Healthy Skepticism Library item: 780

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Publication type: news

Griffin K.
Putting a price on human life: As health costs soar, should PharmaCare pay for expensive but unproven drugs for rare diseases?
The Vancouver Sun 2005 Jan 28

Full text:

Julie Cowie shows an intravenous dose of the drug Fabrazyme she takes for Fabry disease. The drugs costs about $290,000 year.

An American-owned pharmaceutical company that briefly stopped dispensing for free an expensive drug to Canadian sufferers of a rare genetic disease decided Thursday to keep supplying the drug until a dispute over payment is resolved.

Genzyme Canada Inc., owned by Genzyme Corporation of Cambridge, Mass, decided Dec. 29 to send out invoices for Fabrazyme, used to treat Fabry disease in which affected individuals lack an enzyme needed to break down fats and sugars.

Fabrazyme is one of the most expensive drugs in the world, costing about $290,000 for a 70-kg adult patient per year. As Fabry progresses, blood vessels, tissues and organs deteriorate and patients can die prematurely from heart disease, kidney failure or stroke.

Julie Cowie, a Vancouverite who has been receiving Fabrazyme for four years, said she was told during her two-hour infusion Jan. 13 at Vancouver General Hospital that the treatment would be her last until the payment issue between the drug company and the provincial health ministry was resolved.
Cowie, 66, has an enlarged heart and has already had two strokes.

In a meeting Thursday morning at Medical Daycare at VGH, when she expected to receive her usual infusion, Cowie was told by Dr. Sandra Sirrs she wouldn’t be receiving her infusion because no arrangement had been reached with Genzyme.

“I want you to have your treatment as well.” Sirrs said. “Given your severe disease symptoms, your treatment and therapy should not be interrupted.”

But just hours later, Cowie was informed that Genzyme had relented and was not requiring payment for Fabrazyme.

“What I’ve been told is that Genzyme is going to tear up the invoices that they’ve billed,” Cowie said. “They’re going to continue providing it for free until the end of February, when there should be some funding in place.”

Cowie said she wasn’t interested in all the bickering over how the drug was paid for — just that she gets it.

“Treatment — that’s all I want,” she said.

An official speaking for Genzyme confirmed the company has extended the due date for outstanding invoices to the end of February.

Susanne Courtney said in an earlier response to several questions by e- mail that Genzyme has provided Fabrazyme to patients free of charge under various criteria since 2001.

“Genzyme continues to fill orders from hospitals and they are invoiced, as before,” said Courtney, from the Courtney Rainey Group in Toronto. “The decision to treat patients rests with the health system.”

Worldwide, Genzyme sold $209 million US worth of Fabrazyme last year — up from $81 million in 2003. Total sales for Genzyme were $2.2 billion US last year, up from $1.6 billion.

Last November, the issue of whether expensive drugs should be covered by provincial prescription drug programs became a public issue when the Canadian Expert Drug Advisory Committee (part of the Common Drug Review) advised provincial prescription drug programs not to cover the cost of Fabrazyme.

As well, CEDAC recommended that Replagal, another drug used to treat Fabry disease, not be listed. Replagal is produced by Transkaryotic Therapies Inc. of Cambridge, Mass.

Paul McCabe, country manager for TKT Canada, said TKT has never sent invoices for Replagal but has decided to extend its timeline for reimbursement by a month to the end of February. He said TKT has offered to make Replagal available to PharmaCare for $50,000 less per patient per year than Fabrazyme, for in-home treatment that takes only 40 minutes.

In its ruling on Fabrazyme, CEDAC said:

• A 20-week controlled trial along with other “less rigorous clinical trials failed to show” a clinical benefit based on neurologic, renal and cardiac function;

• While it recognized the “small number of patients with Fabry disease makes the conduct of large, randomized trials difficult,” the “observational studies were of poor quality” and it would have been “useful to follow all participants over many years to document progression of Fabry disease and the occurrence of outcomes while on treatment;”

• The studies appear to “report upon sub-groups of patients, rather than all patients, thus raising concerns about why information from some patients was not provided;”

• It has been “argued that the costs of drugs to treat rare diseases are often high because of the relatively small number of patients for whom the drug is indicated. However, it is difficult to justify recommending reimbursement for such an expensive drug which, at this time, has little evidence of effectiveness based on clinical endpoints. Reimbursement of (Fabrazyme) would raise questions about equity, since drugs that have not been shown to be cost-effective for other diseases are not generally reimbursed.”

Provincial Health Minister Shirley Bond said while she’s glad the drug companies have reconsidered and now will continue providing the drugs until the end of February after a meeting with Health Canada on Fabrazyme, they have to realize that the patients come first. She said this is an issue facing patients not only in B.C. but across the country.

“I want to emphasize that this is all about the patients. There is a great deal of concern when drug companies provide drugs to patients when there is not strong clinical evidence that they will benefit,” she said.

“We want to make sure that the drug companies are held accountable.”

Bond said she’s hoping the drug companies can provide new clinical evidence about Fabrazyme and Replagal so CEDAC can reconsider its recommendation.
She said she is confident a resolution can be found once the drug companies realize their responsibilities to patients and sit down with the ministry to resolve the issue.

In a copy of a letter e-mailed to The Vancouver Sun, Peter Brenders, a health affairs executive with Genzyme, said Genzyme has asked CEDAC to reconsider its decision “on the grounds that the recommendation is clearly not supported by the evidence available and the noted reasons are misleading and not reflective of all the available information.

“We remain concerned [about] the validity of the [Common Drug Review] process for this rare disease and question if the approach is relevant considering the situation,” he said.

Regarding the small sample size of the Genzyme studies, Brenders noted in a point-by-point reply that “thousands of patients to place on clinical trials simply do not exist” because so few people have Fabry disease.

A report of a Fabry forum in Toronto in October sponsored by Genzyme said Canada needs a national network for Fabry disease that should be expanded “to address similar rare diseases, fostering a collaborative rather than a competitive approach to obtaining access to treatment.”

Fabrazyme is one of group of drugs called orphan drugs. The name comes from the 1983 U.S. Orphan Drug Act that provides incentives to drug companies to do research into so-called orphan diseases, defined as diseases that affect fewer than 200,000 people.

The U.S. act gives private companies a 50-per-cent tax credit for clinical trials, provides public money for research grants and allows seven years of market exclusivity following product approval. In the past 21 years, the U.S. government has spent $138 million in public money on orphan drug research and production.

The idea behind the legislation is to provide incentives to private companies to develop treatments for diseases that affect relatively small groups of people that otherwise would not provide a reasonable profit. The act has been cited as the single most important factor that has helped spur the biotechnology industry in the U.S.

There is no similar legislation in Canada. As well, Canada lacks a national health policy on treatment for orphan diseases.

More than 231 orphan drugs have come on the market since 1983 — compared to only 10 in the decade before.

Across Canada, there are about 130 patients with Fabry disease but not all have symptoms severe enough to require Fabrazyme. In B.C., about 25 people suffer from Fabry’s disease, but only nine people qualified for the drug.

If PharmaCare started paying that bill, it would cost $2.6 million a year.

Should PharmaCare pay?

It seems unbelievably heartless to ask. If it was you or someone you loved, there would be no question in your mind that PharmaCare should pay.

But one of the issues is this: In coming years, more of these expensive, precisely manufactured drugs geared to treating rare diseases will be coming on the market. When budgets are limited, how do health officials decide on what to spend public money — the next wonder drug or reasonable wages for hospital cleaning staff?

It’s the kind of choice that Tommy Douglas, the country’s founder of socialized medical care, probably never imagined the system would have to face.

On the PharmaCare side is the challenge of rapidly increasing costs.

Between 1996 and 2002, spending by PharmaCare increased 77 per cent, from $371.9 million to $658.1 million.

PharmaCare Trends 2002 Update states that the increased costs are due to more patients using drugs outside of hospitals (which are paid for by PharmaCare rather than hospitals), an aging population, more people living with chronic diseases such as diabetes and arthritis, and “newer, more expensive drug therapies.”

The 10 most expensive drugs per patient in 2003 cost PharmaCare about $35 million — with $27.5 million accounted for by new drugs added to the prescription drug program since 1996.

The most expensive of those drugs is Cerezyme, also manufactured by Genzyme, at $330,000 per patient. Cerezyme is used to treat Gaucher disease, a disorder similar to Fabry.

The $1.98 million spent on Cerezyme for six patients compares with the $35.2
million spent on Lipitor for 93,821 patients. Lipitor is used to fight
high cholesterol.

The cost of the 10 most expensive drugs paid for by PharmaCare in 2003 was
$176 million.

Bond said as more expensive drugs come on the market, especially those for rare diseases, the best way for provincial drug plans to deal with them is to abide by the rulings of the Common Drug Review.

She said it is an arm’s length organization that looks at the scientific evidence of a drug’s effectiveness.

“I think the Common Drug Review gives us a solid foundation we require,”
she said.

Marnie Mitchell, chief executive officer of the B.C. Pharmacy Association, said paying for Fabrazyme should be based on answering one question: Does the drug work?

“I really think it comes to the question of the evidence. What kind of evidence can you bring together and once you’ve done a critical assessment, what does it tell you?” said Mitchell, a former head of PharmaCare.

“My sense is that that’s absolutely what the program is there for.”

She also pointed out that we probably wouldn’t be raising the same kind of questions about cost if the discussion was about taking care of patients with brain injuries, who spend years in institutional care, or about recipients of organ transplants.

At the University of Victoria, drug policy researcher Alan Cassels believes everyone should be cautious before automatically jumping on the bandwagon of what’s being produced by drug companies.

He said the drug company’s word that a new product is effective can’t be taken for granted.

“You have to see all the available data,” he said. “Sure there are some people who will have remarkable recoveries. How representative are they?”

Cassels, whose area of research includes media reporting on drugs and medical treatments, said he receives no money from any pharmaceutical company and has no shares in any drug companies.

Cassels said you also can’t take the word of pharmaceutical companies about the cost of developing a drug because that information is proprietary:
They don’t have to open their books to show how much they spent on research or the cost of manufacturing.

Cassels said the health ministry should play hardball and make drug companies justify their prices.

And when all else fails, he pointed out, drug companies aren’t above using the media to paint government agencies as the villains when public officials question the cost of new drugs.

Cassels said the controversy is, at least, opening up a debate on the cost of prescription drugs.

“On one level it’s good that this is getting press attention because it really means so much about what we value,” he said. “We have to talk about what these things are costing and what we’re getting from them.”

THE COMMON DRUG REVIEW:

The Common Drug Review is a single review process for deciding whether new drugs should be paid for by federal, provincial and territorial drug benefit plans — except in Quebec. The Canadian Expert Drug Advisory Committee critically appraises the evidence and makes a recommendation on whether the drug should be paid for by public drug benefit plans. The main objective of CDR is to have one rigorous and consistent approach to approving new drugs across the country. If CEDA says yes to a drug, the provinces can then decide whether to include the drug. If CEDA says no, the provinces can’t go ahead and approve the drug — unless CEDA is given new evidence and makes a new recommendation. The Common Drug Review began accepting drug applications in September, 2003.

THE 10 MOST-EXPENSIVE DRUGS PER PATIENT IN B.C.:

Chemical Brand Treatment PharmaCare Patients Pharmacare substance name for expenditures expenditures per patient

Imiglucerase Cerezyme Gaucher $1,981,526 6 $330,254

Misc. Hormones eg. Prolastin Liver disease $1,956,111 30 $65,203

Eprostenol sodium Flolan Pulmonary $811,854 16 $50,740

hypertension

Bosentan Tracleer Pulmonary $898,799 39 $23,046

hypertension

Octreotide acetate Sandostatin Acromegaly $17,262 1 $17,261

Epoetin Alpha Epogen-Procrit Anemia $46,845 3 $15,615

Infliximab Remicade Crohn’s,

Rheumatoid arthritis $7,253,475 508 $14,278

Interferon beta Betaseron Multiple sclerosis $15,933,269 1,132 $14,075

Somatropin Genotropin Growth hormone $1,864,161 161 $11,578

Entanercept Enbrel Rheumatoid arthritis $4,313,715 480 $8,987

10 MOST-EXPENSIVE DRUGS, TOTAL COST:

Chemical Brand Treatment Patients Pharmacare substance name for expenditures

Atorvastatin Lipitor (high cholesterol) 93,821 $35,446,201

Ramipril Altace (High blood pressure) 104,039 $22,133,595

Olanzapine Zyprexa (schizo-phrenia) 10,251 $21,943,042

Glucose 30+ (diabetes testing Strip) 58,044 $17,530,655

Interferon Avonex, multiple sclerosis 1,132 $15,933,269

Beta Rebit,

Betaseron

Omeprazole Losec ulcers, 26,608 $14,100,360 gastro-esophageal re-flux disease Venlafaxine Effexor depression 33,228 $13,205,991 Paroxetine Paxil depression, anxiety, 37,944 $12,943,771 Simvastatin Zocor high cholesterol 39,377 $12,613,954 Risperidone Rispardal schizophrenia 22,842 $10,416,854