The power of patient perspectives in the publication of real-world studies

Patients have a unique first-hand insight into their condition, which can be a valuable asset in the design, delivery, and publication of real-world studies, as Envision Pharma Group’s Dr Dawn Lobban and Vitaccess’ Dr Catherine Bottomley discuss.

The value of championing the patient voice throughout the medicine development journey is evident. Patients have a unique understanding of their condition and can provide valuable insights into lived experience, unmet needs, and treatment impact, and yet, historically, detailed insights on the patient perspective have been largely lacking in data generated from clinical trials.

Amid growing interest in real-world evidence (RWE), generated outside of a clinical trial setting, acceptance and appreciation of the patient voice is shifting. Now, life science companies are actively exploring ways to work in partnership with patients to improve the design, delivery, and analysis of real-world studies. But whilst efforts to champion patient engagement in the early stages of study and protocol design have been widely demonstrated, few have explored the potential of extending the patient perspective to authorship and publication.

Patients have much to offer to the development and publication of real-world studies, yet, there is much to be done to improve awareness of the contributions these individuals can make to the peer-reviewed literature.

“The research that we’re doing is either for patients, about them, or both,” says Dr Catherine Bottomley, chief scientific officer at Vitaccess. “Ultimately, they’re the people who know their condition best and can tell us what really matters to them.”

Patient involvement in real-world data generation

To understand how patients can contribute to the publication of RWE, it is important to consider the broader role of patients in data generation and analysis. Existing guidance for where patients can (and should) be involved in randomised, controlled trials for drug development advocates for patient involvement in the initial stages of identifying research priorities and protocol design, through to conducting research and communicating results.

Many of these activities are also applicable to studies outside of the clinical trial setting. However, as Bottomley notes, RWE studies can offer more flexibility for patient involvement.

“Randomised controlled trials are governed by best practice guidance and standardised recommendations, particularly when it comes to the patient-reported components of them, and so you have potentially limited ability to be able to implement changes that patients might suggest during the study design process,” she explains. “For example, they might identify that a range of particular outcomes are important to them when they start taking a treatment, but within the constraints of that clinical trial, there’s no space to ask those questions.

“In the real-world setting, there’s often more flexibility in terms of study design, scheduling data collection, and time points, which means that you can ask additional questions and collect additional information.”

Involving patients right at the beginning of the study planning process can significantly improve study design and delivery. The perspective offered by patients can help investigators to broaden their understanding of patient needs and consider the real-life implications of taking part in the study for the individuals involved, allowing researchers to resolve issues and improve study designs before the study opens for recruitment.

“By involving patients right at the start, you have the potential to develop better study designs and protocols that consider patient needs,” says Bottomley. “And consider those protocols in real-life scenarios, asking, ‘would you actually want to wear a tracker whilst you sleep? Are you willing to answer three questionnaires in a row that ask you about the impact of treatment on different areas of your life?’”

“Often when we’re designing protocols, we make assumptions about what participants are or are not willing to do. Unless we actually involve patients in those discussions, we might be making some really broad assumptions that are then going to result in people not wanting to take part.”

For Bottomley, it is important that project teams approach patient collaborations as an opportunity to foster meaningful partnerships that benefit both studies and patients. Just as researchers provide an expert scientific understanding of the study topic, patients are experts in understanding the reality of managing a condition. As such, their input should be respected, even if their perceptions do not align with those of investigators.

“It’s really important to spend time developing relationships with the key patient stakeholders,” she says. “We always suggest making a plan or an agenda for the patient involvement throughout the study, with all the details of activities, time commitments, and related reference material.”

Encouraging and facilitating patient authorship

Whilst the value of patient contributions throughout the design and delivery of real-world studies has been increasingly recognised in recent years, when it comes to authorship, patient perspectives have been rare. This does appear to be changing, as pharma companies acknowledge the unique insight that patients can bring to the publication of RWE.

According to the International Committee of Medical Journal Editors, to be eligible for authorship, patient partners must meet all four of the following criteria:

Substantial contributions to the conception or design of the work; or the acquisition, analysis, or interpretation of data for the work; AND
Drafting the work or revising it critically for important intellectual content; AND
Final approval of the version to be published; AND
Agreement to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

“When patients are involved in designing questions to really get to the heart of the issue being explored, or in analysing the patient insights generated during a study, they can easily fulfil that first criterion,” explains Dr Dawn Lobban, global lead, patient partnerships at Envision the Patient. “Then, if given the opportunity to be involved from the outset as a co-author, they can actively participate in drafting, critically reviewing, and approving the final version to fulfil the second and third criteria.”

She continues, “Building on this foundation, patients can proceed to the fourth requirement, becoming accountable for all aspects of the work and appropriately investigating and resolving any challenges to the publication, along with their co-authors.”

There are many benefits to be found in partnering with patient authors. Notably, as patient representatives, patient authors can ensure that the needs of their community are considered, keeping the focus of a study publication relevant and applicable. Patient authors can also ensure that the language and format used can be understood by both experts and laypeople.

“When we write medical publications, we write them for a scientific audience, but we also want them to be understood by a wide range of people,” says Lobban. “Patients often ask very insightful questions that can highlight areas of a publication that are perhaps not obvious to non-expert healthcare professionals. Patient co-authors may also consider publication enhancements, such as plain language summaries, which are designed specifically to broaden the reach to a wider non-specialist audience, including patients.”

Despite the benefits, there are barriers that can affect patient partners and their ability to collaborate as co-authors. Whilst scientists working in the industry are likely well-versed in the publication process and specific scientific terminology, patient partners have differing levels of understanding, which can be daunting for potential co-authors. Moreover, patient authors have to balance their participation in the publication with their own personal commitments, including managing their condition.

For Lobban, it is important to provide patients with the tools they need to optimise their potential as authors. Increasingly, the publication process and authorship are being included in training services designed to support patients looking to get involved in medicine development, such as the course developed through a collaboration between Envision Pharma Group and the European cancer patient advocacy network, WECAN.

Supporting patient engagement and authorship through partnerships

Whilst the recently updated Good Publication Practice guidelines (GPP 2022) broadly support patients as authors of company-sponsored biomedical research, further work is needed to optimise patient engagement in the generation and publication of RWE.

“There are still barriers to be broken down,” explains Bottomley. “It’s our responsibility as researchers to bridge the gap between somebody saying, ‘I want to take part’, and them actually being able to sit down with the research team with the knowledge and understanding of the study to contribute in a way that’s going to be beneficial.”

Understanding the needs, knowledge, and abilities of patients as both stakeholders in real-world studies and as publication authors is a central step towards developing respectful and symbiotic partnerships between patients and investigators. It is important to remember that engaging in real-world study and authorship activities usually occurs within the broader context of a patient’s daily life. As such, the industry should prioritise supporting these patients throughout the study and publication process, to ensure that incorporating research responsibilities around work, friends, family, and managing their own health is as straightforward as possible.

Fostering a foundation of respect and support throughout the study and publication process is particularly important as regulators call for the broader inclusion of real-world patient perspectives in research submissions and evidence packages.

As Bottomley notes, “Organisations are understanding and seeing the benefits of involving the person who’s actually suffering from a condition and appreciating and respecting the fact that they have something of vital importance to contribute.”

“What we’re advocating for is that patients can, and often do, qualify to be authors in the same way that other researchers and healthcare professionals qualify to be authors. With support and evidence-based resources, we can ensure that the patient voice is heard in the medical literature,” concludes Lobban.

Dr Dawn Lobban, global lead, patient partnerships at Envision the Patient.

As global lead, patient partnerships, Dawn Lobban heads up the Envision the Patient team within Envision Pharma Group. With a strong background in medical affairs, Dawn is particularly interested in optimal patient involvement in medicine development. Through active participation in key working groups, including ISMPP (International Society for Medical Publication Professionals) and PFMD (Patients Focused Medicine development), Dawn works hard to explore effective ways to ensure effective patient engagement. Her research and opinions are published in a number of abstracts and articles.

Dr Catherine Bottomley, chief scientific officer at Vitaccess

Catherine Bottomley is a PhD pharmacist with expertise in Patient Centred Outcome (PCO) research. She is currently chief scientific officer at Vitaccess, a patient-focused consultancy organisation offering innovative solutions to patient and carer reported data collection in real world settings. Catherine has overall responsibility for the pharma sponsored research studies conducted by Vitaccess including patient-reported tech-based registries, qualitative research, Patient Reported Outcome Measure (PROM) development and implementation, and observational studies. Catherine has over a decade of experience working in PCO and RWE roles across a variety of pharmaceutical consultancy settings, spanning associate researcher through to director.

Envision Pharma Group is a leading provider of evidence-based communication services and industry-leading technology solutions (iEnvision) that have applicability across many areas of medical affairs and related functional responsibility. Envision provides services and technology solutions to more than 90 companies, including all of the top 20 pharmaceutical companies.

Vitaccess

Innovators in real-world research, we connect biopharma to patients and caregivers on any device, worldwide – harnessing our scientific expertise and cutting-edge digital technology to generate real-time, real-world insights through the powerful combination of our Vitaccess Real™ digital platform and expert consultative acumen. Born in Oxford, we have presence across Europe and the US.

We are health economists, researchers, eClinical health experts, data scientists, software developers, linguists, and medical writers, all committed to insightful digital research. We are passionate about inclusivity and open ways of working. We believe the more voices and views we listen to, the better we can make real progress, together.

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The power of patient perspectives in the publication of real-world studies

Patient involvement in real-world data generation

“In the real-world setting, there’s often more flexibility in terms of study design, scheduling data collection, and time points, which means that you can ask additional questions and collect additional information.”

“Often when we’re designing protocols, we make assumptions about what participants are or are not willing to do. Unless we actually involve patients in those discussions, we might be making some really broad assumptions that are then going to result in people not wanting to take part.”

Encouraging and facilitating patient authorship

Supporting patient engagement and authorship through partnerships

About the interviewees

About Envision Pharma Group

About Vitaccess