PATIENTS HAVE IT RIGHT

Good clinical practice emerged to put patients’ safety above any other objective in clinical research. Yet, until recently, patients were not part of the decision makers in the design of the clinical development programs, traditionally looked through the lens of scientists, primarily focused on a mechanism of action. Anyone working in clinical research can acknowledge patients always stated their feelings on the way protocols and tested treatment options accommodated their needs or not. But their voices were like shouting into an echo chamber. Sponsors, clinical teams, site staff, and other professionals have heard hundreds of conversations from trial “subjects” but hardly incorporated their input in presenting the case studies and insights to decide real actionable takeaways. There is a need and an advantage to acknowledge trial participants as people, with a disease or condition being explored, eager to contribute, and to get involved in the workstreams. “To better design our trials, research questions and the outcomes studied must be prioritized by patients. To more efficiently recruit, retain, and ultimately deliver clinical trials we must understand the patient perspective.” 1 We need patients’ perceptions not only to better design trials but also to be better prepared to characterize and support new drugs for health authorities and payers.

Patient centricity journey started at the end of the road. It became obvious that registered drugs and devices that didn’t fit patients’ needs or imposed unbearable burdens were doomed to fail. Patient centered care brought patient judgment to the top of the decisionmaking process. Knowing patients’ main reason for the visit, concerns, and need for information emerged as vital. Diseases signs and symptoms happen in particular patients with individual emotional needs and life issues. Treatment compliance improves when management is mutually agreed upon. And the relationship between the patient and the doctor is enhanced. 2

Evidence of patients’ valuable contributions sneaked into clinical research. Patient reported outcomes have been a common tool integrated into clinical research for a long time now. But “the patient’s perspective is often overlooked or poorly described in the development of PROMs.” 3 This is changing. For example, The International Consortium for Health Outcomes Measurement included in their international working group of experts and researchers, patient representatives to define a standard set of outcomes and riskadjustment variables.

Patients’ participation in medical events is also increasing. Every day more, patients make up a substantial part of the speaker faculty. Their contributions in presentations, panels, and roundtables are being more and more appreciated. Patients’ presence, optimize the integration of their insights in program design, improve relationships, drive improved health literacy, and ultimately, transform outcomes. Derived from the present pandemic, virtual communications have rapidly evolved and even technology skeptical persons, have had to dive into the Information and Communication Technologies (ICT) world. It may be an opportunity to advance patient-centricity evaluating how it has progressed during the crisis. It is a chance to include patient insights and best practice learnings into re-emergence strategies. Think about how digital support programs can be designed for and with patients. Revolutionize patient outcomes so patient voice can be elevated at every step of product development. This approach needs to comprise every stakeholder in the process as illustrated below.

Making patients partners throughout trial design will help to diversify trial participation, increase retention, and improve compliance among other positive effects. Scaling patientpreferred research and considering patient burden, will result in better-designed products with preferred efficacy.

Drug development should be flooded by likeminded peers who believe in the importance of the patient voice and active participation. “Finally, to better characterize the benefits and risks of our products for health authorities, we must understand how patients perceive the burden of the disease and the standard of care. Patients play the most important role in clinical trials, and therefore are in a position to help make a difference.” 4 The earlier we bring patients into drug development, the better treatment options will improve in speed, cost, and quality.

Rosseti, L. Patient Centricity is Essential to Modern Day Drug Development. IFPMA News Center. 2015 Mar 23. https:// www.ifpma.org/science-matters-centre/patient-centricity-isessential-to-modern-day-drug-development/

Steward, M. Towards a global definition of patient centred care. BMJ. 2001 Feb 24; 322(7284): 444–445. doi: 10.1136/bmj.322.7284.444

Peeters G, Barker AL, Talevski J, et al. Do patients have a say? A narrative review of the development of patient-reported outcome measures used in elective procedures for coronary revascularisation. Qual Life Res. Published online 2018. doi:10.1007/s11136-018-1795-6 Rosetti, L. op cit

Thor Nissen

Doctor, graduate in Clinical Pharmacology and MBA in Quality Management. He has managed to collaborate for the development of medicines in world leading pharmaceutical companies both in medical matters and in clinical research in new drugs, regulation, marketing and sales departments.