While my guitar gently weeps (Musings on idiopathic pulmonary fibrosis)

Updated: 3 Dec. 2013

This is the fourth of an ongoing series about idiopathic pulmonary fibrosis (IPF) and the refusal of Canadian provinces to fund the only known effective drug to treat it, pirfenidone (Esbriet).

This blogs title comes from a song that George Harrison wrote for the Beatles in 1968.

As noted in the prior blog, I'll try to explain why Canada and the UK made diametrically opposed decisions based on the same data.

To recap, the Canadian Drug Expert Committee (CDEC), under the auspices of CADTH, decided that Canada should not fund Esbriet (18 April, 2013). 

But in a report also published in April, 2013 the UK's National Institute for Health and Care Excellence (NICE) recommended public funding of Esbriet. 

NICE assessed that Esbriet provided a modest but significant improvement in IPF and "recommended pirfenidone because the benefit to patients justifies the cost."

NICE also recommended pirfenidone be discontinued if there is evidence of disease progression (a decline in per cent predicted FVC of 10% or more within any 12 months). So if Esbriet was shown not to work for an individual, it would not be funded for that person. 

The NICE Report is 64 pages and discusses the evidence for and against Esbriet in detail using the principles of evidence-based medicine. NICE also gives supplementary information. See Further Reading below.

In contrast, CDEC presents a 5-page summary of why they said no.

Judge for yourselves which review is more credible. Perhaps unfair, but NICE is more transparent. With CDEC are we just supposed to accept that they know best, accept the evidence and conclusions in the summary, and not worry about explicit details of their in-depth thinking?

This blog will focus on two key issues: 
1. Efficacy - contradictory studies 
2. Cost - contradictory interpretations 

Like each one of us, I interpret findings through the filter of my education, background, and biases. See original reports to assess for yourself. 

I've chosen selective data to minimize info overload and eyes glazing over. Although findings are simplified, I’ve tried to retain their essence. 'Panel' refers to either CDEC or NICE's expert committees.  

EXECUTIVE VERSION
For those who prefer the bottom line as the first line, my thesis is that Canada’s experts put cost first and chose to see the cup as half empty, whereas UK’s experts put patients first and saw the cup as half full. 

Who decided and how?
The UK NICE process is provided with specific details of who, what, how, and when. 

NICE assessed multiple sources of information and opinion, including clinical specialists, systematic review of scientific studies on pirfenidone, governments, professional medical and nursing associations, representatives designated by patient groups, and the manufacturer of pirfenidone. 

Canada's CDEC used its Common Drug Review process. Information sources included a systematic review of the clinical trials on pirfenidone, a critique of the manufacturers pharmacoeconomic evaluation, information submitted by patient groups on issues important to them, and the opinions of CDEC members. 

Criteria used to decide
Both groups based decisions on three main criteria:

1. Efficacy (inc. FVC, quality of life and mortality rates)
2. Cost and cost effectiveness
3. Safety and tolerability

Readers are directed to the original reports for Esbriet's safety and tolerability. Adverse side effects exist but are not serious enough to contraindicate using the drug to treat IPF, although long term effects will need to be monitored.

1. EFFICACY (Does Esbriet work?)
In medicine, efficacy means clinical effectiveness. Does a drug produce a measurable effect that is meaningful and beneficial to patients? 

To measure the effectiveness of Esbriet, the panels looked at the same two RCTs* of adults with mild-to-moderate IPF in 72-week, double-blind, multicentre trials with similar protocols: 

CAPACITY-2  (PIPF-004 in UK)
CAPACITY-1 (PIPF-006 in UK) 

*RCT The randomized clinical trial (RCT) is the gold standard of medical research. Similar individuals are randomly assigned to a treatment group (who take the drug) and a control group (who take a placebo). Double blind means that patients and researchers do not know who is in which group. Then, if there is a difference (patients on the drug improve more than those on placebo), it is more likely to be real. 

The primary outcome in both studies was the change in the percentage of predicted FVC* from baseline to week 72. 

*FVC. Forced vital capacity (FVC) is the volume of air that can forcibly be blown out after a deep breath in and is universally acknowledged as a reliable measure of lung function. A decline in FVC indicates disease progression. A10% decline within 6-12 months is associated with a significant increase in mortality.

In one study (CAPACITY-2) there was a statistically significant* improvement in the rate of decline in per cent predicted FVC with pirfenidone compared with the placebo (4.4% difference), but not the other study (0.6% difference).  

* Statistically significant. A 'statistically significant difference' means the results are unlikely to be due to chance and are more likely to be real. 

Neither Canadian nor UK assessors could explain why the results of the trials were contradictory.

A pooled analysis (possible because the protocols and methodology in both studies were similar), showed a statistically significant improvement in the rate of decline in lung function (per cent predicted FVC) with pirfenidone compared with placebo of 2.5%.

In other words, if data from both studies were analysed as if in one large study, the results are statistically significant (unlikely to be due to chance) and favour Esbriet, as does the one clinical trial. 

Canada v UK #1 (Efficacy and FVC)
Canada's experts noted that, if the 2 studies were contradictory, pooled results must be regarded as ‘exploratory’, i.e., not as valid.  

In contrast, UK experts noted the near-identical design of both studies and agreed that analysing pooled data, although exploratory, was reasonable. 

They concluded that the trials provided evidence that was adequate for assessing the clinical effectiveness of Esbriet. In one study and using pooled data, the drug showed a modest but significant improvement over a short duration (72 weeks).

Canada v UK #2 (Efficacy and Mortality)
Both studies showed no statistically significant difference in IPF-related deaths. 

However, a pooled analysis of IPF-related mortality suggested that pirfenidone was associated with a statistically significantly higher probability of survival compared with placebo.

Canada's experts noted that, in both studies, Esbriet showed no difference in prolonging life. Results of pooled data were invalid. Hence, there was insufficient evidence to determine if Esbriet prolonged life. 

In contrast, 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. Because of the relatively small number of IPF patients in the studies, there aren't enough patients dying over the short duration of the two clinical trials (only 72 weeks) to detect a difference.

UK experts recognized this limitation as one that would probably always exist in clinical trials of drugs to treat IPF. Although a possible survival benefit was uncertain, the UK decided this was the only evidence available for decision-making. 

Canada's experts chose to dismiss the evidence, apparently ignoring 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. 

Canada v UK #3 (Efficacy and Quality of Life)
Here the two panels agreed. The studies showed insufficient evidence that pirfenidone provides clinical benefit for quality of life. Quality of life was measured with a 50-item questionnaire that measures distress due to respiratory symptoms, mobility and physical activity, and the psychosocial impact of the disease. 

Both panels found that in the studies Esbriet did not affect related health indicators such as dyspnea (shortness of breath), respiratory-related hospitalisation, need for supplementary oxygen, etc.

2. COST AND COST-EFFECTIVENESS
In both Canadian and UK assessments, the manufacturer submitted a confidential estimate of costs, which were analysed by the panels’ experts.

How medical and public health experts decide cost effectiveness is complex and one of life’s great mysteries. Voodoo economics is the term that comes to mind. The key terms are QALY, ICER / ICUR. (Stick with me. It won’t be too painful.)

QALY
QALY is a well recognized method that indicates how many extra years of life - of a reasonable quality - a person might gain from treatment. QALYs offer many opportunities for researchers to influence results, including what constitutes a reasonable quality of life. NICE explained its QALY calculation.

ICER / ICUR
ICER: The incremental cost-effectiveness ratio is the ratio between the difference in costs and the difference in benefits of two interventions per QALY. 

ICUR: Incremental cost-utility ratio is similar to ICER and is calculated as follows (where A & B are interventions, treatments, drugs, etc.):

Cost of A – Cost of  B
---------------------------------------------------------------
No. of QALYs produced by A –those produced by B

Canada vs UK #4
CDEC says the manufacturer reported that pirfenidone compared with placebo is associated with an ICUR of $143,617 per QALY. They found faults with the manufacturer’s cost analysis and thought true costs would be higher.

In contrast, the UK calculated its ICER by comparing Ebriet NOT to placebo but to ‘best supportive care’. The manufacturer's probabilistic ICER was £24,000 ($40,349 CDN) per QALY gained, which was less than the UK’s Evidence Review Group's assessment but ‘an acceptable cost-effectiveness estimate on which to begin to explore further considerations’.

So….assuming ICER and ICUR are the same, Canada’s cost effectiveness model concludes Esbriet is more than 3 times the cost of the UK’s. Huh?

Did Canada’s ICUR compare Esbriet to placebo but not consider actual treatment costs that IPF patients would incur if not on the drug?

Did Esbriet's manufacturer give different cost estimates to Canada vs the UK?

PERSPECTIVE on COSTS
Cost-effectiveness thresholds per QALY vary from country to country:

• UK: £20,000 - £30,000 (NICE)
• USA: $50,000 
• Australia: $52,400 
• Canada: $20,000 - $100,000 

Are these thresholds appropriate? Who knows?

BOTTOM LINE
CANADA
Canada's experts judged the data on Esbriet’s efficacy and costs to be inconsistent, untrustworthy, and insufficient, or as Brit football fans might say about an opposing club, ‘They’re shite, total shite!’ 

Yet CDEC allowed that, although individual patients may benefit from pirfenidone, they were unable to determine the clinical criteria that would accurately identify these patients in clinical practice. 

Can’t identify who may benefit? Then no one gets it. 

CDEC took the approach that any evidence that supported Esbriet was iffy. They decided to interpret all study results as the cup half empty. One can only wonder why.

UK
NICE’s approach was the cup is half full. UK experts accepted the positive RCT results of the one study and the pooled study results of both. They acknowledged that, because of small numbers of patients with IPF on Esbriet, it was unlikely that studies could ever show a different in mortality rates. They found Esbriet to be cost effective compared to best supportive care and their threshold for QALY costs. 

Can’t identify who may benefit? Then stop funding Esbreit for a patient who shows a significant decline in lung function over 12 months. 

So what does all this mean? 

To me, it's similar to Canada's blood experts in the 1980s deciding to use stocks of FVIII concentrate for hemophiliacs because they were probably all infected, which turned out not to be true. And what a pompous, cold-hearted decision to make with the lives of others. 

It’s also analogous to Canada’s blood experts deciding that surrogate tests implemented in the USA for non-A, non-B hepatitis (now hepatitis C) were not scientific enough for them. Inadequate sensitivity and specificity to use the lingo of diagnostic tests. 

In retrospect these expert judgements were huge miscalculations and cost the lives of many Canadians and suffering to thousands of others.

Yes, I know you cannot compare Canada's so-called 'tainted blood tragedy' to its decision not to fund IPF's only known treatment, Esbriet. But some aspects are similar. 

Canada’s blood experts put cost first then. And I suspect that CDEC has put costs first in the case of Esbriet to treat IPF. Its committee members would never admit it, even to themselves. But influence is subtle. Today, cost saving in Canada's health system is the pervasive culture. CDEC's decision no doubt pleases provincial governments. 

In contrast UK experts decided to put patients first. The Esbriet studies with all their limitations - especially because of the short 72 week duration and not enough patient numbers and time to show if Esbriet prolongs life - still show the drug provides a modest and real improvement. 

Some might call that balancing cost with patient-centred care, a principle that Canada’s medical community gives lip service to, but seldom implements. 

FOR FUN
'While My Guitar Gently Weeps' epitomizes how I feel about Canada’s decision not to fund Esbriet. The CDEC decision revives memories of earlier cost-based decisions, lacks compassion, and I suspect lets an inflated sense of scientific superiority blind our experts to the big picture. 

For interest, Harrison song was ranked #7 on Rolling Stone's list of the 100 Greatest Guitar Songs of All Time, and #10 on their list of The Beatles 100 Greatest Songs.

• While my guitar gently weeps (George Harrison with Eric Clapton on guitar and other celebs)

I look at the world and I notice it's turning
While my guitar gently weeps.
With every mistake we must surely be learning
Still my guitar gently weeps.

FURTHER READING

NICE Resources

• IPF overview
• Information for the public
• How pirfenidone guidance was produced
• Key documents

As always, comments are most welcome.

Those were the days (Musings on idiopathic pulmonary fibrosis)

Updated 14 Nov. 2013

This is the third of an ongoing series about idiopathic pulmonary fibrosis (IPF) and the refusal of Canadian provinces to fund the only known effective drug to treat it, pirfenidone (Esbriet).

This blog’s title comes from a catchy 1968 ditty, Those were the Days. 

The blog outlines some of the obstacles to funding Esbriet, or any expensive drug or treatment. 

At its core, it’s a tale of medical experts looking at the same facts and reaching different conclusions. To some readers that may seem impossible but it becomes understandable once you realize that

• Medical experts, like all humans, have biases
• Experts are affected by external influences and pressures
• Medicine is an art as well as a science
• When evidence is borderline, decisions are more susceptible to biases and political pressure
  

The prior blog ended with asking, ‘What to do?’ As you may expect, we’re not accepting the government’s decision without a fight. As Peter’s lung doc asked, we’ve become advocates, asked friends to participate (which they kindly and generously have), and plan to continue on a broader scale via this blog and our Twitter accounts

• grumblingonions
• bogeywheels

To some, getting public funding for IPF, or other pricey drugs, may seem a lost cause. Despite the obstacles, we don’t accept that. Indeed, as James Stewart said in ‘Mr. Smith goes to Washington’, lost causes may be the only ones worth fighting for, and this one is worth fighting for.

Funding Esbriet to treat IPF has many obstacles. Some relate to the milieu in which government decisions, including those on healthcare are made, e.g., social and economic factors. Others are harder to pinpoint, such as the pressures that influence scientists trying to play by the rules of evidence-based medicine. Sometimes, even ‘smoke and mirrors’ may play a role.

We’ve decided to briefly outline 7 obstacles. As you read the obstacles, please examine your feelings. What emotions do you feel (not think)? What's your gut reaction?

OBSTACLE 1: Not my problem

Dismissing relatively rare diseases is easy. Because they’re rare, we think they’ll never happen to us. Kinda like ignoring risky behaviours like smoking or unprotected sex or sex with multiple partners or drinking and driving. Won’t happen to me syndrome.

But it’s even more like laughing at getting a deadly disease from blood transfusion because today’s blood supply is so safe. Except if you do get a deadly, even a minor, transfusion-transmitted disease, guess what? Suddenly you do care. A lot.

That’s the case with idiopathic pulmonary fibrosis. No one much cares about it except

• Physicians who treat it (or try to, given Canada’s provincial governments won’t fund the only known effective treatment, pirfenidone)
• Patients who have it
• Their family and friends

Besides the public not caring, other obstacles exist to funding Esbriet.

OBSTACLE 2: It’s probably your fault

Some smug citizens may be in blame mode. It’s a lung disease? Probably caused it themselves. After all, that’s a factor with so many preventable diseases in which lifestyle choices play a role.

• Lung cancer, emphysema, stroke: All increased by smoking, even though they can happen to those who have never smoked
• Skin cancer: Too much time in the sun without a hat or sunscreen
• Breast, throat, liver cancer: All increased in those who drink alcohol. Yes, alcohol is a known carcinogen.
• Type 2 diabetes: Probably a lifetime of eating junk food, being a couch potato, and growing obese, never mind genetic factors
• Coronary artery disease: Too much fat in diet, too little exercise. Never mind that fit people, including runners, get it
• Sexually transmitted diseases: Unprotected sex, too many sex partners

Turns out that ~30% of all cancers are preventable (Source: WHO). 

Simple answers appeal to all of us, especially folks who are unaware of the influence of factors like genetics, stress, and unknown environmental exposures.

And it's so easy to ignore the cost to our health system of fitness buffs who run marathons, play soccer, downhill ski, etc.

For interest, cancer funding varies greatly: (2006 USA data)

• Breast ca receives the most funding per new case, $2,596 and by far the most $ per death (41,430 deaths), $13,452. 
• Prostate ca, the most common cancer, receives the least funding per new case ($1,318) but, per-death (27,350 deaths), it ranks second, with $11,298.
• Lung ca is the biggest cancer killer (162,460 deaths), yet per-death receives the least funding among major cancers ($1,630)

OBSTACLE 3. It’s only old people

IPF affects mainly seniors. And seniors are in the winter of life, soon to depart this world, even without a disease like IPF. 

Ageism often that means that what happens to us is less important than what happens to those younger. That our concerns can be ignored, that we can be dismissed as old foggies, fuddy duddies, past it, etc. 

It's only natural because those who are young don't want to think of what happens when we age, the deterioration of our physical bodies and inevitable death.

Does ageism exist in Canada and elsewhere? Based on our experience it does. Shows itself in many ways, every day, from how you are treated at the bank, who is served first in stores, to how advertisements focus on the young and how health care professionals speak louder to seniors, as if we were children and deaf. Often oldsters are invisible, at least that's how it can feel.

When it comes to IPF, it's easy to envisage government officials like @premierRedford and @FredHorneMLA

saying, 'They're old people who will die from the disease anyway. Why waste money on them?'

It matters not that the age-related tsunami cost to healthcare is a myth. The issue is multi-factorial with a passionate band of evidence-based medicine gurus leading the charge. So sure they are right, rigid to the core.

OBSTACLE 4: It costs too much

Then there’s the cost fanatics, often people on the right of the political spectrum but not totally, who believe that the less government does, the better. or that we need to balance the budget above all else. Their thinking is as follows:

If we, the taxpayers, will fund an expensive drug like Esbriet, it had better show a BIG improvement in quality of life, lung function, and even extend life. And ideally affect me, my family, or someone I know.

Such voters do not realize many drugs and diagnostic tests funded by government to diagnose and treat other diseases do next to nothing. 

We won’t go into specifics as the blog would become too long, but iffy government-funded tests and treatments exist. Many involve cancer. Cancer is scary, we might get it, whereas IPF, what’s that? It doesn't matter that millions, even billions, are spent, with minimal results.

OBSTACLE 5: Governments obsess about cost

Provincial government fear the aging population and worry about how they’ll fund long term care facilities and the increased use of drugs among seniors. To some, it's a tsunami is about to strike.

Accordingly, provincial governments are more than happy to see expensive drugs like Esbriet nixed by the Canadian Expert Drug Committee (CDEC).

Social and political pressures are the realities that face physicians and IPF patients seeking government funding for Esbriet.

Now for the biggest obstacle...

OBSTACLE 6: Canada's panel of experts said no to Esbriet

The Canadian Drug Expert Committee (CDEC), under the auspices of CADTH, decided that Canada should not fund Esbriet. 

Because all Canadians, especially those affected by IPF, should know who our CDEC experts are:

Dr. Robert Peterson (Chair), Dr. Lindsay Nicolle (Vice-Chair), Dr. Ahmed Bayoumi, Dr. Bruce Carleton, Ms. Cate Dobhran, Mr. Frank Gavin, Dr. John Hawboldt, Dr. Peter Jamieson, Dr. Julia Lowe, Dr. Kerry Mansell, Dr. Irvin Mayers, Dr. Yvonne Shevchuk, Dr. James Silvius, and Dr. Adil Virani

The CDEC decision gives our provinces a convenient out. We don’t need to pay for Esbriet, our experts recommend not funding it. As UK football fans might quip about the drug after reading CDEC’s decision, ‘It’s shite!’

BUT BE AWARE the UK’s NICE looked at the same facts and concluded that the

• UK’s NHS would fund pirfenidone

OBSTACLE 7: Healthcare a provincial responsibility

Because In Canada, provinces and territories are responsible for healthcare, it becomes difficult for one province to fund a drug when others don’t. Difficult, but not impossible.

It's easier if the drug is for a toddler with a really rare disease like Maroteaux-Lamy syndrome with significant morbidity and mortality (only 9 affected children in Canada), so the funding is a one-off.

Naglazyme is NOT approved by Health Canada and costs $300,000 or more per year for children. Because dosage is tied to weight, cost can rise to $1-million/yr for adults. Those on it are on it for life since the drug does not cure the illness but stops it from worsening.

It’s harder to get government funding if it’s for a rare disease like IPF with significant morbidity and mortality that affects 1000s of seniors. 

It doesn’t matter if Esbriet, like Naglazyme, doesn’t cure the disease but stops it from worsening, that Esbriet is approved by Health Canada, but Naglazyme is not. 

Who’s to say Canadians from other provinces wouldn't move to Alberta? Unlikely for most seniors but possible for some. 

BOTTOM LINE

Can you see that societal biases and politics are involved, indeed rampant, in Canada's drug funding decisions?

In the next blog we’ll explain why Canada and the UK made diametrically opposed decisions, focussing on what we see as the two most important issues:

• Contradictory studies (clinical trials)
• Contradictory interpretations of what’s a reasonable use of government money to improve health, prevent and slow illness 

FOR FUN

Two songs fit this blog's issue of our story about IPF and Esbriet. 

The first, Those were the days, speaks to our youthful enthusiasm and certainty that in Canada, with its universal healthcare, we'd always receive needed treatment and the ill would not be financially punished.

• Those were the Days (Mary Hopkins)

Those were the days my friend
We thought they'd never end
We'd sing and dance forever and a day
We'd live the life we choose
We'd fight and never lose
For we were young and sure to have our way.

The second song comes to grips with reality: 

• Money makes the world go round (Lisa Minnelli & Joel Grey from Cabaret)

As always, comments are most welcome. 

You can't always get what you want (Musings on idiopathic pulmonary fibrosis)

This is the second blog in a series on idiopathic pulmonary fibrosis (IPF).

The  blog's title is based on a Rolling Stones ditty. The blog describes what happens when you try to access the only proven treatment for IPF.

AND THE BEAT GOES ON So, to continue our story....We waited for the call from the Esbriet drug representative and it came in about a week. Not surprising as the company no doubt wanted to get new clients for its expensive drug toute de suite.

The drug company is InterMune and it seems that Esbriet™ is its sole product. 

The Esbriet representative asked about our health care insurance company. Because I was fortunate to work at University of Alberta for 20+ years, unlike many seniors, we were able to transition to a decent health plan upon my retirement. 

We identified ASEBP, the insurance provider for ARTA and said it was highly unlikely ASEBP would pay for Esbriet at a cost of more than $40,000 CDN per year. We had 80% coverage but the maximum per year you could buy for drug coverage was $2000. 

Nonetheless, the Esbriet rep set up a conference call with us and ASEBP, at which time the insurer confirmed the details we'd provided. Bottom line: The 80% coverage was meaningless, given the annual drug maximum as $2K. 

Our lung doc's attempt to hold out hope for Esbriet, something we immediately judged as ludicrous, came F2F with reality. 

The drug company rep said it would continue to work to get Esbriet publicly funded. Indeed, a Calgary physician had applied to get Esbriet funded for a patient with IPF and it was to serve as a test case.

When we reported this to the lung doc, he said we should also be advocates to raise public awareness of idiopathic pulmonary fibrosis. Maybe if government officials  knew about it, and realized it was an issue to citizens, they might ultimately change their minds and fund Esbriet.

SAY WHAT?
At this point the initial shock of Peter being diagnosed with a relatively rare but fatal disease we'd never even heard of began to wear off. Our focus turned to investigating why Esbriet was not paid for in Alberta, Canada's oil rich province, its wealthiest province.

And this is when I started to get mad. For we soon discovered that many places in the world did publicly fund Esbriet. 

Having a scientific background, having taught how to write and interpret scientific papers, and having written a book on the topic, I carefully read the reports of  expert panels in different nations with interest.

The Canadian Drug Expert Committee (CDEC), under the auspices of CADTH, decided that Canada should not fund Esbriet. 

But in a report published at the same time the UK's prestigious NICE recommended public funding of Esbriet. NICE assessed that Esbriet provided a modest by significant improvement in IPF and was cost effective according NHS criteria. 

And NICE recommended treatment with pirfenidone be discontinued if there is evidence of disease progression (a decline in per cent predicted FVC of 10% or more within any 12 months). So if it doesn't work for an individual, stop it. Seems fair.

Moreover, it turned out that, besides the UK, many countries did publicly fund Esbriet, including

• Belgium
• Denmark
• Finland
• France
• Germany 
• Iceland
• Italy
• Sweden

So.... why did Canada's experts nix public funding of Esbriet? Are Canada's experts so much smarter than those in the UK and other European countries?

No doubt Canada's cash-strapped provincial governments were delighted not to fund Esbriet. Never mind the mega-bucks they spend on cancer and drugs for other diseases that yield minimal or no results. 

Did politicos like Alberta's Minister of Health Fred Horne and Premier Alison Redford have a clue about the suffering such a decision caused? I doubt it. 

DECISION TIME
So, what to do? 

• Advocate, as the lung doc said, by writing letters to government reps, even though we suspect it's like pissing in the breeze? Ask pals to do so?
• Lie down and give up? After all, if Canadian experts say Esbriet has no value, regardless of what UK and other European experts conclude, how can mere mortals dissent?
• Mount a broad offensive that's hard to ignore because it's realistic, heartfelt, and on the money?

The next blog will reveal what we decided. But we suspect you already know.

FOR FUN

• You can't always get what you want (Rolling Stones, 1969)

As always, comments are most welcome.

Sweet dreams or maybe not (Musings on idiopathic pulmonary fibrosis)

This is the first of a series of many personal blogs on a rare deadly disease, idiopathic pulmonary fibrosis (IPF), and its only known effective treatment, pirfenidone (Esbriet).

It's a tale of well intentioned promises, deceit, ageism, convenient government decisions, and arbitrary recommendations by health experts.

And as we know, the cliche is correct: The road to hell is paved with good intentions.

The blog's title comes from a favorite Annie Lennox song. It fits because we had retired and looked forward to 'sweet dreams', a relaxed life of doing what we loved: writing and drawing for Peter and part-time work in  transfusion medicine for me, as well as travelling the globe as the mood struck us. That was the sweet dream.

Edmonton writer Todd Babiak co-founded a company called Story Engine. Its premise & first step: 

• People are born to understand story.
• The first step in the Story Engine process is to seek your truth, the components of your story: your past and present, the way others see you and the way you see yourselves.

If you don't have IPF, why should you read the blog? Not in my back yard (NIMBY), you say? It's not my story, you say?

Well, you or someone you know may get IPF one day.The disease and its treatment reveal much about our Canadian health care system. The struggle to be treated presents dilemmas:

• How to best advocate for patients who are refused treatment by an arbitrary decision of a so-called 'expert panel' when similar panels in other countries recommend the opposite.
• Whether or not to call upon friends to serve as advocates.
• How to influence a provincial government, in this case Alberta Health, its Minister of Health, Fred Horne and Premier Alison Redford, when their decision may depend upon other provinces doing the same.

So, here's the start of our story about idiopathic pulmonary fibrosis.... 

BACKGROUND
This year we got a real shock. My spouse Peter was diagnosed with interstitial lung disease, aka pulmonary fibrosis. He had gone to his family physician because of extreme shortness of breath and fatigue with routine activities, a dry cough, and unexplained weight loss.

An ECG suggested possible heart disease. An X-ray suggested possible pulmonary fibrosis. Apparently, indications for pulmonary fibrosis were present on an x-ray 4 years earlier but now the fibrosis was more pronounced.

The docs decided to do an angiogram to identify to what extent, if any, coronary artery disease contributed to symptoms. Turns out not much. He had coronary heart disease but no blockages that warranted angioplasty and a stent. The surgeon joked that many people at Peter's age had similar blockages, they just didn't know it. 

The heart specialist told us that future investigation was in the hands of a lung specialist, aka pulmonologist, because the enhanced CT scan confirmed interstitial lung disease. 

The family physician then referred Peter to a pulmonologist, who prescribed multiple laboratory tests to rule out possible causes, as well as lung function tests to determine the extent of the lung damage.

Most importantly, he said Peter needed a lung biopsy to diagnose the type of interstitial lung disease and related cause before treatment could be determined. Turns out interstitial pulmonary disease, like ice cream, comes in multiple flavours. 

We'd never heard of the damn disease.But it apparently affects ~30,000 Canadians, with a projected 5,000 deaths each year.

ESBRIET
At the same time the lung doc gave us a brochure on idiopathic pulmonary fibrosis (IPF), the type with the worst prognosis and with no known effective treatment until 2012 when a drug called pirfenidone (Esbriet) was licensed by Health Canada. 

The doc also explained that Esbriet costs between $40,000 to $50,000 CDN per year, making it impossible to access except for the rich or being insured by insurance companies who will pay for it. Yikes! Cost is a fatal flaw....

In retrospect, we know why the lung doc mentioned IPF before it had been definitively diagnosed. It's the most common type of interstitial lung disease  and also fits with Peter's age group (senior citizen). 

IPF
In the meantime we scoured the Internet about interstitial lung disease, especially idiopathic pulmonary fibrosis. We learned that the idiopathic type is associated with gastro–esophageal reflux disease (GERD) and a history of smoking. Turns out Peter has GERD and smoked in his youth but stopped a whopping 28 years ago. It's important to note that associations do not equal causation. 

As expected, the lung biopsy showed Peter had idiopathic pulmonary fibrosis (IPF), idiopathic meaning cause unknown. 

The downside of IPF is that the life expectancy is 2-5 years from time of diagnosis, although some patients live longer, with what quality of life is unclear. IPF is characterized by excessive scarring in which fibrous tissue make the lungs rigid, and ultimately resemble a honeycomb. 

'SO WHAT'? MESSAGE
So... at this stage of the story Peter has IPF, we're told a drug called pirfenidone (Esbriet) can help, but its cost puts it beyond our grasp. What to do?

The lung doc said he'd fax the drug company's representative, who would contact us to discuss options.

We dig why the lung doc held out the hope of Esbriet. He had good intentions and wanted to give us hope. Must be tough to be a physician who tells patients they have a fatal disease and there's nil we can do. 

Is giving them false hope the way to go? Don't know but gut reaction is we prefer reality.

So....we wait for the call from the drug company and it comes. See next blog.

For fun:

• Sweet Dreams by Annie Lennox (Live 8 concerts to 'Make Poverty History', this one in London, 2005)