Keeping Track: Breakthrough Therapies Becoming Breakthrough Applications For Tezacaftor, Burosumab, Emicizumab

Mechanisms in the draft treaty that the more than 190 member states of the World Health Organization have finally agreed to are expected to “materially affect companies,” particularly those that develop, manufacture or distribute pandemic-related health care products.

Sponsors and regulatory agencies within the 27 EU member states must all be “committed” to working with the EU Clinical Trials Regulation in the most appropriate way to keep pace with other countries, including the UK where competition is heating up, says Miguel Forte, CEO of Kiji Therapeutics.

Europe’s vaccines industry wants to make sure immunizations “are not forgotten” by policymakers who are overseeing the EU Health Technology Assessment Regulation. It has highlighted several nuances of vaccines that should be addressed over the coming years.

Industry feedback on a European Medicines Agency pilot that introduced a “stepwise” approach to pediatric investigation plans was positive, but the future of the new model will depend on the outcome of the pharmaceutical legislation negotiations, the agency says.