Pharma & Patient USA 2023

Nov 16, 2023 - Nov 17, 2023,

Humanize partnerships. Invigorate patient experience.

PED: Turning the patient voice into a dataset

Patients and pharma are teaming up to capture valuable patient experience data and they are only getting started

Get ready to learn a new word: ‘spetspatient’. Coined by Dr. Sara Riggare, a patient researcher in health informatics with the Participatory eHealth & Health Data Research Group at Uppsala University, roughly translated it means ‘lead patient’. 

It’s a term we may want to get used to as the industry comes to see the value, and increasingly the imperative, of reliably capturing what it’s like for patients to experience a condition, to live with it day to day and to treat it.

There is a growing recognition of the value of using the patient voice as a dataset in this way, says Riggare. “I see the start of a new way of thinking. Traditionally patients have been seen as helpless and lacking knowledge, and can only generate data under the supervision of an HCP or researcher.”

Patients can now become key opinion leaders (KOLs) who can work with pharma companies to identify and solve problems, she adds. “Anyone in pharma who wants to find a new solution should find the spetspatient and work with us. We can help with a lot of things.”

From collaborator to co-author

Perhaps the ultimate iteration of this trend and the most potent expression of the spetspatient concept is patients becoming co-authors on papers that explore their experience of a disease.

A Parkinson’s sufferer since her early teens but undiagnosed until her 30s, Riggare has combined her experiences as a patient with her engineering skills to undertake a masters in health informatics, and attaining her Phd in 2022. 
It may be no surprise to learn that with such a background, Riggare is herself is just such a spetspatient, co-authoring a paper on developing transferable methods for optimising medication effect.

Indeed, according to Riggare, co-publishing papers together is one of the most powerful ways to capture patient voice data. It allows other co-authors to learn about the patient perspective, which is often overlooked. “I think one of the most important pieces to transforming knowledge building is by learning together in such a way.

“The other co-authors are forced to learn about the patient perspective. Often they don’t think they need to know it but realise they have much to learn, while patients had much to learn about the other side too.”

There is much exciting patient experience data gathering going on inside pharma beyond co-authoring. 

Authentic insights

Novartis is capturing the patient perspective to help it co-create patient-relevant endpoints, co-design clinical trial protocols, and gather insights before launch. It is engaging the patient community in a consistent and systematic manner throughout the whole lifecycle, from early research and development to loss of exclusivity. 

Jean-Pierre Malkowski, Executive Director Patient Engagement Centre of Excellence at Novartis notes that in the past, patient insights were gathered indirectly through healthcare professionals or primary market research, which he argues lacked authenticity. Now it is implementing a framework that systematically gathers patient perspectives at key decision points or milestones. "It can involve both qualitative as well as quantitative evidence gathering and follows patient-focused drug development principles," says Malkowski.

When co-creating endpoints and designing trial protocols with patients, Malkowski notes that patients can provide feedback on the burden of the study, such as too many hospital visits or blood withdrawals. Patients can also provide insights on what is meaningful to them (e.g. patient-reported outcomes or PROs, and patient preferences), even if it differs from the regulatory-defined endpoint.

And it is gathered in partnership with patients, he adds. "It involves co-creation with the community. It's not to the community or for the community, it's definitely with the community. And that's been the biggest change in the last few years.”

Such patient-based evidence is valuable not only for the patient community but also regulatory submissions, health technology assessment (HTA) and access decision making. 

Others are well underway in their patient experience data journey. Patient experience mapping began three years ago at Ipsen Biopharm and it is now implementing this on a country level. It collects patient experience data through various means, including advisory boards, focus groups, insight sessions, and patient registries. 

Starting the product development cycle with a clear sense of what patients expect from an intended treatment and capturing their views along the rest of the lifecycle is now becoming an expectation, says Dr Oleksandr Gorbenko, Global Patient Affairs Director, Neurosciences at Ipsen. “We develop a comprehensive packet of evidence of the value to be delivered to patients and that should be validated by patients starting from phase 1.”

Ipsen is piloting its own cross-functional patient experience framework designed to ensure the organisation addresses the challenges involved, including identifying the right data capture methodologies, technical and digital solutions and addressing legal, privacy, ethics and compliance issues.
The mission of the Ipsen framework is to collect and consolidate patient experience data within patient experience map for each condition Ipsen works on and ultimately gather well validated data for regulatory and HTA submissions. Ipsen has also developed the concept of ‘target value profile’ to capture the value of a therapy as perceived by patients, healthcare providers, and caregivers. 

Creating robust and actionable insights from the patient voice is becoming more than a nice-to-have dataset. The importance of patient-reported outcomes for regulatory submissions and health technology assessment is growing. In the US, regulators increasingly expect such insights to be included in submissions. 

And there is already a clear link between the success rate of regulatory submissions and the quality of the patient data they include. Regulators are less likely these days to trust statements of comparative effectiveness within submissions and more likely to assign weight to patient experience data as a part of Real-World Evidence (RWE) if it is considered robust and trustworthy.

The challenge is how to gather robust data sets that have a value for R&D, approvals and treatment outcomes. 

The problem hitherto has been that pharma has gathered patient experience data somewhat inconsistently, in a fragmented and episodic way and for a range of different uses and often without rigour, says Gorbenko.
“There is a lack of consistency. Any scientist is looking for robust data but it needs to be robust and it isn’t regulated so far. We don’t know what the expectations are from HTA agenciess and regulators in terms of what format and content they would like to see.”

While the real-world evidence that patients generate is far richer than the in many senses ‘artificial’ data from trials, categorising and gathering it and synthesizing robust insights from it is not easy, says Gorbenko. “It’s a big challenge for all of us to have a robust methodology to sort out this data.”

The way ahead

Various industry initiatives are underway to develop best practices here. The FDA’s 2020 Patient Focused Drug Development Guidelines offers useful guidance on how to interview patients, how to collect data and what format are considered robust, says Gorbenko.

The National Health Council’s Patient Experience Mapping Toolbox, a set of publicly available resources to help researchers engage and document patients’ experiences before during and while living with a diagnosis. The Toolbox aims to help researchers capture patient experience data more holistically and in a standardised way across chronic diseases. 

The IMI PREFER project, meanwhile has developed recommendations for how and when it is best to perform and include patient preferences in decision making during the medical product life cycle. Two powerful uses cases here include involving patients in choosing patient-relevant endpoints, showing which characteristics of a medical  product or disease are most important to patients (qualitative), and how much they matter (quantitative), as well as the acceptability to patients of trade-offs between various medical products. 
As the gathering of data on the patient voice gathers pace, every company is going to have to develop such capabilities to robustly capture the patient voice and so the best way to make progress is to develop guidelines together, says Gorbenko. “This is a multi-stakeholder approach. It would be ridiculous if each company goes its own way.”
Patient organisations also have a role to play here to collect data and so partnering with them will be vital, he adds. “I strongly believe patient organisations are the best source and consolidators of such data and communicators of it.”

Patients and pharma also need to work together to develop the skills needed to collaborate fruitfully. Patients can even be helped to master the methodologies and other skills needed to co-author a paper, even if they are not researchers or medics by training, adds Riggare. “With mentorship and time, for sure we can all learn. More people than we think are capable of doing this.”


Pharma & Patient USA 2023

Nov 16, 2023 - Nov 17, 2023,

Humanize partnerships. Invigorate patient experience.