Europe – Clinical investigation of recombinant and human plasma-derived factor IX products – Scientific...
This guideline describes the information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor IX products is made for use...
Europe – First nasal adrenaline spray for emergency treatment against allergic reactions
EMA’s human medicines committee (CHMP) has recommended granting a marketing authorisation in the European Union for Eurneffy (epinephrine), the first medicine to be taken through the...
Europe – Positive CHMP opinion on first-in-class medicine to treat pulmonary arterial hypertension
EMA has recommended granting a marketing authorisation in the European Union (EU) for Winrevair (sotatercept) to treat adult patients with pulmonary arterial hypertension (PAH), in combination with...
Europe – EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
EMA’s human medicines committee (CHMP) has recommended not renewing the conditional marketing authorisation for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy. Translarna...
UK – Responding to new final draft guidance from NICE for a new gene...
Professor Sir Stephen Powis, NHS National Medical Director, said: “This transformative gene therapy is the first of its kind for haemophilia B patients on...
Europe – EU actions to tackle shortages of GLP-1 receptor agonists
EMA and the Heads of Medicines Agencies (HMA), through the Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG), have issued recommendations to tackle shortages of the...
Europe – Health Technology Assessment : Guidance on reporting requirements for multiplicity issues and...
This guidance describes how to deal in practice with multiplicity issues and
complementary analyses (like specific subgroup analyses, specific post hoc analyses and sensitivity analyses)...
Europe – Health Technology Assessment : Guidance on outcomes for joint clinical assessments
Clinical outcome assessments (COAs), used in clinical studies (whether they are interventional (also called experimental), or observational), are a key component of health technology...
USA – Drugmakers, compounders at odds over FDA’s difficult-to-compound rule
Drugmakers laud the US Food and Drug Administration’s (FDA) proposed rule on drugs that are difficult to compound by pharmacies and outsourcing facilities. Compounders,...
USA – FDA finalizes guidance on drug development for diabetic foot infections
The US Food and Drug Administration (FDA) finalized its advice to drugmakers developing treatments for diabetic foot infections without bone and joint involvement, focusing...
