About one in 12 Canadians has a rare disease or disorder, two-thirds of whom are children, according to the Canadian Organization for Rare Disorders (CORD).

Access to medication and services for these patients can be challenging and costly.

“About half of the drugs available for rare disease in Europe and the U.S. are not available in Canada, and more than half of those that get approved in Canada are not reimbursed by public health plans,” says Durhane Wong-Rieger, President and Chief Executive Officer of CORD.

To ease this burden, pharmaceutical companies have developed Patient Support Programs (PSPs). “PSPs help patients navigate access to reimbursement from both private and or public health insurers and ensure that patients can get access to the appropriate therapy,  ” says Sandra Anderson, Vice President, Consulting and Business Development at Innomar Strategies. PSPs can also help patients and their families through logistical care support, such as coordinating out-of-hospital treatments within private clinics, providing access to specialty pharmacies,  and arranging appropriate patient and family education and counselling.

Health outcome studies – Real-world data to guide decision making

Rare disease drug treatments are often higher priced and treat diseases that are difficult to diagnose, with only a few specialty physicians in Canada who understand the disease. They also treat small unique populations, which makes it challenging to measure the efficacy of these therapies. Payers may want to seek out more information, but there is a lack of real-world data available.

“Canadian payers are looking to pharmaceutical companies to present real-world clinical data to demonstrate the effectiveness of their products and to ensure the right patient is on the right product at the right time,” says Anderson. Real world data is important as it illustrates how these therapies improve health outcomes which supports the payers’ investment for these valuable medications. With access to meaningful data and analytics to highlight the value of these therapies, through a PSP for instance, many are hopeful that access to such specialty treatments will improve.

Patient engagement a key component

As Sandra Anderson explains, “PSP services are meant to ease the patient’s health journey and to ensure they can access their important therapies at a time when they need it the most.” Getting the most from PSPs involves ongoing patient engagement. “We want to engage with patients from the very beginning in identifying what’s important to them so that the clinical trials reflect their concerns all the way through,  as well as contribute to good science,” says Wong-Rieger. “In addition, this engagement helps patients understand not only the strengths and limitations of the drug but also what they can do to fully benefit from it.”