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Sep 1, 2019

Placebos and Blinding in Randomized Controlled Cancer Clinical Trials for Drug and Biological Products Guidance for Industry
The US FDA issued the final guidance on the use of placebos and blinding in randomized controlled cancer clinical trials on August 27, 2019

This guidance provides recommendations to industry about the use of placebos and blinding in randomized controlled clinical trials in development programs for drug or biological products to treat hematologic malignancies and oncologic diseases. This guidance does not address the statistical analyses that can be considered when data are unblinded in these trials.

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The National People's Congress of China issued the 2nd Revision of Drug Administration Law
The new revised Drug Administration Law of China will be effective since December 01, 2019

On August 26, 2019, the 2nd revised Drug Administration Law of China was approved by the 12th Session of the Standing Committee of China’s National People's Congress, pursuant to Presidential Order No. 31. Effective December 1, 2019, the amended law focuses on people’s health, risk management, addressing problems during the drug development period, and the quality of drugs. Among other things, the amended law improves management systems in order to support drug innovation and optimize clinical trial management, including priority reviews and a conditional approval system. More details about the key changes in this major overhaul of the law can be found online in Chinese at the moment.

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EMA/FDA analysis shows high degree of alignment in marketing application decisions between EU and US
EMA and the US Food and Drug Administration (FDA) are aligned in more than 90% of marketing authorization decisions for new medicines

This is the first analysis by EMA and the FDA that compares the agencies’ decisions related to marketing authorizations. It is one of the findings of a joint EMA/FDA analysis comparing decisions on 107 new medicine applications at the two agencies between 2014 and 2016. The study also looked at applications for which the agencies had differing outcomes in terms of type of approval and indication. The most common reason for diverging decisions at the two agencies were differences in conclusions about efficacy. Differences in clinical data submitted in support of an application were the second most common root of divergent FDA and EMA decisions.

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Questions Pile up for Novartis as Senators Call on FDA to Take Action
The importance of having a robust investigation process in place supported by a clear SOP and structured guidance documents on how to conduct an investigation

Why wait three months to tell the US Food and Drug Administration (FDA) about manipulated data? Why wait two months between documenting an initial issue and opening a nonconformance report (NCR)? And how harshly will FDA act? These questions and more are piling up for Novartis, following the announcement on August 6, 2019 that FDA is investigating the company for manipulating data linked to its $2.1 million gene therapy Zolgensma (onasemnogene abeparvovec-xioi).

The arising issue with data manipulation submitted in the biologics license application (BLA) for Novartis’ gene therapy Zolgensma highlights for any sponsor the importance of having a robust investigation process in place. Conducting such an investigation should be supported by a clear SOP and structured guidance documents. Determining the root causes is the key step. Stating that “human error” or "lack of training" are the cause of the error is not considered as an adequate root cause by a regulator like FDA or MHRA. Based on the identified root causes, one then needs to define and describe the actions, including target completion dates and a responsible person assigned to oversee the successful closure of the CAPAs. When all breaches/issues related investigations are completed, i.e., the root cause of the breach/issue was established, CAPAs were defined and endorsed by Quality Assurance and notified to Competent Authorities. It is essential to confirm closure of the breach/issue. As a last step, the Subject Matter Expert responsible for the oversight on the investigation and CAPA processes ought to finalize the Investigation Report, ensure all signatures are present and documentation is archived properly. With this well-defined process one may avoid such inadequate conduct of an investigation.

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Guidance on substantial amendments to a clinical trial if the UK leaves the EU with no deal
U.K. MHRA Updates Information Pages in the Event of a No-Deal Brexit on August 6, 2019

This guidance covers significant amendments to a clinical trial including changes to the trial sponsor/legal representative, Investigational medicinal product (IMP) certification and importation and amendments to the Research Ethics Committee (REC).

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What’s new in Pharmacovigilance? QPPV Update
EMA published the QPPV Update on August 1, 2019

This issue provides the information on recent developments in EU Pharmacovigilance (PV), relating to medicines for human use, and includes updates on the EU network activities, relevant projects and publications. In this issue, it updates the PV IT systems, PV processes and PV dialogue.

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Guideline on Clinical Investigation of Medicinal Products for the Treatment of Gout (Draft)
EMA updated this Draft with the end of consultation on August 31, 2019

The main aim of the guideline is to address general guidance on the development of medicinal products for the treatment of gout. In this document, guidance is provided on the clinical development of new urate-lowering therapy (ULT) and anti-inflammatory treatment options. The study design, inclusion criteria, primary endpoints and trial duration largely depend on the treatment goal and mode of action of the new drug.

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ICH Global Meeting on ICH E8(R1) Guideline on General Considerations for Clinical Trials
ICH is announcing a public meeting on E8(R1), to be held on October 31, 2019 at 08:30 a.m. to 5:00 p.m. ET in Silver Spring, Maryland, USA, with Webcast option available.

ICH is announcing a public meeting entitled “ICH Global Meeting on E8(R1) Guideline on General Considerations for Clinical Trials.” The purpose of the public meeting is to provide information and solicit input from a broad range of non-ICH Member/Observer stakeholders on the draft revised E8(R1) Guideline “General Considerations for Clinical Trials.”

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General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products Guidance for Industry
Comments and suggestions regarding this draft document should be submitted by October 30, 2019

This draft guidance is intended to assist sponsors of new drug applications (NDAs), biologics license applications (BLAs), and supplements who are planning to conduct clinical studies in neonatal populations. This guidance supplements the FDA draft guidance entitled General Clinical Pharmacology Considerations for Pediatric Studies for Drugs and Biological Products (December 2014), as it addresses general clinical pharmacology considerations in neonates, a pediatric subpopulation. The issuance of this draft guidance on clinical pharmacology considerations for neonatal studies for drugs and biological products is required under section 505(d)(2) of the FDA Reauthorization Act of 2017 (FDARA).

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Post-marketing Safety Reporting for Combination Products
FDA issued the final guidance for industry and FDA staff on July 22, 2019

This guidance addresses certain ways to comply with the final rule on post-marketing safety reporting (PMSR) requirements for combination products that FDA issued on December 20, 2016 (81 FR 92603) and that is codified in 21 CFR Part 4, Subpart B (hereafter the “combination product PMSR final rule,” “final rule,” or “rule”). Although the PMSR regulations for drugs, devices, and biological products share many similarities, each set of regulations establishes distinct reporting requirements, including reporting triggers and timeframes. The rule addresses PMSR requirements for combination products that have received FDA marketing authorization, to ensure consistent and complete reporting while avoiding duplication.

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