Patient-centric drug development

By Pekka Simula 17 September 2017

Patient-centricity is one of the new catch phrases of the pharmaceutical industry. The ‘Big Pharma’ emphasize how patient-centric they are; drug development startups boast having been founded around the very idea of patient-centricity. Even our work at Herantis Pharma, when awarded the Nordic Star 2017, was praised for a truly patient centric approach. That’s great, isn’t it! But if you think about it, our industry develops drugs for patients… so aren’t we all patient-centric by definition? Is this new patient-centricity just an attempt to polish the greedy reputation of the pharmaceutical industry?

I had an eye-opener recently when listening to a GlaxoSmithKline presentation on patient-centricity. There was an anecdote where a clinical developer had been asked a very simple question about the clinical study he had designed: “Would you participate in this study?” The answer was ‘No.’ So the obvious follow-up question: “Why on earth did you design a study in which you wouldn’t participate?”

That left me speechless: While it sounds totally obvious, with some experience in drug development I realised that this happens very, very easily. Preparations for clinical studies take years and require taking into account multiple viewpoints. Scientific factors, regulatory requirements, ethics, feasibility of executing the study, cost efficiency, target product profile, and a hundred other factors. Though the patient viewpoint is definitely considered it may easily get lost among all the other details. Even an well-meaning Ethics Committee might, in their efforts to protect the patients, actually forget what the patients would want. If patients are not involved in each step of the clinical study design the result can be a clinical study that doesn’t exactly attract patients. Or even worse, the result could be a marketed drug, which the patients don’t want! One example is a cancer drug with disappointing sales after winning market approval. The administration of this drug is relatively tedious and only performed at specific locations, whereas other treatments are available too. Apparently the targeted patients have largely preferred an alternative treatment, which doesn’t disrupt their peaceful retirement that much. Lesson learned: Drug developers need to share their goals with patients in addition to mastering the science and regulations.

Patients are also more and more active and demanding. Many patients truly understand the scientific background of their disease and treatment options. And they understand that each patient is unique. The traditional “one-size-fits-all” healthcare is not acceptable such patients who also want to be in control of their health. They don’t want drugs developed to please statistics; they want personalized medicine.

AstraZeneca has contributed to the publishing of the first collaborative definition of patient centricity. This definition contains five key elements. Four were already covered above: Inclusiveness, shared goals, patients in control of their own health, and working in partnership. The fifth must be implemented in the daily practices of drug developers: “Working in a way that shows respect, compassion, and openness.”

While small drug development companies envy the vast resources of the Big Pharma, this is probably an area where we have the edge. Our development teams are contacted directly by patients on a daily basis (if we weren’t, we should ask ourselves why not!). We are constantly reminded on what is important; we never forget that patients all over the world are anxiously waiting for our results. And the needs of the patients are heard by the right people, unfiltered.

Big Pharma are always looking for partnerships with innovative small companies to strengthen their own development pipelines. Maybe there could also be some partnering benefits in patient centricity.

Herantis is actively developing two drugs aiming at breakthrough in unmet clinical needs: CDNF for Parkinson’s disease (PD), and Lymfactin® for Secondary lymphedema (LE). Respectively, Herantis is a member of two patient advocacies: The European Parkinson’s Disease Association EPDA, and Lymphatic Education & Research Network, LE&RN.

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