Approval Standards

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.

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.

Sponsors Responsible For Ensuring AI Is ‘Fit For Purpose,’ Says EU Regulator

Sponsors Responsible For Ensuring AI Is ‘Fit For Purpose,’ Says EU Regulator

 

Regulators do not have the resources to “double check” that companies are using AI appropriately, meaning that manufacturers must ensure the AI tools they use meet relevant standards, says an EU regulatory expert.

US FDA’s Drug Approval Norms Could Be Upended In Second Trump Term

US FDA’s Drug Approval Norms Could Be Upended In Second Trump Term

 

Despite the recent anti-vaccine rhetoric in the final weeks of the Trump campaign, pulling an established safe and effective product off market would be difficult. But there’s little to stop political interference in approvals.


Japan Eases Local Clinical Data Requirements For Rare Disease Drug Filings

Japan Eases Local Clinical Data Requirements For Rare Disease Drug Filings

 
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Japan is cautiously easing Japanese clinical data requirements for rare disease drugs to allow faster and more flexible approvals, including on a conditional basis supported by postmarketing studies.

Woodcock Throws Weight Behind Push For New Legislative ‘Substantial Evidence’ Standard For Rare Disease Drugs

Woodcock Throws Weight Behind Push For New Legislative ‘Substantial Evidence’ Standard For Rare Disease Drugs

 

Woodcock and the Haystack Project want to modify a foundational concept of modern FDA drug efficacy assessments, which may be seen as an attempt to more formally codify and define regulatory flexibility.

Gene Therapies: Bluebird Bio Presses US FDA To Reverse Denial Of Lyfgenia Priority Review Voucher

Gene Therapies: Bluebird Bio Presses US FDA To Reverse Denial Of Lyfgenia Priority Review Voucher

 
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Lyfgenia and Zynteglo comprise complex mixtures of transduced cells that represent different active ingredients, the company said in administrative appeals to the FDA. Two lawmakers also raised bipartisan concerns about the agency’s decision on the voucher request for the sickle cell disease treatment.

For Rare and Rapidly Progressing Disease US FDA Has Heard ‘Well and Clear’ Placebo Is Unacceptable

For Rare and Rapidly Progressing Disease US FDA Has Heard ‘Well and Clear’ Placebo Is Unacceptable

 

Remarks at the NORD Breakthrough Summit from the FDA’s Lola Fashoyin-Aje may suggest the agency is becoming more comfortable with single-arm trials.


US FDA ‘Thinking Deeply’ On ‘Digital Twins’ As Placebo Control, But OCE Not Quite Ready To Embrace It

US FDA ‘Thinking Deeply’ On ‘Digital Twins’ As Placebo Control, But OCE Not Quite Ready To Embrace It

 
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Oncology staff also said more work is needed before patient registries can be used as an external control in clinical trials, but they strongly encouraged continued investment.

Novel Pertussis Vaccines: Challenge Models ‘Promising’ But Need More Work

Novel Pertussis Vaccines: Challenge Models ‘Promising’ But Need More Work

 

A US FDA Advisory Committee responded enthusiastically to the potential use of challenge models to enable pivotal trials of novel pertussis vaccines, but most members suggested more refinements are needed before they are ready to use.

US FDA Advisors Urge Faster Movement, mRNA Consideration, In Updating Pandemic Flu Vaccines

US FDA Advisors Urge Faster Movement, mRNA Consideration, In Updating Pandemic Flu Vaccines

 

The FDA’s vaccine advisory committee endorsed a new plan to update pandemic influenza vaccines “inter-pandemic,” but encouraged the agency to consider how to make the approach feasible for novel vaccine technologies.  

Anvisa To Access US FDA-Reviewed Data For More Robust Evaluations In Brazil

Anvisa To Access US FDA-Reviewed Data For More Robust Evaluations In Brazil

 

Brazil has also published its recent regulatory reliance regulations in English, a move designed to increase confidence in its decisions as it presses on with aligning its national practices with global best practices.


Califf: Rare Disease Hub Will Develop Methods To Guide Approvability Determinations

Califf: Rare Disease Hub Will Develop Methods To Guide Approvability Determinations

 

The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads. 

Gene Therapies: CoGenT Pilot Could Help With Regulatory Convergence Earlier In Development

Gene Therapies: CoGenT Pilot Could Help With Regulatory Convergence Earlier In Development

 
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The program for collaborative gene therapy reviews with the European Medicines Agency is starting with simple applications, but after launch could be expanded to earlier discussions of CMC and pharm/tox data requirements, the FDA’s Nicole Verdun said.

Stark Reminders Of Why Antibiotic Space Remains Unattractive On Display At FDA Advisory Cmte.

Stark Reminders Of Why Antibiotic Space Remains Unattractive On Display At FDA Advisory Cmte.

 

As need for new antibiotics keeps increasing, a recent US FDA advisory panel offers a case study of why industry isn’t keen on the space. 

Vanda’s Tradipitant Gets CRL In Gastroparesis But Another NDA Filing Is Still On

Vanda’s Tradipitant Gets CRL In Gastroparesis But Another NDA Filing Is Still On

 

The company will file a new drug application with the US FDA by the end of the year for tradipitant in a different indication, motion sickness.


EMA Tackles Hurdles To Using Single-Arm Trial Data For Pivotal Evidence In Filings

EMA Tackles Hurdles To Using Single-Arm Trial Data For Pivotal Evidence In Filings

 

The European Medicines Agency has highlighted issues for drug developers to consider when seeking to submit clinical data from SATs as the pivotal evidence in their marketing authorization applications instead of randomized controlled trial data.

The Anti-Sintilimab Guidance? US FDA Gives Advice On Oncology Multiregional Trials

The Anti-Sintilimab Guidance? US FDA Gives Advice On Oncology Multiregional Trials

 
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Studies should enroll a representative subgroup of US patients based on the incidence or prevalence of cancer in the US, and the comparator arm should use the US standard of care when possible, the FDA says in draft guidance.

EU Acknowledges Demand For Psychedelic Drug Development Progress: One Million Signatures Now Needed

EU Acknowledges Demand For Psychedelic Drug Development Progress: One Million Signatures Now Needed

 

The European Commission has registered a European citizens' initiative that explains how the EU can foster equitable, timely, affordable, safe and legal access to innovative psychedelic-assisted treatments. Work towards securing the one million signatures needed for the next step is starting soon.

Life After Chevron: US Supreme Court Ruling Cited In FDA Exclusivity Decision Challenges

Life After Chevron: US Supreme Court Ruling Cited In FDA Exclusivity Decision Challenges

 
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Disputes over orphan and new clinical investigation exclusivity are among the early drug and biologic cases where legal filings cite the Loper Bright decision, which overturned the Chevron doctrine of deference to agency actions.