in Fredericton I told them that the Maritimes have the highest proportion of people with PBC and when I sent them a thank you email, I sent them the study I was referring to.”

Look for part 2 of our interview with Ms. Wright next week talking about developing a strategic plan for your patient advocacy group.

For more information about PBC and PBC Canada, visit  pbc-society.ca