Health Canada's Orphan Drug Pilot Project Seeks Patient Involvement

Drugmakers Hoffman-La Roche and Hyperion Therapeutics are taking part in Health Canada’s new orphan drugs pilot project to better understand how patient input may be gathered and incorporated into the drug submission review process.

Both companies say that bringing the patient perspective into reviews of drugs for rare diseases is crucial, as patients increasingly are playing an important role in general drug approvals and health technology assessments. The pilot will tap Hoffman-La Roche’s Gazyva (obinutuzumab) for chronic lymphocytic leukemia and Hyperion’s Ravicti (glycerol phenylbutyrate) for urea cycle disorders.

Patients will be asked how their diseases affect the management of day-to-day life, what treatments are available, which ones are most important to them and their tolerance for risks of any new treatments, the agency said this week.

The pilot is expected to last a year and additional companies may join as time progresses, agency spokesman Gary Holub said.

The focus on patient input will add context to the regulatory review process, Canadian Organization for Rare Disorders (CORD) spokesman Durhane Wong-Rieger said. Most regulators have little familiarity with the diseases in question and little experience reviewing drugs to treat them.

Canada Minister of Health Rona Ambrose said regulators are committed to making the experiences and perspectives of patients an important addition to the scientific assessment of drugs for rare diseases.

The pilot is part of a larger initiative to revamp Canada’s orphan drug review process. Only about 60 percent of orphan drugs make it to regulatory approval in Canada, and sometimes up to six years later than in the U.S., says CORD.

Development of a new framework to address orphan drug reviews began in 2012 and was designed to encourage Canadian and international researchers and regulators to share information. But official approval and launch of the framework has been delayed many times, CORD said.

Once the framework goes online, it would let drug companies apply to bring their treatments to Canada at the same time as the USA and Europe, and create an opportunity for Canadians to participate in clinical trials at the same time as they occur worldwide. — Lena Freund

Originally appeared in Drug Industry Daily, the pharmaceutical industry’s number one source for regulatory news and information. Click here for more information.