New hope for ITP patients dashed by high drug cost

A newly available drug would allow a woman with a life-threatening bleeding disorder to resume 'a normal life,' if she could afford it.

Drug costs $43,000 per year

'It was a dream,' Chrystal Gellein recalled of her time taking the drug. (Submitted by Chrystal Gellein)

Chrystal Gellein was 18 years old and living in Courtenay, B.C., when she was diagnosed with a life-threatening bleeding disorder called Immune thrombocytopenic purpura (ITP). After 20 years of unsuccessful treatments, a newly available drug would allow her to live a normal life again — if only she could afford it.

"Because of ITP, I've had to give up not just my work, but most of the activities that I used to enjoy: skiing, skating, bicycle riding … all because any trivial injury and I could bleed to death," said Gellein.

In ITP, the body's immune system destroys its own platelets, which are necessary to prevent and stop bleeding. An estimated 5 out of 10,000 Canadians have the disorder, which causes anything from extensive bruising and frequent nose bleeds to life-threatening internal bleeding.

This often requires patients to get multiple blood transfusions over the course of their lives to stabilize them in emergency bleeding, but Gellein was a Jehovah's Witness, a faith which does not allow her to receive blood transfusions, putting her at a much higher risk for fatal bleeds.

The main treatments for ITP are steroids or immunoglobulin therapy, often requiring a hospital admission. Failing this, patients usually undergo surgery to have their spleen removed.

Nothing worked for her until she tried eltrombopag, one of a new class of medications known as thrombopoietin (TPO) receptor agonists. She qualified for this during a surgery to have her gallbladder removed, but only for a few months.

"It was a dream," said Gellein about the drugs. "I was finally living a normal life, going back to work and on meds with no side-effects. But when the eltrombopag ran out, my platelet counts dropped. I started medications that caused me anxiety and depression and haven't been able to work."

Before her diagnosis at 18, Chrystal Gellein enjoyed skiing, skating and riding a bicycle. (Submitted by Chrystal Gellein)

Gellein loved working with kids and previously worked in a daycare but has not been able to work since 2014.

Dr. Donald Arnold, a hematologist at McMaster University in Hamilton, Ont., and global leader in platelet disorders, said these drugs have changed the landscape for the treatment of ITP: "More so than any other drug and with more success and fewer side-effects."

High-cost concern

The issue: one month of treatment costs $3,640, and most provinces have very restrictive policies to approve access. To qualify, patients must fail multiple alternative, lower-cost treatments. Many of these alternative medications have little to no evidence for improving ITP.

For Gellein, her platelet counts stabilized to the point where they were not life-threatening and so did not qualify for the new medications. But the side-effects from the other medications made each day a struggle.

"I started new medications that caused me anxiety and depression," said Gellein. "I couldn't go back to work or support my own kids with their issues in their teens because I was too busy with my own health."

Dr. Donald Arnold, a hematologist and global leader in platelet disorders, says access to eltrombopag is a huge problem. (Submitted by Dr. Donald Arnold)

Eltrombopag costs $43,000 per year. It would be more than a million dollars for her to use it over 25 years, which is often the norm for chronic ITP patients. Gellein and her physicians have applied for government coverage of eltrombopag multiple times over the last 10 years, but have been rejected repeatedly. And while her husband did have some private insurance, it was not enough to pay for the medicine out of pocket.

"Access is a huge problem," said Arnold. "My conversations with patients often start with, 'Do you have coverage?' I won't even talk about eltrombopag to many patients because I know it's not an option for them."

Drug fails funding review

When new drugs come onto the market, pharmaceutical companies are required to submit their research to the Canadian Drug Expert Committee (CDEC) for review. CDEC then analyzes the research and makes recommendations for coverage. Provinces use this to guide their coverage policies.

All provinces require that cheaper medications and treatments be tried first to make sure they are not effective, before more expensive medications can be considered for funding.

Promacta (eltrombopag), marketed as Revolade outside the U.S., is a prescription medicine used to treat low blood platelet counts due to chronic immune thrombocytopenia (ITP). One month of treatment costs $3,640. (Novartis)

According to CDEC, eltrombopag failed the initial review for multiple reasons, including no comparative research to show that it was better than the current standard of care, and no justification for its exorbitant cost.

The BC Ministry of Health's special access program stated the onus is on the pharmaceutical companies to resubmit the information for further consideration. The cost for resubmission can be high, and drug companies often will not resubmit if they feel there has not been enough new evidence since the initial submission. There has been no resubmission to date.

'From the dark ages'

Dr. Wendy Lam, a hematologist in British Columbia and co-chair for the upcoming Canadian Conference on ITP in October 2019, said there is now much more evidence now to support funding these patients.

"The current treatment we are using is from the dark ages and it's time to move beyond the status quo," said Lam. "These patients [are] young and their lives are paralyzed by their disease."

Lam and Arnold are part of a group of physicians from across Canada who have banded together to write a letter urging the Ontario government to re-examine its 10-year-old criteria to access these drugs. They hope that changes in Ontario, which has the some of the most restrictive criteria, will lead other provinces to follow suit.

'My life could have been so different'

Without access to eltrombopag, Gellein continues to take other medications and suffer from side-effects of anxiety, depression, weakness, nausea and dizziness. She is admitted to hospital several times each year for emergency treatment, which is common for patients with uncontrolled ITP.

She wants people to know the ripple effect it has caused on those around her, and desperately wants her life back.

"My life could have been so much different with these drugs," said Gellein. "Something needs to change, if not for me, for the hundreds of young people being diagnosed with ITP in Canada every year."