This new blog is to relate what I discovered
- When generous friends wrote Alberta's then Health Minister, Fred Horne asking for Esbriet to be funded for idiopathic pulmonary fibrosis (IPF), including Minister Horne's response when I contacted him directly.
- Interesting tidbits from additional medical research on IPF.
- How physicians vary dramatically in their approach to patients with IPF.
In 2013 my spouse was diagnosed with IPF, a deadly disease with no known cure and a median life expectancy after diagnosis of ~3 years.
Besides the diagnosis, it was a shock to learn that the one known treatment at the time (pirfenidone / Esbriet) was approved for use by Health Canada, but not funded by provincial governments.
I subsequently read many scientific papers on the issue and wrote 4 blogs that examined what it's like to get an IPF diagnosis and why Esbriet is covered by public funding in the UK but not Canada. In brief, reasons include
- The Canadian Drug Expert Committee (CDEC), under the auspices of CADTH, decided that Canada should not fund Esbriet (18 April, 2013).
- The clinical trial results at the time were equivocal and CDEC opted for seeing the glass half empty, no doubt partly because Esbriet is expensive ($40,000 - $50,000/yr). Provinces would prefer not to pay for a drug that slows, but does not cure IPF, a disease affecting mainly the elderly.
- To access the 4 earlier blogs on IPF: 'While my guitar gently weeps' (Musings on idiopathic pulmonary fibrosis).
HOW GOVERNMENT RESPONDS
Many friends kindly wrote the Alberta government, usually the Health Minister, Premier, or their MLA, using a variation of the Canadian Pulmonary Fibrosis Foundations' provincial advocacy packages. Regardless of how personalized they made their appeals - including that they knew the UK but not Canada funded Esbriet - in return all, including me, received what amounted to a government form letter, which totally ignored the content of our letters.
In return I wrote Minister Horne and asked him to cut the crap and reply to 3 simple questions.
To his credit, Fred Horne replied to my direct questions. If I interpret Minister Horne's reply correctly:
- Unless CDEC recommends Esbriet funding, Alberta won't fund it.
- Alberta's expert committee (ECDET) accepts (rubber stamps) CDEC's decisions.
- Forget about Alberta's Short Term Exceptional Drug Therapy (STEDT), unless a drug is approved for public funding by CDEC OR Canada's provinces negotiate what they deem a reasonable cost for Esbriet.
- IPF now a treatable disease (CMAJ)
- Two drugs reduce the progression of IPF
- In 2014 The Journal of the American Medical Association (JAMA) reported that pirfenidone (Esbriet) reduced the relative risk of death or disease progression by 43% compared with placebo.
He put us in touch with Esbriet's maker to investigate if our insurance would pay. He submitted the patient's name to Alberta's Short Term Exceptional Drug Therapy program (STEDT).
But he soon moved his practice outside our city. Although close by, we were not prepared to travel there for continuing care.
- Don't worry about not getting funding for Esbriet. It has significant side effects and will not dramatically alter the course of your IPF disease.
- Let's take a few key tests (lung function test, echocardiogram, CT scan, 6 minute walking test, review earlier sleep apnea test results) so that I can assess your current medical condition. Then we'll discuss where you're at and treatment options, if any.
But giving false hope has its downsides. Dealing with cold hard facts is what we need to plan for the future, whatever that holds.
Some of the key things we've learned:
1. Public advocacy programs for government funding of expensive drugs to treat incurable diseases are worthwhile but work better under certain circumstances.
- Many voters are mobilized to advocate the cause.
- Helps, but does not guarantee success, if
- Celebrities participate
- Disease is common
- Victims pull at heart strings
- Health Canada approval does not equal provincial funding.
- Governments seem content to let advocates 'piss in the wind' and only fess up to reality when pressed.
- Provincial exceptional drug therapy programs to fund high cost drugs for rare conditions do not apply to drugs unapproved for funding by CADTH's CDEC. [Except perhaps for children with heart-wrenching diseases like this little girl.]
- One approach does not fit all because communication involves a communicator (Dr.) and a recipient (patient), and recipients also vary greatly.
- In our case we are more in sync with a reality-based approach and lucked out with second pulmonologist who doesn't give false hope, tells it like it is.
- Canada's CDEC clearly puts cost-effectiveness first. No doubt provinces prefer this.
- Otherwise, why would the UK's NICE committee recommend funding Esbriet to treat IPF, and Canada's CDEC recommend the opposite, based on the same evidence?
- For the record, NICE's 64-page report is transparent and discusses all issues in detail. CDEC's report is 5 pages.
- UK experts noted that it was unlikely that clinical trials for IPF treatments can ever have enough statistical power to detect a difference in mortality. They recognized this limitation.
- Yet new studies show Esbriet reduced the relative risk of death or disease progression by 43% compared with placebo.
- Canada's experts chose to ignore the issue of statistical power.
- Statistical power: Ability of a study to detect a real difference, if one exists. Power is affected by how big the difference is and sample size. If a difference is big, it's easier to detect. And large sample sizes make a real difference easier to detect.
- In discussing why spouse's lung biopsy showed evidence of pulmonary hemorrhage, not one specialist could explain it.
- Seems medicine remains a science and an art.
As we deal with a diagnosis of an incurable disease, idiopathic pulmonary fibrosis, we focus on the glass half full scenario. Every day, every month, every year is a blessing.
Which brings me to an old but fabulous Beach Boys ditty.
- Good vibrations (Love this song)
- Autopsies in acute exacerbation of idiopathic pulmonary fibrosis (Interesting findings. Perhaps they can be of use in treating IPF.)
- Acute exacerbation in idiopathic pulmonary fibrosis.
- Clear diagram of the 3 types of disease progression in IPF.
- Drugs for rare diseases. Evolving trends in regulatory and health technology assessment perspectives (CADTH, Aug. 2013)
- Quote in Summary on Alberta: 'A Short Term Exceptional Drug Therapy program for funding high-cost non-formulary drugs for rare conditions is available for both in-patients and outpatients.' [In theory, not in practice.]