It’s a heart-wrenching story, and one we hear time and again. People with an incurable illness are offered hope by a new drug, only to have it snatched away by uncaring bureaucracy.
This time, parents of children with muscular dystrophy are battling the US medicines regulator, the Food and Drug Administration (FDA). On Monday, families accompanied by young boys in wheelchairs told the agency’s panel of advisers how an experimental drug called had slowed their child’s disease. The manufacturer, Sarepta, claims the drug keeps children walking for longer. But the panel rejected the drug, saying that the small trial hadn’t demonstrated its effectiveness.
In the UK, similar scenes play out over decisions by the National Institute for Health and Care Excellence (NICE), which assesses the cost-effectiveness of drugs. The latest row involves that reduces lung infections in people with cystic fibrosis. The drug’s clinical benefits are small and its cost is high, so .
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“Few diseases are more in need of a cure than muscular dystrophy – but eteplirsen is not it”
But the drug does have some benefits. In a world where these agencies are meant to be acting for public benefit, how much should they be swayed by the voices of patients testifying that a drug is life-extending?
It’s a question that is likely to come up increasingly regularly for the FDA. The regulator is , because of a 2012 law introduced in response to claims that it was too strict at assessing medicines for diseases with few treatment options.
It has also become routine for drug companies to harness patient power to support the push to get a new medicine approved. Such lobbying reached a nadir last year with Addyi, the “female Viagra”.
The daily pill only boosts sexual encounters by one a month and can cause fainting. But the that it had made them feel like new women. The FDA approved Addyi.
Need for objective tests
Muscular dystrophy is a different matter. It is a degenerative muscle condition that strikes down young boys and is usually fatal by the early 20s. There can be few diseases more in need of a cure. Unfortunately eteplirsen does not seem to be it.
No one doubts the sincerity of the parents who spoke at the FDA hearing. But medical history is littered with examples of people feeling like a treatment is helping them, when careful, objective tests show that, in fact, it is not.
In this case the careful, objective test hasn’t even been done. The and their walking abilities were compared with historical patient records.
This approach is notoriously prone to making a medicine look better than it really is. The normal method is to do placebo-controlled trials – and ones involving hundreds of people. In this case, the scientific panel seems to have made the right call. Approving the drug would give families false hope, not to mention wasting everyone’s money.
Patients should be heard but they shouldn’t call the shots. There are widespread perceptions that miracle cures are out there and people would be getting them, if it weren’t for penny-pinching bureaucrats. This just isn’t true. It would be more useful if patients lobbied the pharma industry to bring down prices. Making drugs more affordable could be a miracle cure for countless patients.
This article will appear in print under the headline “When the customer isn’t always right”
