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US FDA Performance Trackers

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EU Performance Trackers

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Global Guidance Tracker

Health Technology Assessment In Europe


Pathways & Standards


EU Pharma Reform: Negotiations Could See Patient Voice ‘Discarded,’ Warn Patient Groups

 

An enhanced role for patients in the European Medicines Agency was a key proposal within the EU pharmaceutical legislation overhaul – but patient groups warn this provision could be scrapped or weakened due to ongoing negotiations.

User Fee Deadline Concerns Emerge As US FDA Buyout Offer Sweetened With Paid Leave

 

The buyout excludes reviewers, but not project managers. Experts said product reviews likely would be harmed if review team-specific project managers could take the buyout.

UK: New ILAP Will Involve Discussions On ‘Commercial Flexibilities’ For Innovative Products

 

The UK drug regulator has made a number of improvements to its innovative licensing and access pathway, including a more “core” role for National Health Service partners, which is expected to facilitate flexible commercial discussions around drug reimbursement.

Sponsors Increasingly Happy With CBER Written Responses To Formal Meeting Requests

 

The FY 2024 fourth quarter had the highest number of formal meetings cancelled because written responses were sufficient since the beginning of FY 2018.

Regional Comparisons


AstraZeneca Says Andexxa Will Not Convert To Full Approval, Quietly Reveals Complete Response

 

Whether the FDA will seek withdrawal of the anticoagulant-reversal agent or request additional confirmatory data is unclear.

How The UK, EU, Canada, Australia And Japan Are Tackling Diversity In Clinical Trials

 
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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. 

EU Revokes Intercept/Advanz’s Ocaliva Approval As Moment Of Reckoning Nears in US

 
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The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked.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.

England Lags Behind Eight European Countries On Innovative Drug Availability

 

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.

Drug Review Profiles


US FDA Review Of Akebia’s Vafseo Informed By Roxadustat, Daprodustat Safety Concerns

 
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Vadadustat's review overlapped with two other oral HIF-PH class drugs. Safety issues with FibroGen/AstraZeneca’s roxadustat drew attention during vadadustat's first-cycle review, while GSK’s Jesduvroq labeling and postmarketing requirements informed the second-cycle approval.

Akebia’s Vafseo: Japanese Postmarketing Data, Narrowed Indication Eased Liver Toxicity Worries

 
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The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.

Vafseo Chronology: Complete Response Letter, Dispute Resolution And A Second-Cycle Approval

 
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The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.

Biosimilars: US FDA’s Updated Scientific Thinking Led To Interchangeability For Amgen's Wezlana

 
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Pink Sheet’s Drug Review Profile explores Amgen’s Wezlana, an interchangeable biosimilar to Janssen’s Stelara. FDA determined late in the review that switching studies generally would not be needed for interchangeable ustekinumab products, prompting Amgen to seek the designation.

Interviews


Cross-Atlantic Alliance Sets Sights On First HTA Methods Recommendations

 

A new collaboration of health technology appraisal bodies in the US, England and Canada could lead to better alignment on HTA methodology in the future.

PICO Exercises: A Glimpse Into Future Joint Clinical Assessments Under EU HTA Regulation

 

The European Commission has released three exercises to help pharma companies prepare for joint clinical assessments under the new EU Health Technology Assessment Regulation. Experts explain how manufacturers should interpret these and what their limitations are.

Navigating the Unknown: EU HTA Regulation’s Impact On Commercial Confidentiality

 

Company dossiers submitted for EU-level joint clinical assessments under the Health Technology Assessment Regulation will be made public. This could conflict with other strategic business decisions and should be planned for in advance, experts say.

Retiring CDER Deputy Director: Misconception That US FDA Regulators Cannot Change

 

Retiring CDER Deputy Director Douglas Throckmorton spoke to the Pink Sheet ahead of his early 2025 retirement about regulatory flexibility, AI and other topics.