Lawrie McFarlane: Pricey drugs create endless ethical issues

Just when you thought the pharmaceutical industry could sink no lower, Novartis proves you wrong.

The Swiss-based company has developed a drug that treats spinal muscular atrophy in young babies. The disease is often fatal, and always heavily damaging to quality of life.

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There are just two problems. First, the company is asking $2.1 million US for a single dose. Yet it isn’t clear, from the clinical trials to date, how much benefit the drug (Zolgensma) confers, and how long the benefits last.

In one trial, just over half the babies gained the ability to sit up on their own. Heartwarming, but how far does it lead?

Second, the company says it doesn’t have enough production capacity to market the medication outside the U.S. So the solution offered is to hold a lottery in countries where Zolgensma isn’t licensed, Canada among them.

The plan is to give away, free, 100 doses to children selected by a lottery. If your child has the disease, you apply and hope to get lucky.

That raises a number of questions. First, if the company hasn’t the capacity to produce enough doses by itself, why not partner with other firms to increase output?

Then again, this looks very much like a blatant attempt to pressure countries into covering the drug. Sign up a handful of families, provide the treatment, and wait for the inevitable clamour from others who lost out on the lottery.

In effect, the company is using a show of fake compassion to gain markets for its drug.

And where did that $2.1-million US figure come from? We’ve seen this movie before.

A new drug for a rare condition is produced, and immediately priced through the ceiling. Behind closed doors (naturally) a lower price is negotiated.

But the process is so shrouded in secrecy, it’s difficult to say what a fair price tag would look like. Here is what we do know. In 2017, across all drug categories Novartis sold in our country, it spent just 3.9 per cent of sales revenue on Canadian-based research and development.

Now that’s the company’s average budget for R&D. No doubt some individual drugs are more expensive to develop. But the line we hear peddled by the industry, that huge price tags are needed to recover their costs, looks more than a little phoney in light of that 3.9 per cent figure.

The broader ethical issue is whether we should permit the licensing of drugs that are far beyond anyone’s notion of affordability, with no end in sight of escalating prices.

Consider the plight of families whose child has been diagnosed with a fatal disorder, but learn there is a drug that could be life-saving. Now consider their state of mind when they are told it will not be offered. That seems unduly cruel.

The federal government licenses new drugs in Canada, and refuses to face this issue. Basically, if a medication has passed clinical trials and the manufacturer applies for a licence, Health Canada grants it, regardless of cost.

In essence, the department treats massively expensive new drugs like any other commodity — something a consumer can choose or not choose to buy — and leaves the ethical issues up to the provinces and territories.

This approach might have worked in the past, when relatively few new medications came on the market each year. However, the number of new expensive drugs (i.e. drugs costing over $100K/patient/year) available to treat rare diseases has increased dramatically from 15 in 2009, to 79 in 2019.

The only workable solution would be an international effort to bring drug licensing under some form of common protocol. It hardly needs saying how difficult that would be.

Yet without some such co-ordinated approach, we will face an endless series of heartbreaking dilemmas.

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