15 research outputs found
DSM-5 and clinical trials in psychiatry: Challenges to come?
No full textThe publication of the fifth version of the Diagnostic and Statistical Manual of Mental Disorders (DSM-5) in 2013 raises important questions for clinical trials and associated regulatory decisions on new drugs for psychiatric disorders. © 2012 Macmillan Publishers Limited. All rights reserved
The European Medicines Agency's strategies to meet the challenges of Alzheimer disease
No full textRegulatory agencies have a key role in facilitating the development of new drugs for Alzheimer disease, particularly given the challenges associated with early intervention. Here, we highlight the strategies of the European Medicines Agency to help address such challenges
European regulation on orphan medicinal products: 10 years of experience and future perspectives.
No full textIn 2000, regulation on orphan medicinal products was adopted in the European Union with the aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. Since then, more than 850 orphan drug designations have been granted by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products (COMP), and more than 60 orphan drugs have received marketing authorization in Europe. Here, stimulated by the tenth anniversary of the COMP, we reflect on the outcomes and experience gained in the past decade, and contemplate issues for the future, such as catalysing drug development for the large number of rare diseases that still lack effective treatments
A Delphi-method-based consensus guideline for definition of treatment-resistant depression for clinical trials
No full text\ua9 2021, The Author(s).Criteria for treatment-resistant depression (TRD) and partially responsive depression (PRD) as subtypes of major depressive disorder (MDD) are not unequivocally defined. In the present document we used a Delphi-method-based consensus approach to define TRD and PRD and to serve as operational criteria for future clinical studies, especially if conducted for regulatory purposes. We reviewed the literature and brought together a group of international experts (including clinicians, academics, researchers, employees of pharmaceutical companies, regulatory bodies representatives, and one person with lived experience) to evaluate the state-of-the-art and main controversies regarding the current classification. We then provided recommendations on how to design clinical trials, and on how to guide research in unmet needs and knowledge gaps. This report will feed into one of the main objectives of the EUropean Patient-cEntric clinicAl tRial pLatforms, Innovative Medicines Initiative (EU-PEARL, IMI) MDD project, to design a protocol for platform trials of new medications for TRD/PRD
Precision psychiatry roadmap: towards a biology-informed framework for mental disorders.
No full textThe current classification systems for mental disorders provide a uniform symptom-based language to describe and diagnose mental disorders. Clinicians use these classifications to communicate with their patients and colleagues, to treat patients, and when applicable, to request reimbursement from payers. In clinical research and drug development, diagnostic categories are used as enrollment criteria for clinical trials and to inform prescribing information for the appropriate use of therapeutic interventions. However, like other neuropsychiatric diseases, mental disorders arise from the biology of the brain and its bidirectional interaction with the environment. Current classification systems do not reflect this knowledge. With scientific progress in neuroscience, the time has come for global stakeholders to align research efforts to work toward integrating symptomatic, biological, and behavioral information into the definition of mental disorders to advance the development of effective treatments. The European College of Neuropsychopharmacology (ECNP), following the 2024 New Frontiers Meeting, is coordinating a global initiative to design and implement a Precision Psychiatry Roadmap. By mobilizing resources and harmonizing translational methodologies and datasets, the aim is to discuss, design, and implement an iterative framework that incorporates biology-informed evidence into symptom-based syndromes, allowing for more discovery and implementation of mechanism-based effective treatments for mental disorders
Psychopharmacology in children and adolescents: unmet needs and opportunities
No full textPsychopharmacological treatment is an important component of the multimodal intervention approach to treating mental health conditions in children and adolescents. Currently, there are many unmet needs but also opportunities, alongside possible risks to consider, regarding the pharmacological treatment of mental health conditions in children and adolescents. In this Position Paper, we highlight and address these unmet needs and opportunities, including the perspectives of clinicians and researchers from the European College of Neuropsychopharmacology–Child and Adolescent Network, alongside those of experts by lived experience from national and international associations, via a survey involving 644 participants from 13 countries, and of regulators, through representation from the European Medicines Agency. We present and discuss the evidence base for medications currently used for mental disorders in children and adolescents, medications in the pipeline, opportunities in the development of novel medications, crucial priorities for the conduct of future clinical studies, challenges and opportunities in terms of the regulatory and legislative framework, and innovations in the way research is conducted, reported, and promoted
A Delphi-method-based consensus guideline for definition of treatment-resistant depression for clinical trials
No full textCriteria for treatment-resistant depression (TRD) and partially responsive depression (PRD) as subtypes of major depressive disorder (MDD) are not unequivocally defined. In the present document we used a Delphi-method-based consensus approach to define TRD and PRD and to serve as operational criteria for future clinical studies, especially if conducted for regulatory purposes. We reviewed the literature and brought together a group of international experts (including clinicians, academics, researchers, employees of pharmaceutical companies, regulatory bodies representatives, and one person with lived experience) to evaluate the state-of-the-art and main controversies regarding the current classification. We then provided recommendations on how to design clinical trials, and on how to guide research in unmet needs and knowledge gaps. This report will feed into one of the main objectives of the EUropean Patient-cEntric clinicAl tRial pLatforms, Innovative Medicines Initiative (EU-PEARL, IMI) MDD project, to design a protocol for platform trials of new medications for TRD/PRD. © 2021, The Author(s).EU/EFPIA/Innovative Medicines Initiative 2 Joint Undertaking
