Rafal Swierzewski, PhD
Cancer Survivor; Associate Consultant, European Cancer Patient Coalition (ECPC), Brussels, Belgium; Expert, European Medicines Agency (EMA), London, Great Britain; Co-founder and CEO, Foundation for Patients with Rare Diseases “COCARDA,” Lodz, Poland
I was diagnosed with a rare cancer as a young student of chemistry. As a young scientist, working in the field of chemistry and biochemistry on biologically active molecules that were then and are currently used as medicines, I was never interested in cooperation with patients. Moreover, I didn’t regard myself as a potential source of experience that could be helpful in my own investigations. It was even worse: I saw no connection between my experience as a patient and my investigations directed to interactions between chemotherapeutical compounds and intracellular environment.
That personal approach changed when I was diagnosed for the second time with the same cancer, almost 10 years after my first diagnosis. It appeared that my knowledge of the nature of my own disease and the nature of chemical compounds that can be used in its treatment helped me in my fight with cancer. With my oncologists, I was able to assess my condition and treatment options, and choose the most adequate one.
Now, after the next 10 years, I know for sure how important the cooperation is between researchers and patients in all fields of medical sciences. That cooperation builds knowledge and discovers new areas for common dialog and new medicines development and analysis. That cooperation can save patients’ lives. And, due to that cooperation, patients shall be included not only in decision-making processes in such research as clinical trials, but also as authors, co-authors, and reviewers of medical publications, because we, the patients, are the basic source of medical knowledge.
Modern vocabulary includes two terms, “patient centricity” and “patient involvement,” that reflect inclusion of the patient community among other health care stakeholders as main subjects and partners for health care systems. While “patient centricity” refers to the focus on the most important link in the chain of health care services and reflects a passive meaning, “patient involvement” has become an expression of real participation of patients in different health care processes (eg, decision-making, policy shaping, clinical trials design). It’s important to notice that both terms complement each other: planning and development of “patient-centered” health care services and policies relies on patients’ opinions – an understanding of their needs and preferences, as well as those of their caregivers.
The last few years have shown that “patient involvement” signifies an active form of cooperation and interaction between patients and regulatory authorities, politicians, researchers, the pharmaceutical and medical industry, and academia at national and international levels. These interactions are poised to create improvements in the organization and functionality of health care systems, due to a more direct understanding of patients’ experiences and needs. The key element offered by patients is first-hand experience with a disease and its treatment on their overall well-being.
This experience, when carefully analyzed, can aid in determining specific conditions or broader medical areas in which a lack of technological development and/or treatment possibilities exist. Nowadays, these conditions and medical areas are defined in one term, “unmet medical needs.” The broad ocean of “unmet medical needs” indicates directions for life sciences and medical research, including the processes for new medicines development and clinical trials. Simultaneously, new possibilities are formed from or because of patients’ involvement in medical research.
Patients’ Involvement in Medical Research
Medical research focuses on a scientific problem, proposed action(s) for solution, gathering of the results of the implemented solution, and an analysis of the comparison groups between the proposed solution and other available solutions. Hence, the results and their dissemination in a scientific publication become an indicator of scientific progress and development. A publication also indicates a researcher’s work and becomes one of the most important determinants of a scientist’s career, building the scientist’s reputation and position in the research community. This is common for all areas of research, including medical.
Throughout many years, patient involvement in research was limited to participation in a clinical trial. They did not have a scientific background, and there was not a recognized role or value in patient involvement. Hence, there was no need and no expectation from any side that patients can help with study design or endpoint selection. But now, that view has changed due to technological developments in life and medical sciences, and greater interest in patients’ opinions of their needs and treatments, without which further development of “patient-centric” medical technologies seems almost impossible (eg, personalized medicine).
Patients’ involvement in health care decision-making processes and their active participation in the shaping of health care policies has created a new kind of opportunity: the involvement of patients in study design, endpoint selection, and articulation of study results. This opportunity developed from patients’ involvement in two processes: 1. regulatory, connected to medicines authorization; and 2. research, connected to clinical trials.
The evolution of patients’ involvement in research, with special emphasis on clinical trials, has a well-established historical background. Currently, patients can participate in clinical trial design and conduct in many ways:
- They can serve as members of Bioethics Committees.
- They can provide a highly specific assessment of clinical trial protocols (eg, selection of study/control groups, need of randomization or need of limitations in randomization, medicine dosing, route of administration, definition of patient-oriented outcomes).
- They can participate in safety assessments.
Patients can also be involved in the development of informational materials on clinical trials, such as general information in lay language and informed consent forms. Additionally, their insights on information on a particular clinical trial, access to care, and coordination of care can improve study design within the processes of patient recruitment, retention, compliance, and advocacy. They can also participate in the process of redaction and development of a Risk Management Plan.
Patients’ Involvement in Medical Publications
Patient involvement in clinical trial design has now evolved to open the possibility for patient involvement in medical publications as co-authors and journal reviewers. However, patient involvement in publications depends on several different factors. It should not only be based on the patient’s personal experience with the treatment and disease; the patient should also have relevant experience cooperating with research communities and a level of knowledge and literacy in understanding medical-scientific language. Such knowledge and skills inherently develop in those individuals already involved in a voluntary basis within patient communities through patient advocacy groups and associations. Development of the skills is not based on any specific courses on medical writing or reviewing of scientific publications. It depends largely on patient experience achieved through their own involvement and subjective individual views and opinions, as well as on their self-assessment of skills.
The area of patients’ involvement in medical publications is in its infancy but developed rapidly in parallel to patients’ involvement in medical research. Although both areas have great potential for exploration, it should not be forgotten that they arose from the patients’ experience with a disease and its treatment. Patient involvement in medical research and publications across the many trials that are conducted can be fairly viewed as idealistic; however, the benefits are important motivators to more actively engage patients when the potential exists.
The possibility of co-authoring medical publications (eg, clinical trial results) raises some key challenges for patients:
- Understanding the character of scientific publications, in general: their specific vocabulary, terminology and systematics, purposes, internal editorial demands, submission processes
- Understanding the composition of scientific publications (abstract, introduction, methods, results, discussion, conclusions)
- Maintaining the continuous learning process that is necessary to follow the development of medical sciences in a chosen area and contribute that knowledge to the publication
- Ensuring that ICMJE guidelines need to be followed; the patient or patient representative must be able to fulfill these guidelines
Involvement as co-authors differs from patient involvement as medical publication reviewers at journals. First of all, there are two roles that patients can have in publications: as a reviewer or as an author. It should not be misleadingly interpreted as involvement of the patient in the professional assessment of a publication. The main role of the patient reviewer is to place special attention on the level of understanding of the publication for the broader population, rather than only the research community. This need for a broader understanding and lay-language accessible publications has been stimulated by the general public due to the availability of open-access scientific journals. Consequently, it has also created a challenge for well-known scientific publishers of medical research to introduce patient reviewers that help address both needs: help researchers by providing them with knowledge on understanding of patients’ literacy; and help the general public, including patients, by providing them with scientific publications in a more understandable way.
The challenges and opportunities for patient involvement in publications are both known and unknown. What is known is that there is an opportunity for evolution in this area; it is inevitable. What is unknown is how it evolves and how fast it evolves.
 Vahdat S, Hamzehgardeshi L, Hessam S, Hamzehgardeshi Z. Patient Involvement in Health Care Decision Making: A Review. Iran Red Cres Med J. 2014 January; 16(1).
 Commission Regulation (EC) No. 507/2006
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