Clinical trials are essential for the development of new therapies. In these studies, the participation of patients and healthy volunteers is crucial to assess the efficacy and safety of new treatments being developed.

Traditionally, researchers have evaluated results primarily from a clinical perspective. But increasingly, more and more importance is being assigned to patients' perceptions: they are no longer passive subjects of research, but play an active role. This paradigm shift is key to obtaining a more complete overview of trials, taking into account both clinical safety and efficacy as well as the impact on daily life as described by patients.

The involvement of patients allows for a more direct understanding of the effects of treatments: the symptoms they experience, their satisfaction with the therapy they receive, the benefits in their quality of life, the impact on the lives of caregivers, among others. In this way, research progresses based on the experience of people who need new treatments.

Patient feedback: another factor in assessing the efficacy of new drugs

An example of this new trend is the study led by the Childhood Cancer and Blood Disorders group at the Vall d'Hebron Research Institute (VHIR), recently published in Pediatric Blood & Cancer. This paper has analysed how many early-stage clinical trials in childhood cancer incorporate the voice of patients or their families when assessing outcomes, i.e. how many of these studies take into account the impact on quality of life of the new therapy being tested. This is known as patient-reported outcome measures (PROMs), which are often collected through questionnaires.

The study, conducted in collaboration with the Royal Marsden Hospital in London, analysed 72 clinical trials in childhood cancer conducted between 2005 and 2022. In particular, it focused on early phase trials, i.e. initial phase 1 or 2 studies that assess the safety, dose and effects of a new treatment in a small number of patients, often for the first time.
We found that between 2005 and 2016, only 14% of trials looked at patients' quality of life, while this percentage increased to 19% between 2017 and 2022. In addition, drug acceptability, i.e. how well patients agree to follow a treatment, was measured in 39% of studies in the first period, rising to 63% in the second period.

"There is still a long way to go, but it is good news to see that gradually the voice of patients is being incorporated in clinical trials from early stages, since working together professionals and patients is the only way to obtain good results in our studies", says Dr. Lucas Moreno, head of the Paediatric Oncology and Haematology Department at Vall d'Hebron University Hospital and of the Childhood Cancer and Blood Disorders group at VHIR.

In order that these percentages continue to grow, the team stresses the need for more resources and training to have the time and staff to conduct the surveys, support families, and analyse and interpret the data collected.

The work led by Dr. Moreno has been funded by the Instituto de Salud Carlos III (FORT23/00034), co-financed by the European Regional Development Fund/European Social Fund ‘A way to make Europe’/‘Investing in your future’.

More involvement for better outcomes

Taking into account the opinion of patients and caregivers has a direct impact on improving their quality of life, as it helps to more comprehensively manage symptoms that may appear or disappear during the course of treatment. It also improves adherence to treatment, i.e. patients are more likely to follow medical instructions correctly. All this, in the long term, improves their survival and favours more complete results in the clinical trial.

Moreover, collecting this direct feedback from patients is important for decision-making in the development of new drugs. Patient experience is a factor to consider when prioritising the development of drugs that are not only clinically effective, but also have a positive impact on quality of life. This is especially relevant in early phase trials, such as the study mentioned above, as they are involved from the beginning.

Vall d'Hebron's commitment to patient involvement in research and care

Vall d'Hebron is working to progressively increase patient involvement in research and care.

With regard to research, a growing number of patient advocates are playing an active role from the trial design process. They are trained to bring their expertise and perspective throughout the disease research process, working with researchers to identify the needs of all patients with a disease and propose possible solutions.

An example of the incorporation of patient involvement from the beginning at VHIR is the TACTIC project, which focuses on research in chronic and minority diseases and has recently been funded by the FORTALECE-Salud programme of the Instituto de Salud Carlos III. By actively promoting the participation of patients and citizens, the aim is for research to be not just about patients, but with them. This collaborative approach allows patients to contribute throughout the research process, from diagnosis and prognosis to the development of innovative therapies. Citizen input enables results to be more personalised and focused on their needs.

In the specific field of childhood cancer, the BEACON project in neuroblastoma also considers patient and family feedback and the impact on quality of life when analysing trial results. In addition, it is an example of a project that involves patient advisors from the inception of the trial and in deciding on the next steps in research.

In terms of clinical practice, Vall d'Hebron is a pioneer in promoting the ICE (Information, Co-creation and Strategy) model of patient and professional participation, as well as having a Citizen Participation Unit to institutionalise relations between professionals and patients during all stages of life and illness. One of the courses of action implemented is to open up participation through various stable working committees by area of knowledge, which enables all kinds of needs to be proactively detected and proposals to resolve them to be jointly developed, in addition to the promotion of a Strategic Participation Committee with a healthcare and research perspective.

Efforts are also being made to incorporate the results reported by patients in day-to-day care. Using health status questionnaires, the prevention of complications and early detection is facilitated, and communication between patients and professionals is improved.