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Oct 1, 2018

New FDA Guidance on the use of Blinding and Placebo in Clinical Trials:
Hematologic Malignancy and Oncologic Disease: Considerations for Use of Placebos and Blinding in Randomized Controlled Clinical Trials for Drug Product Development

This guidance provides recommendations to industry regarding the use of placebos and blinding in randomized controlled clinical trials in development programs for drug or biological products for the treatment of hematologic malignancies and oncologic diseases.

Placebos, defined as inert substances with no pharmacologic activity, are commonly used in double-blind, randomized controlled clinical trials. Because investigators and patients in these trials do not know what treatment patients are receiving, this can decrease the likelihood of biased observations, decrease differential patient drop out, and allow for unbiased assessment of outcome measures, particularly when the assessment includes subjectivity, such as for quality of life measures. A placebo design may be useful or preferred in maintenance therapy, add-on trial designs, or in trials of adjuvant therapies (where standard of care is surveillance). However, the use of placebo in double-blind, randomized trials conducted in development programs for drug products for the treatment of malignant hematologic and oncologic disease sometimes presents both practical and ethical concerns.

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FDA issues new Guidance: Adaptive Design for Clinical Trials of Drugs and Biologics
Adaptive Designs will potentially allow sponsors to shorten the drug development timeline significantly

On September 28th, the U.S. Food and Drug Administration (FDA) issued the draft guidance titled, “Adaptive Design for Clinical Trials of Drugs and Biologics” for public comments. This guidance will help sponsors of investigational new drug applications (INDs), new drug applications (NDAs), biologics license applications (BLAs), or supplemental applications develop appropriate adaptive clinical trial designs to support the effectiveness and safety of a drug or biologic. When finalized, this guidance will represent FDA’s current thinking on this topic.

Adaptive clinical designs may streamline drug development programs by saving time and reducing costs. This guidance describes the advantages of adaptive designs in a variety of situations and offers recommendations on the basic principles for designing, conducting, and reporting the results from an adaptive clinical trial, which are critical to the acceptance of adaptive designs in a regulatory setting.

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New FDA Draft Guidance on post-approval Changes to Drug Substances
Considerations for risk management on changes to manufacturing of drug substances and its implications on drug product

This guidance provides recommendations to holders of approved new drug applications, abbreviated new drug applications, new animal drug applications, abbreviated new animal drug applications, and holders of drug master files and veterinary master files who may want to make a change to the drug substance manufacturing process during the drug product application post-approval period. Specifically, it addresses how the risk of one or more change(s) to the drug substance should be assessed and provides recommendations regarding the documentation needed to support such changes for the drug substance, and where applicable, for the drug product made with modified drug substance.

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EMA launches new Corporate Web Site
New EMA Web Site includes many improvements

The website has a number of new features to improve user experience, including:

  • an improved search, allowing users to find content easily and to filter their search results. EMA plans to further refine this functionality in the future;
  • a ‘responsive’ design for cleaner display on mobile devices;
  • simpler URLs based on the location and title of webpages or documents;
  • an updated visual design offering users a clearer reading experience and simpler navigation.

“This new version of our website will help us to further improve our communication with our partners and stakeholders, including the European Commission and our networks, and will support us in reaching out to European Union citizens by providing them with evidence-based and accessible information on medicines”, said Guido Rasi, EMA’s Executive Director.

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Development of medicines for rare diseases
An overview of the EU’s orphan designation program

A disease is considered rare if fewer than five in 10,000 people have it. Around 30 million people in the European Union (EU) suffer from a debilitating rare disease, which means one in 17 people. Finding effective treatment for these rare diseases is a huge challenge.

To find out more about the EU program and the incentives available to developers, check out the infographic.

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EMA’s post-authorization procedural advice for users of the centralized procedure
A Q&A guide (299 pages) to post-authorization procedures issued by EMA

The European Medicines Agency issued a comprehensive catalogue of questions and answers as guide for post-authorization procedures for users of the centralized procedure. It includes the following chapters:

1. Type IA Variations

2. Type IB Variations

3. Type II Variations

4. Extension of Marketing Authorization

5. Grouping of Variations

6. Worksharing of Variations

7. Classification of Changes

8. Pre-submission Queries Service

9. Changing the (Invented) Name of a Centrally Authorized Medicinal Product

10. Annual Re-assessment

11. Renewal

12. Annual Renewal of Conditional Marketing Authorizations

13. Post Authorization Safety Study (PASS)

14. Post Authorization Efficacy Study (PAES) NEW Nov 2015

15. Post Authorization Measures (PAMs)

16. Risk Management Plan

17. Periodic Safety Update Reports (PSURs)

18. Article 46 Pediatric Study Submission

19. Transfer of Marketing Authorization

20. Transparency Revision August 2016

21. Pharmacovigilance System Summary

22. Article 61 Notifications

23. Withdrawn Products Notification

24. Marketing and Cessation Notification

25. Sunset Clause Monitoring

26. Other

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