In June 2016, the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee approved the use of Ilaris (canakinumab) to treat children with systemic juvenile idiopathic arthritis (SJIA) who have not responded well to other treatments.

As with many CADTH reviews, this one came with a caveat – they stated that the cost of the drug should not exceed that of Actemra (tocilizumab) another drug used to treat SJIA but does so at a much lower cost since it is also used to treat more common condition (rheumatoid arthritis).  The logic behind the reasoning to keep the two different drugs at the same price is not necessarily clear given that canakinumab would only be prescribed if toculizumab was not effective or not tolerated by the patient. As such, the use of canakinumab would only be used for an extremely small, but extremely sick, group of children. Since that is the population canakinumab is indicated for, it is not surprising that the makers of the drug have charged more for the drug since they are targeting a smaller patient group.

Canakinumab is a monoclonal antibody that blocks certain proteins involved in the immune response and is approved to treat numerous rare auto-inflammatory syndromes, including SJIA.  Its price is set at more than $225,000 per year.

One of the provinces that is hesitant to offer the drug (unless the cost is dramatically lowered as per CADTH’s recommendation) is British Columbia (B.C.). Last fall, children with SJIA in B.C. made headlines when they were denied access to the drug even though other treatments were not proven to be effective.

That is when the non-profit group, Cassie and Friends, learned about the importance of advocating at the government level.

Cassie and Friends is a charity organization formed in 2007 and is solely dedicated to helping children with various forms of juvenile arthritis. SJIA is an extremely rare form of juvenile arthritis.

Recently, we talked with Jennifer Wilson, executive director of Cassie and Friends about their experience in talking with the B.C. Health Ministry about allowing access to drugs that other countries have approved at the current $225,000+ pricetag.

Wilson said, “There are not many options for these children. Toculizumab is one option that is given by bi-weekly infusion in the hospital. Also available is Kinerit (anakinra), which is off-label. It is given daily and can be a very painful injection.

So those are two options available to families right now. And when they are failing those options or the treatments aren’t effective or tolerable, the only other medicine really available out there is canakinumab,” added Wilson.

The challenge Wilson faced was to convince the B.C. Health Ministry that the handful of patients that are in need of canakinumab should be given access, on a case by case basis, regardless of the cost since all other treatment options had failed.

“We really focused on the fact that this is not an experimental drug nor is it for thousands of people. It is for maybe 5 or 6 children. TAnd these are exceptional cases that can and should  be reviewed on a transparent case by case basis forinvolving children who were either failing other therapy or who found it intolerable,” noted Wilson, adding, “We worked very closely with the pediatric rheumatologytoid medical community and on the other side, we were in contact daily with the families that were in need of better treatment for their SJIA.”

Also important to presenting to the B.C. Ministry was having both the patient and the medical community behind them. Wilson said that was the key to creating the strongest campaign possible.

Wilson added that they also worked with Arthritis Consumer Experts who provided guidance on how to approach their campaign. “

“Whenever you’re a patient organization thinking about launching your campaign you should definitely reach out to other organizations who been there before and who haves some expertise on how to talk to government,” advised Wilson.

Some of the advice those organizations gave Cassie and Friends was to present to the government in a positive and productive manner that lets the Ministry know that the advocacy group appreciates the work that the Ministry has done in its review and then explain the concerns from the family’s and doctors’ perspective.

So did their petition work?

Yes.

Wilson informed Zeal Access that this past Monday, one child was given exceptional access to canakinumab. “And just in a few days, it has absolutely transformed her life already,” confirmed Wilson.

Moving forward, Wilson said her organization will continue to advocate for more transparency for allowing families to gain exceptional access to the drug as well as advocate for a permanent paediatric rheumatologist to be part of the special committee.

One final piece of advice Wilson has for other advocacy groups preparing to fight for a child’s access to care is to be ready for the media. “Be sure to put some thought and planning into how you are going to handle the media frenzy that can happen when the media gets ahold of a heartbreaking story.”

 

Image of the Prime family (recipient of getting access to canakinumab recently) courtesy of Cassie and Friends.