Pink Sheet Perspectives

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While at least 25 accelerated approval cancer indications or drugs have been pulled or requested withdrawn since 2020, there has not yet been a similar concerted regulatory effort in the non-oncology space despite a large number of products with overdue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

The Federal Trade Commission blocked or restricted several deals last year, broadening what it considers to be anticompetitive transactions. Stakeholders are looking to see if the agency will bring similar cases this year and what remedies it will seek to allow deals to go forward.

Innovation metrics like first-in-class and breakthrough therapy approvals declined as a share of novel agents in 2023, even as the US FDA’s drugs center posted one of its biggest novel approval totals ever.

US FDA has not had a new drug application focused advisory committee meeting in more than two months, and none are currently scheduled in 2024. After a blistering pace in the first half of 2023, FDA’s sudden slowdown in expert panels is perplexing.

CDER holds steady with a median time to approval of 11 months, while CBER posts 10.5 month median review time for its largest novel approvals class.

After a year of revising and opening up its regulations for new drugs in 2023, Japan is aiming to add more reimbursement pricing premiums for innovative medicines in 2024, while cautiously discussing other detailed revisions to regulations and the pricing system.

CDER missed six goal dates among its 2023 novel approvals, reflecting safety concerns as well as pandemic-related inspection delays; Duchenne muscular dystrophy accelerated approval disagreements caused the biologics center’s lone missed goal.

A plethora of new and revised regulations in China to regulate drug distribution and storage, the set up of new corporations, administrative appeals, as well as the export of gene editing technology, are set to come into effect in 2024, ushering in more challenges to pharma and life sciences companies operating in the country.

A round up of reform policies targeting the biopharma industry and its pricing practices that have been implemented or proposed by the Biden Administration to date.

Current and former commissioners note their positive expectations for the coming year and discuss efforts to standardize drug review and globalize filing of generics, while Janet Woodcock strikes a more sober tone on the ‘limitations of what both government and medicine can do.’

AI initiatives in a country known for its software prowess, along with startup support, clinical trial rule changes, crunched approval timelines, unified digital regulatory interfaces and a R&D policy laying out $600m in funds, are all meant to build something greater than the sum of its parts. In a bottom-up approach, India is building an innovation ecosystem

Gene therapies, bispecific antibodies and vaccines stand out among 2023’s 34 novel biologics and the US FDA’s 38 new molecular entities include fewer breakthroughs.

Pink Sheet reporter and editors discuss key US and EU legislation that impacted 2023 and how it also could affect 2024, including EU legislative reform, US Medicare drug price negotiation, and the US Food and Drug Administration user fee reauthorization.

Steps to foster the biopharma industry, a regulatory science innovation law and a new pan-government policy control tower were among the highlights of policy and regulatory activity in South Korea in 2023 set to have an ongoing impact throughout this year.