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Current regulation of T cell receptor (TCR)-based therapeutics may require repeated testing of patients for specific HLA alleles as well as companion diagnostics development, despite the invariant nature of the HLA genotype and availability of robust clinical HLA tests. This increases the burden on patients and the organizations developing these products. We propose regulatory flexibility to facilitate the development of and access to TCR-based therapeutics.
The total number of rare conditions is debated, partly because of the variety of definitions of what constitutes rare. A broader consensus view of what rare means, based on improved understanding of individual group and patient clinicopathological characteristics, will help maximize the impact of technological advances in therapeutic development programmes.
Despite major advances in cancer research, we are falling short in bringing innovative medicines to children with cancer. We propose a new business model to incentivize investors, private companies and public organizations to invest in drug development for paediatric cancers.
Innovative approaches to conducting proof-of-concept trials could accelerate the evaluation of repurposed drugs. Drawing lessons from repurposing efforts for COVID-19 therapies, here we present a vision for a drug repurposing platform that could maximize efficiency and improve patient outcomes for many different conditions.
Thirteen pharmaceutical companies have shared and integrated preclinical and clinical data for creating computational resources that enhance translational drug safety assessment.
The low number of clinical trials in Africa and biomedical datasets that disproportionately represent populations of European ancestry contribute to the suboptimal efficacy and safety of some medicines in African populations. To address these disparities, we propose greater incorporation of African data into drug discovery and development, as well as the development of African-centric preclinical and clinical models and tools.
The last decade has seen major progress in the molecular diagnosis of rare inherited diseases, underpinning gene-targeted therapies that are now in the clinic. However, new treatment development is slow and very expensive. Here, we highlight the opportunities for mitochondrial disorders, the challenges and some of the potential solutions.
Patients and the wider public are beneficiaries of scientific research that leads to new drugs and medical technologies, but they can and should be able to contribute to these advances through participation in clinical studies, co-design of research and input into regulatory processes.
Improved understanding of regulatory requirements by academic researchers can accelerate the translation of new medical interventions to the clinic. Here, we highlight the recommendations of the ‘Strengthening Training of Academia in Regulatory Science’ (STARS) project to improve the interaction and knowledge exchange between academics and regulators, and thereby advance academic drug development.
Error-corrected next-generation sequencing (ecNGS) is an emerging technology with the potential to revolutionize the field of genetic toxicology. Here, we present recommendations from an expert working group convened to discuss potential applications, advantages and challenges associated with implementing ecNGS in nonclinical safety studies.
