Patient Power: trials subjects no more

Are MSers ready to take-on Academia and Pharma? #MSBlog #MSResearch

“Will progressive MSers take on Pharma and Academia in the same way that ALSers have done? You don’t need to read too many comments on this blog from progressive MSers to realise how frustrated they are with the slow progress in getting effective treatments. We the academic community are partly to blame; we often start trials without thinking about what happens if the study is positive, i.e. we haven’t really got a path sorted out for the licensing of repurposed drugs that are off patent; for example simvastatin.”

Paul Wicks. Subjects no more: what happens when trial participants realize they hold the power? BMJ 2014; 348 doi: (Published 28 January 2014).


….. The social contract of the randomized controlled trial is imbalanced: patients adhere to arduous protocols, are randomized to placebo, and are blinded to their health status. Although most participants (>90%) would like a lay summary of results, only a minority (<10%) receive one, with the remainder left with the option of paying around $30 (£18; €22) to read the results once the study is published in a peer reviewed journal…..

…. In 2007, patients with amyotrophic lateral sclerosis (ALS) used Google to translate an Italian conference abstract suggesting that lithium carbonate might slow their illness. In a publication titled “Lithium delays progression of ALS” 16 patients treated with lithium (all of whom survived 15 months) were compared with 28 control patients (a third of whom did not survive the trial). Within six months of the abstract’s publication 160 patients reported obtaining lithium off-label and tracked their progression using Google Spreadsheets and the validated ALS functional rating scale (ALSFRS-R).  A patient in Brazil and a caregiver in the United States initiated this patient led study, raising the question of where ethical oversight lay….

…. The concept of “scientific altruism” may be being trumped by “maximize your chance of survival.” For better or worse, digital tools enable greater self knowledge and rapid dissemination. The consequence is that scientific design, informed consent, and ethical oversight can be short circuited by patient led “disobedience.”…

…. Patients increasingly realize that they are both statistically and literally the “power” in trials and we need to build systems that redress the imbalance. If we collectively do nothing, a phase III study might be rendered scientifically null by a critical mass of participants making intentional protocol violations on PatientsLikeMe, Facebook, or Twitter…..

…. With the new tools at their disposal patients will hold us all accountable in new and necessary ways. Patients themselves have already laid much of the groundwork; let’s ask them to continue building on these new systems together as equals….

5 thoughts on “Patient Power: trials subjects no more”

  1. I suggest you study and reflect on 'The March of Dimes', USA 1954. MS should have been presented to the public as the next grest problem after Polio.

  2. This whole situation reflects on the limited breakthroughs that have been made with regard to progressive MS and repair of damage. What do MS paitents want? To have the disease stopped, to regain some of the lost function, to get back their former lives. It's not really fair to expect those with this disease to be protesting or running campaigns. We fund researchers to get a better understanding of the disease and then to identify targets for treatment. We've set up a system which puts the patients' interests last. Reseacher are identifying currently available drugs but which will never get to market – simvastin is one example, another is minocycline. If such treaments do have a beneficial effect and have a proven safety record, there should be another way to make these treatments available. A patient should be offered simvastin, minocyline etc on the basis of informed consent i.e they accept any risks and there will be no comeback. ALS is a dreadful disease with death usually with 4 years. The idea of getting an ethics committee to take its time to consider whether a potential drug can be used is ludicrous.

  3. There is only one answer to this. The patients with clear perception want to know the CAUSE of MS. They rightly deduce that until the key pathogenic mechanism of the lesions is found, therapeutic attempts will be questionable and relatively ineffectual guesswork. One problem lurking around the current medical and scientific establishment is that because modern powerful medical research has failed to find the cause, it does not exist. MS is merely an unfortunate mixture of various environmental 'triggers' combining with a miscillaneous mixture of susceptibility genes. One day an obligatory link in the chain of events leading to MS WILL be discovered. The link will be confirmed, and then specific therapeutic, diagnostic and preventative strategies can be put in place. A consummation devoutly to be wished! Believe. It will happen!

  4. It seems that PwMS (yes I am one) need to do more to further their own cause – not easy to do but what you describe could be a way forward – PwMS need to form a pressure group or become more militant ??

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