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Andre Picard in Montreal on September 17, 2010.John Morstad/The Globe and Mail

Ontario announced last week that it will pay for the drug eculizumab for the treatment of some sufferers of the ultra-rare disease atypical hemolytic uremic syndrome.

The decision is, by all accounts, the right one.

The genetic disease aHUS can be debilitating – requiring hours daily of dialysis and frequent blood transfusions – and life-threatening. Drug treatment can stabilize the condition and give sufferers some semblance of normality.

But the saga of Soliris (the drug's brand name) is a reminder that doing the right thing can be painfully difficult for policy-makers.

First of all the drug is exorbitantly expensive – about $700,000 a year.

The Patented Medicine Prices Review Board, which regulates brand name prescription drug prices in Canada, is embroiled in a legal fight with the manufacturer, Alexion Pharmaceuticals Inc. about the price.

So what is the province to do while that dispute is playing out? Should it refuse to pay and let patients suffer? Should it try to negotiate a lower price? If so, should it wait until all provinces are on board so there can be a single, best price across Canada?

The other challenge is to pay for treatment only for those who will benefit most – in this case, patients who have had repeated blood transfusions. Once a drug is in use, it's hard to say "no" and costs can soar.

So far, only Quebec and Ontario cover Soliris for the treatment of aHUS. All provinces cover it for treatment of another ultra-rare condition, paroxysmal nocturnal haemoglobinuria (PNH).

The drug approval system is complex, and the payment approval system even more so.

A new drug first has to be approved by Health Canada, which determines if it is safe (relatively) and effective (meaning it works as well as other drug treatments on the market, of which there are often none for rare conditions.) Then, the Common Drug Review, an independent committee, analyzes the cost-effectiveness of a drug and recommends whether it should be covered by provincial drug plans (or not). Initially, it said "no" to Soliris.

Provinces, in turn, decide if they will accept that advice. Ontario did follow the CDR recommendation but, when new research was published, it reversed its decision.

As mentioned, the PMPRP sets a list price (which is essentially the median price of seven other industrialized countries). But provinces, if they decide to list a drug, will try to negotiate a lower price with the manufacturer.

In theory, this is done collectively. But the Pan-Canadian Pharmaceutical Alliance has only negotiated a national price for 49 of the thousands of drugs on the market.

Orphan drugs – the name given to medications for rare and ultra-rare conditions – pose a particular challenge because the market is tiny and, as a result, the drugs are expensive. (Manufacturers want to recoup their investment and make a profit and to do so with a tiny client base, prices are high.)

For example, there are only 90 Canadians with the genetic condition aHUS. Even if all of them were treated with the drug at full list price (which they won't be), it would cost $63-million. That pales in comparison to an anti-arthritic drug like Remicade, which has sales of close to $700-million a year.

But, of course, aHUS is only one of about 8,000 rare conditions which, all told, affect about 2.8 million Canadians.

The real problem for governments in Canada is their lack of preparation.

There has been a determined effort to find treatments for rare disease since the U.S. adopted the Orphan Drug Act in 1983, and it has paid off. There were only 10 drugs to treat rare conditions before the law; since then, there have been more than 350.

Yet, Canada still does not have a plan in place. It does have an Orphan Drug Framework, which was unveiled in 2012 but is not yet fully implemented. It also has Orphanet-Canada, a useful portal for information on rare disorders and highly successful research efforts like FORGE (Finding of Rare Genes) Canada that are identifying genetic causes of rare disease and potential treatments.

What Canada doesn't have is a coherent policy for how it will approve and fund orphan drugs.

What happens is that groups of patients with rare and ultra-rare conditions mount public campaigns to draw attention to their plight – usually with funding from drug makers – and to demand access to a new drug.

Policy-makers are pressured to make important decisions with scant evidence. For example, Soliris was studied in a total of 87 patients with PNH and 67 patients with aHUS. Of course, sufferers want a drug funded, even if it offers a marginal benefit.

But there are opportunity costs: If a aHUS patient gets a $700,000 treatment, what other treatments will the provincial health system not be able to offer? After all, budgets are not unlimited, even if demands for funding are.

A few provinces – namely Ontario, Alberta and B.C. – have "special pathways" for reviewing and funding orphan drugs but there is not enough consistency or transparency in these policies.

These decisions – what to fund? how much to pay? where to draw the line in the sand? – are already causing a lot of hand-wringing.

But the challenges are only just beginning. Of the 350 new orphan drugs – and more to come as genetics research opens new treatment pathways – Canadians only have access to about half of them.

There needs to be a coherent plan on how to review new treatments quickly and fairly. It's not good enough to do the right thing by accident.

André Picard is The Globe's public health columnist.

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