Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy

DT Michaeli, T Michaeli, S Albers, T Boch… - The European Journal of …, 2023 - Springer
Background Over the past decades, US Congress enabled the US Food and Drug
Administration (FDA) to facilitate and expedite drug development for serious conditions …

Decision Criteria for Partial Nationalization of Pharmaceutical Supply Chain: A Scoping Review

PV Marrone, FR Mathias, WM Bernardo, MF Orlandini… - Economies, 2023 - mdpi.com
(1) Background: Any disturbance in the pharmaceutical supply chain (PSC) can disrupt the
supply of medicines and affect the efficiency of health systems. Due to shortages in the …

The cost of biotech innovation: exploring research and development costs of cell and gene therapies

MT Sabatini, M Chalmers - Pharmaceutical Medicine, 2023 - Springer
Background Clinical development paradigms for cell and gene therapies appear to be
different to those of more conventional treatments: therefore, it is informative to explore this …

The long‐term impact and value of curative therapy for HIV: a modelling analysis

GF Guzauskas, TB Hallett - Journal of the International AIDS …, 2023 - Wiley Online Library
Abstract Introduction Curative therapies (CTx) to achieve durable remission of HIV disease
without the need for antiretroviral therapy (ART) are currently being explored. Our objective …

The Application of Evidence-Based Medicine in Individualized Medicine

P Van de Vliet, T Sprenger, LFC Kampers… - Biomedicines, 2023 - mdpi.com
The fundamental aim of healthcare is to improve overall health of the population by
providing state-of-the-art healthcare for individuals at an affordable cost. The foundation for …

Disentangling the cost of orphan drugs marketed in the united states

H Althobaiti, E Seoane-Vazquez, LM Brown… - Healthcare, 2023 - mdpi.com
The increasing number and high prices of orphan drugs have triggered concern among
patients, payers, and policymakers about the affordability of new drugs approved using the …

Neurosymbolic ai for reasoning on biomedical knowledge graphs

LN DeLong, RF Mir, Z Ji, FNC Smith… - arXiv preprint arXiv …, 2023 - arxiv.org
Biomedical datasets are often modeled as knowledge graphs (KGs) because they capture
the multi-relational, heterogeneous, and dynamic natures of biomedical systems. KG …

The Right to Ask, the Need to Answer—When Patients Meet Research: How to Cope with Time

M Priolo, M Tartaglia - … Journal of Environmental Research and Public …, 2023 - mdpi.com
Reaching a diagnosis and its communication are two of the most meaningful events in the
physician–patient relationship. When facing a disease, most of the patients' expectations …

Financing repurposed drugs for rare diseases: a case study of Unravel Biosciences

B Abouarab, C Bazarian, Z Ben Chaouch… - Orphanet Journal of …, 2023 - Springer
Background We consider two key challenges that early-stage biotechnology firms face in
developing a sustainable financing strategy and a sustainable business model: developing …

[HTML][HTML] Modality diversification and best-in-class small-molecule drugs: recent trend of orphan drug development

R Okuyama - Medicine in Drug Discovery, 2023 - Elsevier
Only 5% of orphan diseases have approved drugs, leading to a high demand for new
treatment. As a result, pharmaceutical companies have shifted their focus of drug …