Research areasOncology Cardiovascular Neurology and dementia diabetes obesity GLP-1s Immunology and autoimmune Infectious disease and vaccines Rare disease Mental health research Ageing and longevity Womens health researchClinical trialsPhase 1, 2, 3 and 4 explained randomisation blinding control arms primary secondary surrogate Reading a trial readout Trial failures and what they mean Finding and joining a trial Diversity in trial enrolment Placebo, standard of care and ethics of control arms Adaptive and platform trials Basket and umbrella designs Trial registration and ClinicalTrials.gov Data monitoring committees and early stopping Expanded access and right to try Decentralised and remote trialsRegulationFDA approval pathways Accelerated approval and confirmatory trials Advisory committees Breakthrough and fast track designations EMA and international divergence Post-market surveillance Generic and biosimilar approval PDUFA dates and what they mean Complete response letters Orphan drug and rare paediatric designations Boxed warnings and label changes Recalls and safety communications Drug shortages and manufacturing inspections Compounded copies of approved drugsEvidenceHow to read a study Absolute vs relative risk Statistical significance and p-values Effect size and confidence intervals Preprints vs peer review Meta-analysis and systematic review Replication and retractions Conflicts of interest and fundingThe systemNIH funding Academic labs and translation Biotech and venture funding Drug pricing and development cost Publishing and open access Research integrity Patents and exclusivity Medicare drug price negotiation Coverage decisions and CMS national coverage analysis Health technology assessment and cost effectiveness Grant terminations and funding disruption Clinical trial diversity requirements Data sharing and individual participant dataNewsDrugs & therapiessemaglutide tirzepatide retatrutide in trials lecanemab donanemab anti-amyloid class pembrolizumab nivolumab antibody drug conjugates CAR-T exagamglogene autotemcel lovotibeglogene onasemnogene mRNA platforms RSV vaccines next generation COVID vaccines inclisiran bempedoic acid SGLT2 inhibitors Biosimilars and generics entering the market Drugs withdrawn or restricted after approvalWho does the researchNIH and its institutes NCI NIAID NHLBI BARDA ARPA-H FDA CBER CDER EMA MHRA Broad Institute Dana-Farber MD Anderson Mayo Clinic Memorial Sloan Kettering Johns Hopkins Pfizer Merck Eli Lilly Novo Nordisk AstraZeneca Vertex Regeneron Moderna Wellcome Gates Foundation CZI patient foundations Contract research organisations and trial sites

Orphan drug and rare paediatric designations

Orphan drug and rare paediatric designations - reporting from Benchtobed.

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