Issue 9 / 13 March 2017

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.


To find a doctor, or a job, to use GP Desktop and Doctors Health, book and track your CPD, and buy textbooks and guidelines, visit doctorportal.



Specialists should be required to publish their fee schedule so patients can make a free choice
  • Strongly agree (51%, 174 Votes)
  • Agree (23%, 79 Votes)
  • Disagree (10%, 34 Votes)
  • Neutral (8%, 27 Votes)
  • Strongly disagree (8%, 26 Votes)

Total Voters: 340

Loading ... Loading ...

2 thoughts on “Strings attached to patient lobby groups

  1. Prof Jane Andrews says:

    Agree with Jane McCredie that open disclosure is a must and that the example she cites of a hugely efficacious, and apparently not very effective drug was approved under duress is appalling – however we have a very different system to the USA – whilst slow and unwieldy at times, our TGA and PBAC (mostly) have good evidentiary rules which are followed to ensure this sort of result doesn’t occur.

    I also agree with anonymous that patients alone simply do not have the resources to represent themselves and their interests effectively in discussions with regulatory agencies and so need to be able to garner support/resources where they can.

    The best way to balance these 2 competing interests is to be pragmatic and realistic – rather than moralistic.
    If all interests are disclosed – motives can be judged and we should likewise insist our regulators adhere to consistent rules when making judgements – they can also be guilty of bias… it is not a one way street.

    We could however, do things EVEN BETTER if instead of having a “user-pays, cost recovery model” for drug access, which means submissions MUST be bank rolled by big pharma to succeed, our regulators were charged to liaise with medical experts to proactively look out for the treatments we’d like to have in Australia which would most benefit our health system. Those with large commercial backing could be invited to apply, and unsponsored (or minimally commercial) therapies could be assisted.

    The same approach could/should be taken with MSAC for consistency!

    Now, there’s an idea – us seeking the therapies and investigations we want/need – not simply passively accepting/evaluating the commercial ones coming to us!

  2. Anonymous says:

    There is an assumption in this article that patient advocates have the necessary personal resources to put their views to government, its departments and authorities in matters of their concerns. The reality is that many patients and the families of patients with chronic or life threatening diseases are under significant time, resource and emotional pressures that preclude them individually or as a group from engagement in processes that require levels of expertise they do not have and may need to purchase and are inevitably expensive and time consuming.

    This is in stark contrast to government bodies with very significant resources. Often the whole weight of departments are put to the front in processes such as MSAC that might seem to outsiders to be driven more by fiscal constraints than patient outcomes. The lack of real transparency and the uninterpretable rationales behind some decisions of these panels should also give cause for concern in what is really a David and Goliath battle.

    It is true that medications or procedures which over-promise should not be funded but it is also true that ones that do deliver to patients should. Under-resourced patients will get help where they can to be able to have their interests put forward and given the history of reviews in this area if that comes from sources seeking to ensure their patients get that best treatment for their conditions then so be it.

    On this article’s analysis doctors should not help patients put their case for extensions of MBS funding to new, safe, efficacious and beneficial treatments. I think in the age of bureaucratic self-righteousness, fiscal-apologias and political correctness that more thought needs to be given to critiquing the bias and predetermination in fully publicly funded government sponsored review processes. Doctors may hold concerns about some that are currently underway or restrained in providing their patients from securing new and innovative treatments.

Leave a Reply

Your email address will not be published. Required fields are marked *