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By Bettina Ryll

My husband’s diagnosis with melanoma and our struggle to access effective therapy challenged what I had learnt about medical research. I have since founded a patient network, becoming a vocal advocate for patient- centric drug development. Herein, I discuss some of the lessons I have learnt.

I clearly remember the first lecture about evidence- based medicine I attended. I was a third- year medical student, and after 2 years of theoretical training I was now seeing my first patients.

Scientific rigour in the form of blinded, placebo-controlled randomized clinical trials seemed to be the solution to my worries: I would be able to test whether treatments were actually as effective as hoped. The structured approach would protect me from falling prey to my own biases and my patients would be safe! I was sold.

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The personal experience that changed my views

In the spring of 2011, and without any warning, the little hard lump under my husband’s arm turned out to be a melanoma that had already spread to his lungs and spine. We were told not to hope for a cure and that any treatment would be palliative. Less than half an hour from returning from the appointment, my computer-savvy husband found what I hadn’t had the heart to tell him. “I won’t be here for Christmas,” he said.

Chemotherapy, the then standard-of-care therapy, was largely ineffective, as I knew since medical school. It was also the comparator in any available clinical trial. Friends referred to it as a ‘nocibo’, a non- effective treatment with adverse effects.

All this happened while, for the first time, not one but two new therapeutic classes were making tumours ‘melt’ in early phase clinical studies that were not even intended to evaluate efficacy — tumours like the one I watched growing daily in despair.

He did, however, live to see Christmas — thanks to clinical trials or, rather, thanks to us learning how to use clinical trials to obtain what he desired most: more time with his daughters.

Receiving a life- shortening diagnosis causes unbelievable suffering for patients and families. Withholding effective therapies in this setting, all in the name of ‘good science’, is inhumane.

It was an eye-opening experience to be at the receiving end of what I had once considered the solution to, not the cause of, suffering. Knowing that I could have been the person inflicting this experience on others horrifies me to this day and has become the driving force behind my advocacy efforts.

"Most European cancer patient advocacy groups welcome structured patient involvement and have started to systematically learn from strong patient communities that have been able to shape the drug development process."

This article first appeared on Nature.com. Read the full article

The search for new ways to design clinical trials

"In my experience, patients with melanoma join clinical trials for one of several reasons: a largely ineffective standard-of-care therapy (as is currently the case for uveal melanoma), the patient has exhausted all existing lines of
therapy, or the therapy is approved but not reimbursed in a given country. The motivation invariably is access to treatment."

"If the interest of a patient is to seek the most promising treatment, then an ethical cancer trial design needs to ensure this, ."

"Furthermore, a situation of high unmet need alters our perceptions of acceptable benefit and risk, a fact that is also increasingly acknowledged and accounted for by decision makers such as the European Medicines Agency...For a chance to survive, patients with melanoma prefer an unknown risk to a known risk with insufficient benefit, in the full understanding that the novel therapy might turn out worse."

"Most European cancer patient advocacy groups welcome structured patient involvement and have started to systematically learn from strong patient communities that have been able to shape the drug development process."

"A notable example is the community of patients with HIV or AIDS, which successfully challenged prevalent clinical trial designs by introducing novel surrogate end points, kick-started the testing of combinations of unapproved therapies and pushed for expanded access programme."

"Thanks to the Internet, nowadays patients are better educated and connected than ever before... Today’s research subjects are becoming emancipated and able to enforce their preferences. Drug development and clinical trial designs will have to follow suit... New trial designs and data sources will come with their own challenges. However, it will become increasingly hard to ignore what should have always had precedence: a patient’s best interest."

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