2026 ECNP Congress

PS04-4187

Network initiative: advancing patient-centered and regulatory-ready clinical outcome strategies in early-phase neuropsychiatric drug development

Silvia Zaragoza Domingo — R. Schreiber, M.T. Acosta, L. Alphs, E. Arce, F. Di Cesare, M. Davidson, G. Dorffner, C.J. Edgar, J. Engler, S. Galderisi, J. Hubeňák, M. Leboyer, I. Magaraggia, M. Martinez-Cengotitabengoa, A. Mucci, S. Ochoa, S. Pallanti, C. Spuch, D. Tinoco, I. Del Cerro, J.Á. Aibar · Neuropsynchro Neuropsychological Research Organization s.l., Neuropsynchro Neuropsychological Research Organization s.l., Barcelona, Spain

2026 ECNP Congress

Background

Neuroscience clinical trials face disproportionately high failure rates, partly due to inadequate clinical outcome strategies that fail to capture the true benefits of investigational therapies. This challenge underscores the urgent need for practical standards to guide the clinical outcome assessments (COAs) strategy and the selection of core outcome sets (COS). Simultaneously, regulatory agencies increasingly emphasize patient experience data, qualification of novel methodologies, and early stakeholder dialogue.

The ECNP Network on Clinical Outcomes in Early-Phase Clinical Trials. We are a multidisciplinary platform spanning academia, industry, and regulatory agencies, including members from other societies composing the Outcomes Research in Neurosciences Taskforce, dedicated to promoting standard research practices and innovation in outcomes selection and strategy. Building on its foundational work, which includes a consensus-based 7-step framework [1,2], the network is now focused on dissemination and education, as well as on implementing the 7-step standard approach in challenging therapeutic areas such as neuropediatric rare diseases, immunoneuropsychiatry, and negative symptoms in schizophrenia [2,3,4]. We will present our current activities and the Global Network for New Clinical Outcome Measure Standards (NEWCOMS) project, which will pilot different disease-specific research initiatives in a pre-competitive format.

Network structure. The internal structure comprises subgroups addressing outcomes-allied domains, including biomarkers, advanced analytics (AI/ML), outcomes sciences, cognition, and transdiagnostic neurotherapeutics , aiming to establish the standards required to advance outcomes research. So far, the network has made available a freely accessible “playbook” that supports clinical scientists in selecting, modifying, and developing COAs, as well as in selecting COS, ensuring alignment with patient-centered approaches, thus meeting EMA and FDA regulatory requirements. This tool outlines the minimum steps required to substantiate a COA strategy while transparently communicating the risks of streamlining the process.

Ongoing activities. Dissemination efforts include publishing relevant papers on the future of outcomes research, notably in a Special Section of Neuroscience Applied, which offers knowledge and tools for drug developers and independent researchers alike. Educational activities, ranging from introductory courses held in 2025 to advanced formats currently in preparation, are addressed to professionals seeking either a deeper methodological understanding or career development in the field. The 2026 webinar series, “From Data to Value”, will be delivered through the ECNP Knowledge Hub, and feature interactive, expert-led sessions on key topics in outcomes research. Efforts are consolidated through the NEWCOMS consortium, which comprises more than 30 members from different countries, committed to sharing ideas, developing standards, piloting the method, and pursuing collaborative grant funding—including European initiatives such as ERDERA and COST Action. The development of the first Platform for Collaborative Validation of Novel Instruments is planned to be co-created with diverse stakeholders to accelerate the validation of COAs in underserved and methodologically challenging fields.

In conclusion: the network’s activities and strategic plans reflect the current landscape of outcomes research in neuroscience clinical trials and chart a path toward improving the efficiency and conclusiveness of clinical research, ultimately contributing to higher success rates in a field where innovation is critically needed.

References

  1. Zaragoza Domingo, S., 2026. Driving innovation of clinical trials outcomes in neuroscience. Neuroscience Applied. https://doi.org/10.1016/j.nsa.2026.106997
  2. Zaragoza Domingo, S., Alonso, J., Ferrer, M., Acosta, M.T., Alphs, L., Annas, P., Balabanov, P., Berger, A.K., Bishop, K.I., Butlen-Ducuing, F., Dorffner, G., Edgar, C., de Gracia Blanco, M., Harel, B., Harrison, J., Horan, W.P., Jaeger, J., Kottner, J., Pinkham, A., Tinoco, D., Vance, M., Yavorsky, C., 2024. Methods for Neuroscience Drug Development: Guidance on Standardization of the Process for Defining Clinical Outcome Strategies in Clinical Trials. Eur. Neuropsychopharmacol. 83, 32-42. https://doi.org/10.1016/j.euroneuro.2024.02.009
  3. Magaraggia, I., Di Cesare, F., Alonso, J., Edgar, C.J., Heckman, P., Engler, J., Zaragoza Domingo, S., Pani, L., Schreiber, R., 2026. Application of the research domain criteria in early-phase clinical development of transdiagnostic neurotherapeutics: A multidisciplinary perspective. Neuroscience Applied 5, 106996. https://doi.org/10.1016/j.nsa.2026.106996
  4. Acosta, M.T., Zaragoza Domingo, S., Arango, C., Bishop, K.I., Busner, J., Dorffner, G., del Cerro, I., Diaz, J.C., Gruenblatt, E., Holter, S.M., Harel, B., Krishna, V., Mas, M., Moreno, C., Pallanti, S., Plasencia, C., Kittel-Schneider, S., Tinoco, D., Vance, M., Singh, M.K., Giorgi, S., Aibar, J.A., Santuccione Chadha, A., Arce Cirauqui, E., 2025. Methodological challenges in outcomes research for early-trials for implementation of new therapies in neuropediatric rare diseases. Orphanet J. Rare Dis. 20, 521. https://doi.org/10.1186/s13023-025-03814-0
  5. Mucci, A., Davidson, M., Zaragoza Domingo, S., Alphs, L., Bitter, I., Cohen, A., Dorffner, G., Fernandez-Egea, E., Leucht, S., Ochoa, S., Reininghaus, E., Galderisi, S. Negative symptoms in schizophrenia: methodological challenges and emerging solutions for outcome measurement. Neuroscience Applied (in peer review).