However, there are still many obstacles faced by investigators in achieving effective patient involvement in research
The role of patients in sarcomas, and more broadly in rare cancers, has evolved over the years because the disease rarity has been – and still is – an opportunity to seek new ways of patient advocacy. Today, patient advocacy goes beyond a simple action of raising awareness among the general public and institutions with regard to a specific group of diseases/patients, aiming to influence the decision-making process in a multistakeholder context, so that patients and other players can find adequate solutions together.
Conceptually, ‘awareness’ has turned into ‘partnership’, bringing attention to the relationship between patients, researchers and other stakeholders, but also requiring a rethinking of the existing hierarchies in healthcare and a concrete shift in research culture. Patients as partners means, in fact, that cancer care should be more open to innovation, looking for new business models and approaches to advancing patient-centric options of care. For example, in academic research, innovative grant funding approaches requiring a patient-involvement/engagement plan as part of every grant application could potentially overcome some of the main hurdles affecting patient involvement in trials.
But is a true partnership between patients and oncologists an attainable goal? Lack of knowledge about how to involve patients and who exactly should be involved, fear of losing control over the research process and the need for robust methodological approaches to incorporating patient input are some of the main obstacles faced by investigators in achieving effective patient involvement in research.
Although in 2014 the European Medicines Agency (EMA) recommended the implementation of patient reported outcomes (PRO) measures in oncology clinical studies and incorporation of PRO endpoints into protocols at the earliest stage, their use remains suboptimal. A recent EMA reflection paper for the development of new ICH guidelines aims to provide a globally harmonised approach to including patient perspectives in a way that is methodologically sustainable both for industry and regulatory authorities.
Structured frameworks, tools, guidelines and checklists to involve patients in research more efficiently have already been developed and put into practice. A recent systematic review suggests a taxonomy of five main framework categories, emphasising that there is not a one-size-fits-all framework, rather a range of evidence-based resources that stakeholders can and should use to co-design their own research (Health Expect. 2019;22:785–801).
Patients are also exposed to concerns in taking part in research, including a lack of adequate preparation in the scientific domain and limited resources for training, which may vary from one subgroup to another. The word ‘patient’, in fact, should be used as an umbrella term to describe a heterogeneous group of stakeholders including individual patients, patient experts, patient advocates, patient organisations and patient advocate organisations. Each of these may contribute to research in a unique way, depending on the particular research area, project or research question and, therefore, on the research strategy.
But even though including a variety of perspectives in research can be beneficial, the question of who exactly should be involved and why, should be addressed critically. Patient organisations can play a central role in helping researchers choose who to involve, by identifying patient representatives, providing support (information and education) to help patients participate in the research process and providing access to patient networks. This is why the EMA asks for support from organisations and patient organisations to identify patients to be included in the Register of Patient Experts for regulatory issues in drug development.
When I started out as a patient advocate fifteen years ago, the rare cancer world was a sort of wasteland. The policy context has significantly advanced over time, thanks to vigorous patient advocacy, the dedication of healthcare providers, political commitment and industry innovation. But in sarcomas, there are still many unmet needs, including late or incorrect diagnosis and difficulties for patients in accessing appropriate treatment and expertise.
For researchers, the lack of public funding for basic research, the difficulty in translational research and the organisational, methodological and regulatory limits affecting clinical research are all critical issues calling for closer partnerships and novel approaches/models. This is compounded by the current challenging scenario, in which the multiomics integrated approach together with new technologies, such as artificial intelligence, are reshaping the oncology research landscape and fostering patients’ hopes. That is to say: new opportunities not only for novel drug development but also for a drug repurposing approach for accelerating drug access in rare cancers.
Clinical drug development, including new clinical research methodologies, licensing, reimbursement, utilisation in clinical practice and monitoring of treatment outcomes, should be viewed as a continuum and, to the greatest extent possible, should be planned in a prospective and integrated way, with input from all these stakeholders, starting as early as possible.
Partnership in rare cancers and sarcoma requires a shift in research culture as a whole. Collaboration and innovation are definitely the key words required to make this cultural change a reality.
Sarcoma Patient Advocacy Global Network (SPAGN) session: Research for patient benefit, 20.03.2023, h. 14:30 - 15:50, Hall B3
Patient advocacy on rare cancers, 20.03.2023, 16:20 - 17:40, Hall B3