Pink Sheet Perspectives

External reference pricing is one of several drug pricing control measures used by a number of countries to contain drugs prices.

The UK-US trade deal offers the “the most encouraging signs the industry has seen for many years,” but UK companies had little influence over it and details are still scarce, according to one industry expert.

The US pharmaceutical market is critical for European pharma companies, but new policies introduced by the Trump administration are creating an increasingly complex and uncertain environment for them.

Products likely to be subject to evidence generation agreements under England’s Innovative Medicines Fund are those that are costly and which serve a low number of patients. Orphan drugs appear most likely to receive interim funding through the IMF.

Restrictions on commonly used chemicals, increased reporting requirements and enhanced environmental risk assessments are just some of the new EU sustainability and environmental rules pharmaceutical companies are potentially facing. While some measures are multi-sectoral, others are pharma specific. The Pink Sheet takes a look at some of the developments in 2024 and expectations for 2025 and beyond.

The European Medicines Agency is preparing to roll out simplified transparency rules for its Clinical Trials Information System in mid-2024. This article looks at what will change and the impact on the thousands of trials that are already in the system.

The numbers may be down, but Europeans will in future be able to gain access to a number of highly innovative medicines, including several products for hard-to-treat cancers, a gene therapy for severe and moderately severe hemophilia B, and two vaccines against disease caused by respiratory syncytial virus.

Immuno-oncology will remain a major force at both FDA’s drugs and biologics centers, but watch for psychiatry and antibiotics to make some noise; rare pediatric diseases will remain prominent as the priority review voucher program heads into the sunset.

While at least 25 accelerated approval cancer indications or drugs have been pulled or requested withdrawn since 2020, there has not yet been a similar concerted regulatory effort in the non-oncology space despite a large number of products with overdue postmarketing requirements.

CBER director suggests more flexibility on confirmatory study timing is warranted for accelerated approvals in rare diseases and even in some more common scenarios such as infectious disease outbreaks, although a ‘relatively stronger approach’ will apply to most large indications.

Of 11 NMEs and novel biologics, only one – a vaccine for a tropical disease – did not have confirmatory studies underway or completed at the time of accelerated approval. Statutory changes are forcing companies to prioritize confirmatory trials earlier than in the past.

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.”