1. New clinical trial designs in the era of precision medicine
- Author
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Alejandro Piris-Giménez, Irene Brana, Rodrigo Dienstmann, Josep Tabernero, Jordi Rodon, Elena Garralda, Institut Català de la Salut, [Garralda E] Servei d'oncologia, Hospital Universitari Vall d'Hebron, Barcelona, Spain. Vall d'Hebron Institut d'Oncologia, Barcelona, Spain. Grup de Desenvolupament Clínic Precoç de Fàrmacs, Vall d'Hebron Institut d'Oncologia, Barcelona, Spain. [Dienstmann R] Servei d'oncologia, Hospital Universitari Vall d'Hebron, Barcelona, Spain. Vall d'Hebron Institut d'Oncologia, Barcelona, Spain. Grup d’Oncology Data Science (ODysSey), Vall d'Hebron Institut d'Oncologia, Barcelona, Spain. [Piris A, Brana I] Servei d'oncologia, Hospital Universitari Vall d'Hebron, Barcelona, Spain. Vall d'Hebron Institut d'Oncologia, Barcelona, Spain. [Rodon J] Servei d'oncologia, Hospital Universitari Vall d'Hebron, Barcelona, Spain. Vall d'Hebron Institut d'Oncologia, Barcelona, Spain. MD Anderson Cancer Center, Houston, United States. [Tabernero J] Servei d'oncologia, Hospital Universitari Vall d'Hebron, Barcelona, Spain. Vall d'Hebron Institut d'Oncologia, Barcelona, Spain., Vall d'Hebron Barcelona Hospital Campus, and Hospital Universitari Vall d'Hebron
- Subjects
0301 basic medicine ,Cancer Research ,Oncologia ,Review Article ,Basket protocols ,0302 clinical medicine ,terapéutica::medicina de precisión [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS] ,adaptive designs ,Neoplasms ,umbrella protocols ,Medicina personalitzada ,Cooperative Behavior ,Precision Medicine ,Health Occupations::Medicine::Internal Medicine::Medical Oncology [DISCIPLINES AND OCCUPATIONS] ,Review Articles ,Clinical Trials as Topic ,basket protocols ,Health Care Quality, Access, and Evaluation::Quality of Health Care::Health Care Evaluation Mechanisms::Epidemiologic Study Characteristics::Clinical Studies as Topic::Clinical Trials as Topic [HEALTH CARE] ,General Medicine ,lcsh:Neoplasms. Tumors. Oncology. Including cancer and carcinogens ,3. Good health ,Calidad, Acceso y Evaluación de la Atención de Salud::Calidad de la Atención de Salud::Mecanismos de Evaluación de la Atención de Salud::Características de Estudios Epidemiológicos::Estudios Clínicos como Asunto::Ensayos Clínicos como Asunto [ATENCIÓN DE SALUD] ,Oncology ,Drug development ,Risk analysis (engineering) ,Novel agents ,030220 oncology & carcinogenesis ,Molecular Medicine ,Adaptive designs ,Process (engineering) ,Cancer Core Europe ,Umbrella protocols ,Empleos en Salud::Medicina::Medicina Interna::Oncología Médica [DISCIPLINAS Y OCUPACIONES] ,lcsh:RC254-282 ,03 medical and health sciences ,profesiones sanitarias::medicina::medicina interna::oncología médica [DISCIPLINAS Y OCUPACIONES] ,Cancer Medicine ,Genetics ,Humans ,European Union ,Basket of Baskets ,Therapeutics::Precision Medicine [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES AND EQUIPMENT] ,business.industry ,Precision medicine ,calidad, acceso y evaluación de la atención sanitaria::calidad de la atención sanitaria::mecanismos de evaluación de la atención sanitaria::características de los estudios epidemiológicos::estudios clínicos como asunto::ensayos clínicos como asunto [ATENCIÓN DE SALUD] ,Review article ,Clinical trial ,030104 developmental biology ,Personalized medicine ,business ,Assaigs clínics - Abstract
Adaptive designs; Umbrella protocols; Basket protocols Dissenys adaptatius; Protocols de paraigua; Protocols de cistell Diseños adaptativos; Protocolos de paraguas; Protocolos de cesto Cancer treatment has made significant strides towards the promise of personalized medicine. Recent scientific advances have shown that there are numerous genetic deregulations that are common in multiple cancer types, raising the possibility of developing drugs targeting those deregulations irrespective of the tumour type. Precision cancer medicine was born out of accumulated evidence matching targeted agents with these tumour molecular deregulations. At the same time, the therapeutic armamentarium is rapidly increasing and the number of new drugs (including immune-oncology agents) entering drug development continues to rise. These factors, added to strong collaboration with regulatory agencies which have approved novel agents based on data obtained from Phase 1/2 trials, have led to unprecedented evolution in the design of early stage clinical trials. Currently, we have seen rapid Phase 1 dose escalation trials followed by remarkably large expansion cohorts, and are witnessing the emergence of new trials, such as adaptive studies with basket and umbrella designs aimed at optimizing the biomarker-drug co-development process. Alongside the growing complexity of these clinical trials, new frameworks for stronger and faster collaboration between all stakeholders in drug development, including academic institutions and frameworks, clinicians, pharma companies and regulatory agencies, have been established. In this review article, we describe the main challenges and opportunities that these new trial designs may provide for a more efficient drug development process, which may ultimately help ensure that precision cancer medicine becomes a reality for patients
- Published
- 2018