Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.
Sponsors developing drugs for rare diseases should pursue several strategies to assess safety, including gathering robust natural history data and using a concurrent comparator arm, the US FDA advises in a new draft guidance.
The draft guidance, "Rare Diseases: Common Issues in Drug Development," revises and replaces a draft by the same name issued in August 2015. (Also see "As Orphan Drugs...
The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.
Sanofi’s Xenpozyme and Sentynyl’s Nulibry are the first two case studies the FDA is using to continue educating rare disease sponsors on best practices.
The first two classes of negotiated drugs under the Inflation Reduction Act would not benefit from a rare disease adjustment House Republicans included in their reconciliation package.
Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.
Joint procurement, if used by member states to respond to potential pricing and access challenges caused by a US most favored nations pricing policy, would cause more uncertainty for pharmaceutical companies, warn industry representatives.
Sponsors can expect faster evaluation processes for key medicines and greater support in mitigating medicines supply shortages this year as part of the European Medicines Agency’s digital transformation overhaul.
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
