Initial efforts to commence patient engagement initiatives across Novartis were greeted with both enthusiasm and scepticism, writes David Parrish, Patient Engagement Early Research and Dr Francesco Patalano, Head of Center of Excellence, Patient-Reported Outcomes at Novartis, and Laura McKeaveney, Global Head of Patient Advocacy with the Novartis Group.

Pharmaceutical companies have been on a steep learning curve when it comes to embedding patient engagement at all stages of the drug development process. A number of representatives from Novartis outline some of the challenges they faced along the way.

David Parrish explains that efforts to engage with patients at the very earliest stages – long before trials had commenced – faced issues such as buy-in from colleagues.

“Upholding the Novartis Commitment to Patients and Caregivers, when we initiated the creation of patient engagement strategies for projects that were transitioning from the bench to clinical trials, we faced both a great degree of enthusiasm for the patient but also scepticism surrounding creating any potential impact at this early stage,” explains Parrish.

Despite this, the organisation ploughed ahead. The first step was listening, Parrish says, and Novartis was quick to embrace multiple tools for collecting and evaluating patient insights; from social media listening through to small patient panels, where all aspects of clinical trials were discussed at length.

“Our first patient panel was for amyotrophic lateral sclerosis (ALS) in which we asked patients to share their journey and to evaluate the components of the proposed protocol. The feedback covered everything from critiques on inclusion/exclusion criteria, preference for remote testing, impact of physical limitations to participation and preference for a short study duration,” he explains.

Immediately it was obvious that patient insights had the potential to be a game-changer: “Some of this feedback was the opposite of what we received from key opinion leaders,” admits Parrish.

Based on this patient feedback, Novartis made significant changes to both the proposed study and site activities. By the end of 2019, they had completed their fifth patient panel discussion – all of these before the first dose in the first study in patients.

Dr Patalano outlines how Novartis set about gathering structured patient insights to drive the Patient Reported Outcome (PRO) strategy in Chronic Obstructive Pulmonary Disorder (COPD).

“Our Commitment to Patients & Caregivers calls out the need to listen, respect and understand the patient community perspectives,” he says. “So we have asked ourselves, what are the most patient relevant symptoms in COPD?”

Although COPD is a well-known disease with many efficacious drugs available, Novartis’ goal was to take a step back and ask if patients are happy with these treatments and/or their resulting quality of life.

“Our approach has evolved and today we consider it essential to challenge our beliefs and understand the patients view on the disease. What matters to them? What are their patient relevant outcomes? Which symptoms are most bothersome?

Novartis thus conducted extensive search in the literature as well as social media, and carried out interviews with patients, both online and in person. The organisation is now asking patients, as part of a formal Patient Preference study leveraging  PREFER, an IMI-funded project, what are the desired effects from a new drug, and how they would rank them. 

Similar to what was observed in the example above, the patient perspective was markedly different to conventional wisdom in this area.

“The result of this work has given us a different picture on the disease, as seen from the patients’ point of view,” says Dr Patalano. “The integration of these insights is now leading to the selection of patients’ meaningful endpoints and the implementation of appropriate Patient Reported Outcomes (PRO) measurements within the clinical program for the development of a new drug in COPD”, he adds.

Given the positive results of these patient engagement endeavours, Novartis is keen to continue this work. Parrish says: “We continue this approach of building on patient insights and knowledge to help create patient centric strategies for a substantial breadth of diseases covered in our early portfolio.”

Laura McKeaveney explains that devising a approach to patient engagement is a priority for the organisation notably shown by the strong involvement of Novartis in pre-competitive endeavours such as IMI PARADIGM – towards a favourable framework- and IMI PREFER – aiming to better design, conduct and use patient preference studies in decision-making processes- and they both go hand in hand.

“We continue to strive to build a systematic and consistent approach to the inclusion of patient insights and patient involvement in our key decision-making processes, as seen by the examples above.”

She acknowledges that this is a major change management exercise, not just within the company but within the entire healthcare system. 

“We are excited and optimistic about the output of IMI PARADIGM, to provide industry-wide guidance on inclusion of patient insights into the research and development machine of the industry. It will enable all players in the healthcare ecosystem to have a shared opinion on how to make this a 2020 and beyond reality. Now that we have the guidance, there is no excuse not to do it.”

Editorial note:

To help you embed patient engagement across your organisation, look out for the PARADIGM Recommendations on required capabilities for patient engagement* that can help your team is prepared for engagement. Use the Monitoring and evaluation framework* to help you set the parameters to evaluate the progress and evidence success from the get-go.

* All PARADIGM tools will be released during the summer of 2020. Follow the information on our website. The final names of each tool might change.

This is the second article of the mini-series of six articles. Find the other articles from here.