Pharmaceutical innovation is about more than just developing new treatments. Researchers from the Ramón y Cajal and 12 de Octubre hospitals are examining the contribution of clinical trials to clinical practice.

Clinical trials that lead to pharmaceutical innovations generate far more than just cutting-edge medicines for unmet medical needs. They also represent investment, job creation, funding and cost savings for healthcare systems, research competitiveness, and, for the specialists involved, a “transformation of clinical practice”. This is the view of Sergio Serrano, from the Infectious Diseases Department at Ramón y Cajal University Hospital in Madrid; coordinator of Area 2 at the Ramón y Cajal Institute for Health Research (Irycis) – Microbiology, Immunology and Infection – and co-head of the Infectious Diseases group within that area. “They require questions to be better structured, results to be measured rigorously, and integration into national and international collaborative networks, which increases the visibility of the researcher and the group, facilitates contributions with a real impact on clinical guidelines and care standards, and reinforces the centre’s reputation as a generator of its own knowledge,” he adds.

Cristina Domínguez González, from the Neuromuscular Unit of the Neurology Department at the 12 de Octubre University Hospital in Madrid, and a member of the Research Group on Rare Mitochondrial and Neuromuscular Diseases at the i+12 Research Institute and Ciberer, adds that “for the clinical researcher, participation in clinical trials is primarily of clinical significance, as it enables patients to be offered access to innovative therapies prior to their commercialisation and to play an active role in their evaluation”.

The latest figures are striking. According to the Spanish Register of Clinical Trials (REEC), the Spanish Agency for Medicines and Health Products (Aemps) authorised 962 clinical trials in 2025, a record figure that places our country as the European leader in terms of the highest number of authorised trials, as well as the one conducting trials across the widest range of therapeutic areas, and has recorded a sustained growth rate for over 10 years. And according to the Guide to Excellence for the Conduct of Clinical Trials in Hospital Pharmacy. Innovation, decentralisation, and the environment and sustainability, published in September 2025 by the Spanish Society of Hospital Pharmacy (SEFH), the General Council of Official Colleges of Pharmacists and Farmaindustria, 40% of the clinical trials currently being conducted in Europe with new medicines involve Spanish participation.

Both Sergio Serrano and Cristina Domínguez highlight the contribution of clinical trials to the healthcare system: “They raise the quality of care, facilitate early access to innovation and strengthen the international standing of our hospitals,” says Serrano, whilst Domínguez emphasises that “they are an essential tool for rigorously determining the efficacy and safety of medical interventions and ensuring that clinical practice is underpinned by scientific evidence. Furthermore, they foster a culture of continuous evaluation and improvement in the quality of care. In this context,‘ he adds, ’it would also be important to facilitate academic studies and real-world analyses of already approved therapies, enabling their use to be optimised and supporting evidence-based decisions from an independent perspective”.

Ongoing trials

Both are actively involved in various clinical trials, both those sponsored by the pharmaceutical industry and independent ones. Thus, the group in which Serrano works is engaged in ‘a significant line of research that focuses on analysing the biological effects of antiretroviral treatment strategies that do not always form part of the classic regulatory endpoints’, he notes, and explains that “beyond virological suppression, we are studying persistent inflammation, immune activation and viral reservoir dynamics—factors that influence long-term comorbidity in people with HIV and which require more mechanistic approaches”.

At the same time, this same group is also developing “interventions at the frontier of knowledge, particularly in the field of the microbiome. The Morelia trial is probably the most notable example at present; it is being conducted in collaboration with various groups both within and outside Irycis, and in it we are assessing whether modulating the microbiome can improve the response to immunotherapy in advanced lung cancer. We start with a donor selected on the basis of a bacterial signature associated with a better clinical response, and we analyse in an integrated manner not only the oncological outcomes, but also the induced immunological and molecular changes, together with the evolution of the gut ecosystem.”

‘The ultimate goal,’ says Serrano, ‘is to go a step beyond individual trials and use the knowledge gained to identify biological pathways and candidate molecules with potential for drug development. It is not merely a matter of evaluating interventions, but of establishing the scientific basis that can underpin future therapeutic innovations.’

Domínguez, for his part, is currently participating in a trial aimed at treating myotonia in myotonic dystrophy type 1 (Steinert’s disease), and plans to launch a gene therapy trial for this condition in the near future. ‘We are also part of a combined study to optimise the treatment of Pompe disease by reducing the accumulation of muscle glycogen, and we will shortly be launching a trial with a monoclonal antibody in selected forms of congenital myasthenia.’ The specialist also highlights the academic and independent trial she is leading as principal investigator to “assess the safety and efficacy of pyrimidine nucleosides in adults with thymidine kinase 2 (TK2) deficiency, an ultra-rare mitochondrial disease. This is a study designed and led by our centre with the aim of generating evidence in the adult population, as current regulatory approval has focused on the most severe paediatric forms. The aim is to provide robust data on its usefulness in a group of patients for whom there are currently no therapeutic alternatives.”

Insufficient recognition

However, this type of research work is not always sufficiently recognised at an institutional level. According to Domínguez, “pharmaceutical innovations resulting from clinical trials are generally well recognised within the scientific community and in specialist publications, but the activity of conducting clinical trials itself receives far less visibility, both at an institutional and a societal level. In many cases, the end result—the approval of a new treatment—is valued, but the clinical and organisational burden involved in participating in its development is not always recognised. This activity requires time, trained teams and specific infrastructure, and is often not structurally supported by the healthcare system itself, which places an additional burden on the professionals who undertake it,” he says.

The specialist at the 12 de Octubre Hospital believes that ‘given the vital role that clinical trials play in the development of innovative therapies, the healthcare system should take a more decisive stance in supporting the creation and consolidation of teams dedicated to this activity, integrating it as a strategic part of care rather than an additional burden’.

Serrano agrees that, in his experience, ‘institutional recognition does not always match the effort involved in this work’, but acknowledges that “patients’ perceptions are usually very positive. There is a high level of commitment to clinical research; many understand that participating in a trial contributes to the advancement of knowledge and, on occasions, provides early access to innovative therapies. That trust is an extraordinary asset to our system and reinforces the need for clinical trial activity to have institutional support commensurate with its collective importance”.

Boosting activity

And how can we drive innovation in clinical trials? A recently published study in *Frontiers in Economics* reveals that Europe’s share of global clinical trials fell from 22% in 2013 to 12% in 2023. “Having professionals with dedicated time, institutional recognition and adequate infrastructure is key to ensuring that clinical research forms a structural part of the healthcare system and does not depend solely on individual effort. Without that structural support, it is difficult to maintain competitiveness in the long term,” argues Domínguez.

Serrano, for his part, points out that “Spain maintains a strong position in clinical trials thanks to the quality of care, recruitment capacity and the commitment of clinical teams. However, the global environment is becoming increasingly competitive and, in day-to-day practice, the main challenge is not a lack of talent or ideas, but the structural conditions in which we work. We need more agile and predictable regulatory processes, with less variation in the interpretation of regulations and less redundant administrative burden, which currently consumes a disproportionate amount of researchers’ time.”

‘It is also essential,’ adds the specialist from Ramón y Cajal, ‘to establish stable professional infrastructures: well-resourced research units, coordinators and support staff with structural funding that is not dependent solely on one-off projects. And one crucial factor: dedicated time for the clinician-researcher. If research relies solely on individual overwork, the model is neither sustainable nor competitive in the medium term.’

‘If Europe wishes to regain its relative influence, it must significantly increase funding for independent clinical trials, promote closer collaboration between academia and industry, and make progress towards genuine interoperability of clinical data. Spain has clear strengths; maintaining and expanding this leadership requires a strategic commitment to infrastructure, streamlining and talent,’ he concludes.