Jason Gardner, PhD, ISMPP CMPPTM, CMC CONNECT, a division of McCann Health Medical Communications, Macclesfield, UK; Lynn Hagger, PhD, AstraZeneca Pharmaceuticals, Gaithersburg, MD, USA; Alex Chin, PharmD, RPh, AstraZeneca Pharmaceuticals, Gaithersburg, MD, USA; Anna Georgieva, MD, PhD, ISMPP CMPPTM; Excerpta Medica, Amsterdam, The Netherlands
Few will have failed to notice the wave of adoption, integration, and implementation of “patient-centricity,” “patient involvement,” and “patient engagement” by the pharmaceutical industry.1 Currently, the involvement of patients in medical research is becoming more common, and the area of medical publications, while making important moves towards this goal, is still behind the curve compared with other stages of the drug development process. A call-to-action is provided below for next steps to address this gap.
What are the Current Practices for Involving Patients in Medical Research?
The patient community is effectively the end-user for all pharmaceutical and medical device products; so, it makes sense that patients should be involved as partners throughout the clinical development process.
Some leading pharmaceutical companies have published their commitment to patient involvement in research.2-4 Others are not disclosing their commitment so clearly, although many are investing substantially. The benefits of patient involvement include5:
- Direct insights from patients that increase confidence of industry sponsors developing study concepts and protocols.
- Increased relevance of clinical studies to the patient, greater willingness to participate in the trial, improved protocol adherence, and better overall experience.
- Selection or development of clinical trial endpoints that demonstrate meaningful value to patients. This results in greater efficiency and effectiveness of clinical plans, supporting regulatory approval processes and Health Technology Assessments (HTAs), thus helping to bring potentially beneficial new treatments into clinical practice.
- More effective dissemination of clinical study results to medical and patient populations leading to better understanding of the disease, compliance and adherence to medication, adverse event (AE) management, and ultimately better disease management.
There are many points in the clinical research development cycle in which patient involvement can add value; see Figure 1 below and Geisler et al 2017, which provide an excellent “Practical Roadmap” for patient involvement in medicines research and development.5
To outline an example of how one pharmaceutical company has involved patients in its clinical research: AstraZeneca has an established Patient Research Framework within Clinical Operations that incorporates patient insight as early as possible into protocol development and engages patients throughout each study. Strategic, qualitative insight about clinical study design is initially gathered from members of AstraZeneca’s Patient Partnership Program. This program provides an open line of communication between AstraZeneca employees with functional expertise and groups of patients in a given disease area, where they can learn from each other and ensure that the patient is at the center of scientific strategies and studies. This allows for more focused clinical study designs and data collection to reflect what is important to (and reasonable to ask of) the patient, as well as improved retention in clinical studies and protocol adherence. Furthermore, patients involved as participants in clinical studies today are being better informed on drug access and future research opportunities at study completion.
In establishing a patient partnership program, companies should be aware of the distinction between patients as an audience and patients as partners. In addition to their own experiences as patients or caregivers in a given indication, patient partners typically exhibit good communication skills, and are able to interpret scientific information and relate it not only to their own situation but ideally to those of the broader patient audience. Individuals who fit this model may be difficult to find; as such, patient engagement organizations are becoming established that specialize in identifying patients and facilitating patient input (eg, https://patientfocusedmedicine.org/). Companies should also consider establishing an appropriate contracting and reimbursement structure for the patient partners; this is a new type of expertise being provided to pharmaceutical companies that will require careful discussion to reach a fair-market-value framework.
What is Required to Drive Patient Involvement in Medical Publications?
It has become widely recognized that patient input into medical publications is of great value. It is clear that patients are not necessarily experts in medicine, trial design, or statistics, but that should not stop them from being engaged as experts in their own disease. A way forward needs to occur to facilitate patient participation in medical publications by helping them understand complex medical topics, discuss which elements of the research are important to them and why, and integrate their input into publications in a way that is relevant for both medical and patient audiences.
Medical publications activities in which patients could be involved include publication planning, reviewing/contributing to draft publications, or authoring/co-authoring publications (Figure 2 below)6. However, this practice is presently not commonplace. Why is involvement of patients in medical publications still in its infancy when it is well established in other aspects of clinical research?
More and more, patient involvement is becoming professionalized. Regulatory bodies, HTA bodies, and academic institutions are including patient representatives in their decision-making process due to their unique perspective on the day-to-day impact of disease and treatment. In all such cases, the outputs and relevance to patients are clear. Perhaps the delayed adoption of patient involvement in publications is a result of a persisting lack of clarity of the end-users and identifying the optimal format for medical publications to meet the needs of patients.
Historically, health care professionals have been the target audience for medical publications. However, there is also a clear demand from patients for medical information through credible channels. For example, in a survey of 100 US patients and 50 US caregivers of patients with atopic dermatitis/eczema, 45% of patients searched for and read peer-reviewed medical publications online; and 62% of these patients found the publications by Google search.7
Access to disease and treatment information through medical publications may empower patients to take control of their conditions,7 have more informed discussions with their health care professionals,7 and ultimately obtain better outcomes. But, with a self-guided search for medical information comes the risk of misinterpretation or encountering false or incorrect information.
Patients consider peer-reviewed medical journals as a trustworthy source of information, nearly as much as their treating physicians.7 Some journals are already encouraging patient involvement and are updating author guidelines accordingly. For example, authors submitting manuscripts to The BMJ are now required to disclose how patients were involved in the study design and the communication of results.8 The BMJ also appoints patients to journal editor and peer-reviewer roles, as outlined within its “Patient and Public Partnership” strategy.8 However, examples like this are few in medical publications. Relatively few journals provide an open-access facility to engage with patient/caregiver audiences. Any available plain-language summaries are often difficult to find,9 and there is currently no standard facility on PubMed to help drive uptake.
So, what’s the position of pharmaceutical companies? If patients and non-specialists become accepted audiences for medical publications, companies may be reluctant to fund patient involvement in materials developed for patients if there is a risk of it being regarded as direct-to-patient promotion about their products. How can companies avoid plain-language or graphic summaries being perceived as biased cherry-picking?
What are the guidelines included in GPP3 and the International Committee of Medical Journal Editors (ICMJE) recommendations? Currently, there are no universal guidelines to address patient involvement and how medical publications may be extended to reach non-specialist audiences. But, importantly, there is also no guidance suggesting that this should NOT be done.
The authors of original medical research, acting within a peer-review process, involving oversight and governance from editorial boards of credible scientific journals, can serve as the best conduits to summarize their findings for non-expert audiences. To do so must involve patients and/or caregivers, as noted above (Figure 2).
In addition, the whole dynamic of medical publications requires rethinking. Here are some suggestions to start driving change (also see Figure 3 below for a graphic representation):
- National Institutes of Health (NIH) to work with publishers to drive awareness of publicly available plain-language summaries on PubMed
- Journals and publishers to consider:
- Including plain-language summary sections in the structure of all original-research articles
- Initiating open-access (or at least reduced-fee) versions of their journals that specifically target broader audiences by publishing plain-language summaries or full plain-language articles (with links to the original research articles)
- Medical congresses to promote the inclusion of abstract plain-language summaries (APLS) to accompany poster presentations
- Authors and sponsors of medical publications to consider:
- Mandating inclusion of plain-language summaries with every original-research article (eg, via updates to company standard operating procedures)
- Targeting publications to journals that allow plain-language summaries
- ICMJE, ISMPP, and the GPP4 Steering Committee10 to consider providing recommendations and guidance for the medical publication community to follow (eg, recommending plain-language summaries are always written; a process for getting them published; guidance on style/complexity/format; any disclaimer text)
Action at any of these levels is likely to trigger a cascade of effects that facilitate and increase the involvement of patients in medical publications, as well as an expansion of medical publications to reach broader audiences. In the meantime, medical publication professionals should continue driving more creative and innovative solutions to increase reach and enhance impact of medical publications with the involvement of patients/caregivers. The potential to make plain-language summaries available or discoverable through advocacy or patient-focused platforms should also be further investigated.
Patient involvement in medical research is beneficial at many levels. If patient involvement in medical publications brings benefits to health care professionals by helping them understand a patient’s perspective on the data, then this alone seems to justify their involvement. But so much more could be achieved if mindsets are changed regarding how to communicate about medical publications and to whom. Medical publication professionals have a responsibility to facilitate the appropriate communication of medical knowledge and are in the perfect position to drive change through the effective integration of patient partners in the medical publication process.
- Swierzewski R. The Evolution of Patient Involvement in Medical Research: A Patient’s Perspective. Available at: https://ismpp-newsletter.com/2018/07/06/the-evolution-of-patient-involvement-in-medical-research-a-patients-perspective/. Date accessed, 04 February 2019
- AstraZeneca: Yeoman G, et al; Defining patient centricity with patients for patients and caregivers: a collaborative endeavor. BMJ Innovations Published Online First: 24 March 2017
- Pfizer: https://www.pfizer.com/files/about/Patient_Engagement_and_Involvement_in_RnD_April_2018.pdf
Date accessed, 04 February 2019
- Roche: https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/patient-as-partner.htm
Date accessed, 04 February 2019
- Geissler J, Ryll B, di Priolo SL and Uhlenhopp M. Improving Patient Involvement in Medicines Research and Development: A Practical Roadmap. Ther Innov Regul Sci. 2017;51(5):612-619. https://doi.org/10.1177/2168479017706405
- Figure adapted from: Georgieva A and Sloane D. Delivering real patient value. PMLiVE 2018. Available at: http://www.pmlive.com/pharma_intelligence/delivering_real_patient_value_1214427
Date accessed, 04 February 2019
- Georgieva A, McNamara M, Nuottamo N, Lukose A, Moreland N and Bijvank S. Patients read peer-reviewed medical publications. Poster presented at ISMPP 13th Annual Meeting 2017. Available at: https://ismpp.memberclicks.net/assets/docs/Education/AnnualMeeting/13thAM/13AMPosters/29_poster_georgieva_etal_13am.pdf. Date accessed, 04 February 2019.
- The BMJ. Patient and public partnership. Available at: https://www.bmj.com/campaign/patient-partnership. Date accessed, 04 February 2019.
- FitzGibbon H, King K, Magee R, Piano C, Wilk C and Gaskarth M. Where are biomedical research plain-language summaries (PLS)? Poster presented at ISMPP 2019 European Meeting. Available at: https://ismpp.memberclicks.net/assets/docs/Education/EuropeanMeeting/2019EM/Posters/20Poster.pdf. Date accessed, 04 February 2019.
- GPP4 Twitter hashtag. Available at: https://twitter.com/hashtag/GPP4. Date accessed, 04 February 2019.