Patient engagement and efficiency in clinical trials: an opportunity the industry cannot ignore
A qualitative study reveals that early and meaningful patient involvement improves the quality, ethics and efficiency of pharmaceutical research.
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Patient involvement in clinical research and the development of new medicines has become widely accepted in the pharmaceutical industry in recent years. However, beyond theoretical consensus, there remains a significant gap between discourse and practice. A recent qualitative study based on in-depth interviews with patients, carers and advocates reveals both the transformative value of patient engagement (PE) and the persistent challenges that prevent this collaboration from reaching its full potential.
The study, conducted in the context of drug development and with participants mainly linked to rare diseases, confirms a trend already noted in the literature: when involvement is genuine, continuous and bidirectional, patients cease to be passive subjects and become active partners who influence research priorities, trial design and the definition of relevant clinical outcomes.
The study participants agreed that the main value of PE lies in its ability to identify unmet needs, preferences and acceptable risks from the patient’s perspective, elements that are often not reflected in protocols designed exclusively from a scientific or regulatory perspective. This contribution is key to improving the clinical relevance and acceptability of trials, especially in complex areas such as rare or chronic diseases.
Research shows that patients consider engagement to be meaningful only when it is based on two-way communication, using accessible language and explicitly acknowledging their lived experience. In this regard, the study points to a recurring criticism: the industry often hides behind regulatory barriers to limit dialogue, when participants perceive the real obstacle to be cultural rather than regulatory.
Tangible impact on clinical trials and outcomes
From an operational perspective, interviewees pointed out that patient involvement can directly improve the efficiency of clinical trials, especially in the recruitment and retention of participants, one of the major bottlenecks in pharmaceutical development. The co-creation of endpoints and outcome measures aligned with what really matters to patients also emerges as a critical factor in avoiding studies that fail due to lack of clinical relevance.
This approach has not only scientific implications, but also economic ones. Some participants emphasised that involving patients from early stages can prevent costly design errors, saving time and resources for pharmaceutical companies and increasing the likelihood of success for development programmes.
Empowerment, identity and ‘wearing multiple hats’
Beyond the benefits for the industry, the study highlights a profound impact on patients themselves. Many describe PE as an empowering experience that increases their confidence, knowledge, and ability to actively participate in decisions about their health. Formal recognition is perceived as one of the greatest symbolic achievements.
A particularly relevant finding is that most participants do not identify solely as patients. Many combine roles as carers, advocates, healthcare professionals or even pharmaceutical industry employees. This multiplicity of identities brings a wealth of perspectives, but also highlights a tension: the industry tends to view them solely through the prism of their illness, failing to capitalise on their experiential and professional capital.
Structural barriers and the risk of tokenism
Despite consensus on the value of EFP, the study highlights persistent obstacles. These include late participation in development processes, administrative complexity, lack of standardisation between companies and a lack of feedback after collaborations. More than half of those interviewed said they had experienced tokenism, where their participation was perceived as a purely formal exercise with no real impact on decisions.
The absence of cycle closure emerges as one of the most frustrating and damaging factors for trust. From an analytical perspective, the study proposes understanding communication as a hygiene factor: it does not generate motivation on its own, but its absence causes dissatisfaction, mistrust, and abandonment of engagement.
Equity, diversity and capabilities
Another critical issue is representativeness. Participants pointed out shortcomings in the inclusion of ethnic minorities, people with language barriers, and communities with fewer resources, which limits the generalisation of results and perpetuates inequalities in research. Likewise, the need for two-way training was emphasised: patients trained to contribute effectively and industry professionals trained in empathy, communication, and collaboration.
Towards a new model of collaboration
Overall, the study suggests that patient engagement should not be understood solely as a tool for optimising clinical trials, but as a paradigm shift in the way drug development is conceived. Moving from a transactional logic to a relationship implies recognising the intrinsic value of patient empowerment and its long-term impact on the quality, ethics and sustainability of research.
The underlying message is clear: patient involvement is a question of how and when. Companies that manage to integrate the patient’s voice in a structural, early and transparent manner will not only improve their development results, but will also be better positioned in a healthcare environment that is increasingly oriented towards shared responsibility, personalisation and trust.
