The U.S. Food and Drug Administration (FDA) has approved the use of Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec-rzyl) for the treatment of a rare inherited form of vision loss and blindness. This marks the first approval by the FDA of a directly administered gene therapy for the treatment of a disease caused by mutations…
The U.S. Food and Drug Administration (FDA) published its recommendation for a new approach to drug development for rare pediatric diseases. The new approach supports the FDA’s commitment to increase the efficiency and speed of developing drugs, as well as supporting the sponsors of the research. The draft guidance, ‘Pediatric Rare Diseases: Collaborative Approach for…
From 30th March 2019, when the United Kingdom’s (U.K.) is expected to withdraw from the European Union (EU), all EU law will no longer apply to the U.K. The European Medicines Agency (EMA) and the European Commission have therefore published additional practical guidance to help pharmaceutical companies legally prepare for this transition. This guidance will…
Amsterdam has been selected as the new post-Brexit home for the European Medicines Agency (EMA). It was chosen in a draw of lots between it and Milan, after three rounds of voting had failed to pick a favourite. The EMA has been located in London since its formation in 1995, where it employs around 900…
The European Medicines Agency (EMA) and the European Network for Health Technology Assessment (EUnetHTA) have finalised “a joint work plan outlining key areas of collaboration for the next three years”. The main areas of collaboration are information exchange at market entry, data generation post approval and early dialogue. These areas are expected to also provide…
Partners4Access (P4A), the specialists in orphan drug access, are launching a weekly newsletter focussing on key developments in the rare diseases and orphan drugs market access landscape. The service will offer a comprehensive weekly summary of the most interesting and relevant news articles, beginning on Thursday 16th November 2017. The newsletter will allow readers to…
Sophie Schmitz, Managing Partner of Partners4Access, the specialists in orphan drug access, is presenting at the 8th annual meeting of the World Orphan Drug Congress in Barcelona on November 15, 2017 on Gene Therapy. Schmitz will be reviewing the potential of Gene Therapy to revolutionise the healthcare industry, highlighting recent successes and discussing key challenges. “Gene…
Partners4Access (P4A) the specialists in orphan drug access, are presenting 3 posters at the International Society for Pharmacoeconomics and Outcomes Research’s (ISPOR) 20th European Congress in Glasgow from 4th to 8th November 2017. Select members of the Partners4Access specialist team will be presenting the posters in person: Evaluation Framework for digital medicines in Europe (A…
Partners4Access (P4A), the specialists in orphan drug access, are launching a new webinar series, in line with their expanding activities in gene therapy. Launching in November 2017, P4A invites participants to register now to secure their place for this inaugural webinar. As with most innovative and novel areas, gene therapy is facing misconceptions guided by hopes,…
Earlier this month, Italy’s Health Minister, Beatrice Lorenzin, announced that two additional countries- Slovenia and Croatia- had signed up to the Valletta Declaration. Of the 28 EU member states, over a third (10 countries*) are now part of the initiative which aims to strengthen their bargaining power over pharmaceutical companies through collective price negotiations and…