Strings attached to patient lobby groups

WHEN a new treatment for Duchenne muscular dystrophy (DMD) was put up for approval in the US in 2016, patient advocacy groups and hundreds of individual patients and their families packed the Food and Drug Administration’s (FDA) Advisory Committee meeting to give emotional testimony in favour of the drug.

Committee members found themselves under “intense and near-incessant pressure from a large public audience”, Nature reported.

Despite that pressure, the committee voted narrowly against approval of eteplirsen (Exondys 51), based on a lack of clear evidence of efficacy for the treatment, which costs around US$300 000 a year per patient.

But, as FDA scientists were soon to find out, it’s hard to resist the appeals of children who are suffering and their parents.

In September 2016, after a sustained campaign from patient advocacy groups, the regulator overruled the scientific committee’s findings and announced that the drug had been granted accelerated approval.

A member of the Advisory Committee responded, telling Nature the decision lowered the agency’s evidentiary standards for drug effectiveness to “an unprecedented nadir”.

Of course, everybody wants the best for children facing a devastating genetic condition like DMD. But we also need to know that approved treatments are safe and effective and to understand the interests of those lobbying on their behalf.

If patient advocacy groups are going to wield this kind of influence in the regulatory system, they need to be transparent about any connections they may have to the commercial interests they are supporting.

I’ve written about this before, but not much has changed, at least in the US, if a new study is any guide.

Researchers from the University of Pennsylvania examined tax records, annual reports and websites of 104 large patient advocacy organisations to assess their connections to industry and how fully these were disclosed.

The report card, published in the New England Journal of Medicine, isn’t good.

At least 83% of organisations received financial support from drug, device or biotechnology companies, but almost none of them fully disclosed the amounts of those donations or how they were used.

Of the 57% that gave some information about donation amounts, almost all gave a range rather than a precise figure. And the ranges could be very wide, extending in some cases from below US$250 to over US$1 million.

Inadequate disclosure meant the researchers were unable to estimate the proportion of most organisations’ revenue that was provided by the industry, making it impossible to know just how dependent they were on the companies whose interests they could end up promoting.

For what it’s worth, for those organisations that did provide enough information to estimate this, the researchers found that about 40% received more than 10% of their revenue from industry donations.

It’s not just a question of cold, hard cash.

At least 39% of organisations had a current or former industry executive on their board, and at least 12% had an industry executive in a board leadership position, such as chair or deputy chair.

Those figures are almost certainly an underestimate, as more than a quarter of organisations did not disclose employment details for board members.

In Australia, we perhaps have slightly more transparency around relationships between patient advocacy groups and industry. Members of Medicines Australia are required to disclose direct financial support for health consumer organisations, though the organisations themselves may well not disclose it.

And, as the example of board positions shows, direct cash donations are not the only way industry can influence the direction of an organisation.

Corporate generosity is a good thing, provided there are no strings attached. Full disclosure is the only way to ensure that’s the case.

Jane McCredie is a Sydney-based health and science writer.

 

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