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US FDA Performance Trackers

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EU Performance Trackers

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Pathways & Standards


Japan Adopts Early Approval Scheme For Infectious Disease Drugs

Japan Adopts Early Approval Scheme For Infectious Disease Drugs

 
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Japan has implemented a streamlined regulatory system designed to enable speedier approvals of selected drugs for designated categories of infectious diseases, which does not require Japanese clinical data.

Woodcock Warns of ‘Excessive Worship Of The RCT,’ Pushes New Drug Approval Pathway

Woodcock Warns of ‘Excessive Worship Of The RCT,’ Pushes New Drug Approval Pathway

 

In the second part of the Pink Sheet’s interview with the former senior FDA official, Woodcock says her push to create a rare disease drug approval pathway that wouldn’t require a randomized controlled trial will not return the agency to “an anecdote standard” or cause a downward creep in the evidentiary standard for approval.

UK Regulator Wants More Patient Input At Pre-Authorization Stage

UK Regulator Wants More Patient Input At Pre-Authorization Stage

 
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The MHRA is taking steps to embed “meaningful and impactful” patient involvement across its regulatory pathways.

Woodcock On Proposed Rare Rx Approval Pathway: US FDA’s Current Flexibilities Come Too Late

Woodcock On Proposed Rare Rx Approval Pathway: US FDA’s Current Flexibilities Come Too Late

 

The former CDER Director spoke to the Pink Sheet about why a new statutory standard is needed for certain rare disease drug approvals. We lay out some of her thinking in part one of a multi-part series on the topic.

Regional Comparisons


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

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

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

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.

Decoding Regulatory Agency Decisions: The Case of Amylyx’s Relyvrio/Albrioza In The US And EU

Decoding Regulatory Agency Decisions: The Case of Amylyx’s Relyvrio/Albrioza In The US And EU

 

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.

Drug Review Profiles


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

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

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

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

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


ICH Q5A(R2): ‘More Regulatory Flexibilities Than Hurdles’

ICH Q5A(R2): ‘More Regulatory Flexibilities Than Hurdles’

 
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CRO executive Horst Ruppach discusses the critical factors that drug manufacturers must consider to ensure compliance with the revised ICH Q5A(R2) guideline on biologics viral safety. Thorough documentation and proactive engagement with regulatory bodies are key to navigating the complexities of viral safety evaluation in biotechnology products, he says.

Has Competition Reassurance Helped Counter Combination Woes In The UK?

Has Competition Reassurance Helped Counter Combination Woes In The UK?

 
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The rate of non-submissions for combination therapies in England has been on a downward trend in the last two years.

‘There Is No Silver Bullet’ – Sandoz’s Haruvi Talks US Shortages

‘There Is No Silver Bullet’ – Sandoz’s Haruvi Talks US Shortages

 
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As Sandoz publishes a report on US drug shortages, including a call for policy and other improvements, the firm’s North America head and AAM Chair Keren Haruvi discussed potential solutions to ongoing supply-chain failures.

UK’s ISRCTN Registry Makes It Easier To Track Clinical Trial Transparency

UK’s ISRCTN Registry Makes It Easier To Track Clinical Trial Transparency

 
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The ISRCTN clinical trials registry has launched an improved dashboard to provide metrics that reveal how many studies are complying with key transparency requirements. Badges are in place for individual studies meeting the transparency criteria.