United Kingdom
The pharmaceutical industry was not sufficiently involved in designing updates to England’s health data infrastructure, leading to a “misalignment” between company needs and what is currently being delivered, according to the results of a survey by the Association of the British Pharmaceutical Industry.
The UK drug regulator says that men under the age of 55 years who are already taking valproate will not need to be reviewed by two specialists, as this measure is “unlikely to minimize risks” of neurodevelopmental disorders in children conceived by them any further.
The world’s first CRISPR gene editing therapy, Casgevy, has been made available to patients with sickle cell disease in England, adding to access arrangements in the US, Austria, Bahrain, Germany, Luxembourg, Italy and Saudi Arabia.
The UK’s drug regulator is developing a “clear and streamlined” regulatory pathway for individualized cancer mRNA immunotherapies.
Companies that are admitted to the UK’s revised innovative licensing and access scheme will be mandated to work with the regulator on developing a target development profile for their product.
European regulators should develop a harmonized medicines shortages prevention and mitigation system and look to target the root causes of shortages, says pharmaceutical industry federation EFPIA.
Health technology assessment bodies in England, the US, Canada, Australia, the Netherlands and Colombia have joined forces to produce guidance for drug developers on using surrogate endpoints for cost-effectiveness analyses.
The MHRA’s new scientific dialog program will offer drug developers “closed-door meetings” that will offer “confidential, commercially sensitive discussion” with its staff to help them “refine” their real-world evidence generation strategies.
The UK’s “world-first” tailored regulatory framework for point of care manufacturing is set to enter into force this summer.
“Significant progress” has been made in setting up the UK’s Rare Therapies Launch Pad pilot scheme, which will construct a “new regulatory pathway tailored specifically for ultra-rare diseases,” a senior scientist involved in the project says.
New regulations on clinical trials should help the UK remain a popular destination for multinational trials, according to the MHRA, the country’s medicines regulator.
The UK’s proposed approach to recording informed consent for lower-risk trials is designed to ensure that patients still receive the critical information they need to consider participation, but are supported to give their consent in an easier way.
More than 90% of companies have submitted new medicines packaging artwork to the UK’s drug regulator, the MHRA. The agency urges the remaining marketing authorization holders of its 31 December deadline to comply with the new Windsor Framework arrangements.
As the consultation period draws to a close for the UK’s new inclusion and diversity guidance, researchers and sponsors are being invited to participate in a pilot next year to submit a diversity plan as part of their clinical trial application.
The UK’s MHRA is urging drug companies to engage in discussions on their strategies relating to using real-world data earlier in their development cycle.
NICE, England’s health technology assessment institute, says it was unable to reach a price agreement for AstraZeneca/Daiichi Sankyo’s HER2-low breast cancer drug Enhertu, breaking a line of 21 positive recommendations for breast cancer therapies.
The pharma industry wants regulators around the world to engage with companies and “articulate the value added” when introducing new regulations and guidance around the use of AI in drug development, AstraZeneca’s director for data and AI policy says.
While many companies demonstrated an interest in the UK’s licensing and access pathway for innovative medicines, few made use of the various benefits the scheme had to offer, the MHRA said.
The MHRA is taking steps to embed “meaningful and impactful” patient involvement across its regulatory pathways.
Companies are being encouraged to look at whether they will be impacted by new UK rules on supplementary protection certificates that come into effect from the beginning of next year.