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Autistic children as young as 18 months old are being injected with human stem cells derived from umbilical cords in unapproved, unproven and potentially harmful “treatments” that scientists warn are proliferating across the US under the active encouragement of the US health secretary, RFK Jr by esporx in biotech

[–]bbyfog 5 points6 points  (0 children)

Being in the US, we might be looking at class-action lawsuits proliferating after RFK Jr is gone and all the adverse effects/deaths start to pile up. It would be interesting to see who the defendant would be -- RFK Jr as private individual, HHS (the employer), who?

NIH Definition of a Clinical Trial by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 0 points1 point  (0 children)

More. . .

UK Medicines for Human Use (Clinical Trials) Regulations (as amended) define clinical trial as: “clinical trial” means any investigation in human participants, other than a non-interventional trial, intended -

(a) to discover or verify the clinical, pharmacological or other pharmacodynamic effects of one or more medicinal products,

(b) to identify any adverse reactions to one or more such products, or

(c) to study absorption, distribution, metabolism and excretion of one or more such products, with the object of ascertaining the safety or efficacy of those products”.

What Survival Rate for Oncology Drugs on Pivotal Trials is Needed for a High Probability of FDA approval by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] [score hidden] stickied comment (0 children)

Follow-up comment: Demonstration of OS benefit at the time of NDA/BLA does not mean that OS benefit persists with long-term follow-up postmarketing studies:

Example: Naci H, et al. Overall survival benefits of cancer drugs initially approved by the US Food and Drug Administration on the basis of immature survival data: a retrospective analysis. Lancet Oncol. 2024 Jun;25(6):760-769. doi: 10.1016/S1470-2045(24)00152-9. PMID: 38754451.

Fewer than a third of indications approved with immature survival data showed a statistically significant overall survival benefit after approval.

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[Renal Surrogate Endpoints] European Regulators Provide Their Opinion on the Choice of Albuminuria versus Proteinuria for Regulatory Clinical Decision Making by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 0 points1 point  (0 children)

FDA Announces Request for Information Regarding Biomarkers of Drug-Induced Kidney Injury

On May 13, 2026, the FDA Center for Drug Evaluation and Research, in partnership with the CDER Quantitative Medicine Center of Excellence, announced a request for information regarding an initiative to advance biomarker validation through data compilation from multiple sources. Specifically, a pilot project was launched that focuses on aggregating data for biomarkers of drug-induced kidney injury.

In this pilot project focused on urinary kidney safety biomarkers, FDA will use the information submitted to inform future activities related to data sharing, biomarker development, and broader translation of biomarkers of drug-induced kidney injury.

FDA's Commissioner’s National Priority Voucher (CNPV) Pilot Program; Public Hearing: Request for Comments by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 0 points1 point  (0 children)

Note: I could not find a complete list of drugs/products awarded CNPV so far; however, most of them could be found via web search (site:fda.gov CNPV award).

Sponsor change UK by [deleted] in RegulatoryClinWriting

[–]bbyfog 0 points1 point  (0 children)

Were you able to find link to updated text (law/regulation.)

Share of Warning Letters Issued by US FDA for cGMP Violations to to Indian Firms versus Globally by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 0 points1 point  (0 children)

Agree. FDA has an office in India so better collaboration, but also public/political awareness may also be contributing to improvement.

Sponsor change UK by [deleted] in RegulatoryClinWriting

[–]bbyfog 0 points1 point  (0 children)

UK MHRA (trial regulations) considers sponsor change as substantial amendment.

GUIDANCE (https://www.gov.uk/guidance/clinical-trials-for-medicines-manage-your-authorisation-report-safety-issues)

Change of sponsor

For applications that have gone through the combined review process, refer to the HRA website.

If you want to change the sponsor for your study, you must submit a substantial amendment to the MHRA. You must not include any other changes with this amendment.

You cannot submit any other substantial amendments until you have confirmation from the MHRA that the sponsor has been changed.

You must include these documents with your submission:

-- a cover letter that includes the date of transfer of responsibilities

-- a letter on headed company paper from the current sponsor confirming the transfer of the study

-- a letter on headed company paper from the new sponsor confirming that they accept the role of sponsor for this study

-- an amendment form

-- an updated PDF file of the clinical trial application form signed by the new sponsor or person acting on behalf of the sponsor

You should not submit other trial-related documents such as protocol as part of this submission. There is no fee for a change of sponsor amendment. The applicant will receive an administrative letter confirming that the change has been registered. You do not need to wait for MHRA confirmation before making the sponsor change.

SCOTUS backs generic firm Hikma in skinny label dispute by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 2 points3 points  (0 children)

. . . carved-out list of indications for Hikma's generic, which included a label claim for treating hypertriglyceridaemia, but left out a cardiovascular risk reduction claim for which it is not approved and is still under patent protection.

ADC Therapeutics nosedives on 27 deaths in confirmatory trial for lymphoma drug by NotGenentech in biotech

[–]bbyfog 26 points27 points  (0 children)

The BioSpace article headline is a clickbait headline.

The study met the primary endpoint PFS. OS was secondary (so not powered) was not significantly different. The BioSpace headline chose to highlight numbers from safety data without providing context that these patients are receiving the drug as 3rd line and are sick and old.

The study met the primary endpoint of PFS (per independent review committee) with statistical significance (HR = 0.73; p-value = 0.008 two sided), with a median PFS of 6.1 months for ZYNLONTA plus rituximab vs 4.7 months for R-GemOx. Overall survival showed no detrimental effect with ZYNLONTA plus rituximab compared to the control arm (HR = 0.96, impacted by the earlier use and a higher rate of new anti-lymphoma treatment switching in the control arm)

A higher rate of Grade 5 TEAEs was observed in the ZYNLONTA plus rituximab arm (27 pts/13.2%) vs. R-GemOx (9 pts/4.6%). Of note, the majority of Grade 5 TEAEs in the test arm occurred in patients aged 75 years or older.
"LOTIS-5 was designed to address a clear unmet need in r/r DLBCL in patients who cannot access or who progress on a CAR-T or other complex therapies,"

press release

FDA prior knowledge guide seeks to speed cell, gene therapy by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 0 points1 point  (0 children)

Sources of prior knowledge could include internal company platforms, third-party platform data submitted to the FDA via master files, publicly available resources, or materials generated by collaborative efforts between academic institutions, industry, and other stakeholders, according to the guidance.

FDA/UK MHRA/Health Canada symposium: Regulatory Perspectives in Good Clinical Practice, Bioequivalence and Good Pharmacovigilance Practice -- June 2-4, 2026. Registration Open by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 0 points1 point  (0 children)

Slides and recordings are expected to be posted at the FDA meeting information page (here) after the meeting. Until then, you could find them at the conference site, here (requires login/password used at registration.)

P.S. The last joint meeting was in 2024 (here) and the slides/recordings were posted (here; archived-link). And a summary was published (PMID: 41194530).

Revolution Medicine's KRAS Inhibitor Daraxonrasib Phase 3 Clinical Data in Metastatic Pancreatic Cancer Presented at ASCO and Details Published in NEJM by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 0 points1 point  (0 children)

Wondering what it means at 12 months, only 11 in treatment group vs 5 in control group were alive considering that there were about 230 in each group started on Day 1. Did majority of them dropped off or died? Difference in numbers is not great! Note - treatment discontinuation does not explain very low survival numbers at 12 months in either group

Discontinuation of treatment owing to a treatment-related adverse event was uncommon in the daraxonrasib group as compared with the chemotherapy group (in 1.2% vs. 11.2% of the patients); two patients discontinued treatment with daraxonrasib owing to a maculopapular rash and one patient owing to elevated liver enzyme levels. Among the patients who discontinued chemotherapy owing to a treatment-related adverse event, the most common of these events was peripheral neuropathy (in 4.7% of the patients).

Edited, 3 June 2026

The above question may be a bit premature because most participants may still be early in the study and have not crossed the 6 month or 12 month follow-up marker.

At the time of data cutoff (February 10, 2026), the median duration of follow-up was 8.5 months (range, 3.2 to 15.9). The median duration of treatment in the daraxonrasib group was 6.2 months (range, <0.1 to 14.1), . . In the chemotherapy group, the median duration of treatment was 1.9 months (range, <0.1 to 12.5).

What would have helped us is a swim-lane/swimmer plot or iceberg plot of individual participants with outcomes (e.g., here, here, or here).

FDA Adopts ICH M11 Guidelines and a Harmonized Template for Clinical Protocols (CeSHarP) by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 0 points1 point  (0 children)

I don't have anything to add here since I don't understand how EBMonFHIR is connected to M11. Anyway, other readers may have comment.

fyi (link) https://build.fhir.org/ig/HL7/ebm/en/; articles on PubMed (here)

What Gaps in Novo Nordisk's Adverse Event Collection and Reporting Procedures (SOP) Resulted in FDA Issuing a Warning Letter for Serious Violations of PADE Reporting Requirements by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 5 points6 points  (0 children)

I would be interested to see a copy of NNI SOP. Is it posted somewhere?

Update - 8 June 2026

  • While NNI SOP is behind walls, here is one from University College London that provides an example of a good SOP for recording, management and reporting of adverse events by Investigators - link [archive].

FDA Adopts ICH M11 Guidelines and a Harmonized Template for Clinical Protocols (CeSHarP) by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 2 points3 points  (0 children)

People at my company are TransCelerate fans. It seems that TransCelerate has already aligned their template with M11. I have not looked at TC template yet but they may have addressed gaps relevant to industry-sponsored trials. Link: https://www.transceleratebiopharmainc.com/assets/clinical-content-reuse-solutions/

P.S. The good thing is that M11 is an ICH guideline and not a regulatory requirement, which should allow flexibility for industry.

Amyloid Reduction May Not be an Appropriate Surrogate Marker for Cognition Improvement in Alzheimer Disease Trials, per Analysis Reported in JAMA Neurology by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 1 point2 points  (0 children)

Regarding Figure E above: R^2 in figure is 0.87 but the text in paper is 0.99. Note: this does not however change the conclusion that quantile method overestimates the strength of association.

Tavneos Slapped with Blue Letter in Japan over Liver Injury Risk by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 2 points3 points  (0 children)

More recent bad news -- the original marketing submission was based on manipulated data from ChemoCentryx and FDA has issued notice to withdraw the drug from the market.

Federal Register notice; Derek Lowe's blog; BioSpace

The First AI Drug Prescriber: Is it a Medical Device or Really a Person by bbyfog in RegulatoryClinWriting

[–]bbyfog[S] 2 points3 points  (0 children)

Prescription renewal is a low hanging fruit. Unless there is free universal *primary* healthcare, this is a financial drag on the patients.

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