Regional Comparisons

As efforts to improve diversity in clinical trials gain momentum globally, the Pink Sheet asked regulators in the UK, the EU, Canada, Australia and Japan about their efforts to support representative enrolment. 

The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked. Meanwhile the drug's approval is in jeopardy in the US, where an advisory committee will opine on whether the accelerated approval drug has confirmed clinical benefit.

In this third article of a series on new drug reimbursement recommendations by the HTA body NICE, the Pink Sheet finds that fewer innovative medicines are reimbursed in England than in eight other European nations.

Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.

Pink Sheet reporter and editors discuss the impact of a US and EU drug approvals analysis that found the US FDA still clears many novel products first, including most new cell and gene therapies and cancer treatments.

Comprehensive table comparing all the novel agents approved by either the US FDA or the European Commission over 2023 through April 2024.

Pink Sheet infographic shows how a large majority of novel drugs make it past the US FDA before receiving approval in the European Union. Lag times ranged from almost five years to less than two weeks.

Ninety-five percent of novel cancer treatments approved in both the US and Europe received the FDA’s OK first, but Europe usually followed within a year, a Pink Sheet analysis shows.

Only three of the 10 new therapies approved by the US FDA also have been approved in the European Union, while both of the EU gene therapy approvals followed FDA clearance, a Pink Sheet analysis shows.

A Pink Sheet analysis finds US approval preceded European Union clearance for 80% of products approved in both areas, but when EU approval came first, it beat the FDA by a median of 13 months. US-first approvals came close to six months before the EU.

Regulators from the US, Canada, European Union, Switzerland and Japan talk about the benefits of global collaboration, and reasons why they might reach different decisions on the same drug, at the American Society of Clinical Oncology annual meeting.

EMA charges participants to receive scientific advice through the fledgling program, unlike the US FDA, which may not fit the budgets of some complex generic sponsors. At the same time, sponsors also may simply not be aware the program exists yet.

Acknowledging that it may be a barrier to some extent, EMA’s senior scientific specialist of clinical pharmacology Kevin Blake notes that EU reference medicinal products are a legal requirement for bioequivalence studies and cannot be circumvented.

While EU preparations are underway to introduce strict legal requirements for all AI systems, the UK has doubled down on its flexible, non-regulatory framework. In this second of a two-part article, a lawyer explains the pros and cons of each for pharma and medtech firms.

Flexible thinking and rigorous standards will both be needed to develop psychedelics as drug therapies in order to surmount the many complicating factors, from unique ‘set and setting’ aspects to functional unblinding, speakers at Reagan-Udall Foundation meeting agree.

While approaching the third year of its launch, the parallel advice program has seen a handful of applicants, but regulators on both sides of the Atlantic remain optimistic.

International interest in fast-track regulatory reliance and collaboration pathways such as Project Orbis and the ACCESS Consortium has grown in recent years, a trend that looks set to continue in 2024. But some policy experts have warned that faster approvals are not always better, with one academic claiming the bar for marketing authorizations has dropped “far too low.”

Sixteen cancer therapies have been granted marketing authorizations or label variations in the US this year via the Project Orbis scheme. However, the majority of these are still under review by Orbis partners, which have also been outpaced by the non-Orbis EU.

European Union reform proposals could reduce ‘clock stop’ time in drug reviews, which a new study identified as the primary driver of longer review times at the EMA compared with the US FDA.

Regulators from Canada, the UK and Australia discuss their experiences of working together under both the ACCESS Consortium, an international regulatory worksharing coalition, and Project Orbis, a parallel review process designed to speed up access to oncology medicines.