WSQMS Homepage


Sep 2, 2020

Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation
On 28 August 2020 the FDA announced the availability of the draft guidance for industry and FDA staff, and other stakeholders.

The U.S. FDA encourages the collection, analysis, and integration of patient perspectives in the development, evaluation, and surveillance of medical devices. Patients’ perspectives on living with their health condition and its treatment or management are most useful in medical device evaluation when they are relevant to the regulatory decision and reliably measured. Patient-reported outcome (PRO) instruments facilitate the systematic collection of how patients feel, function, and survive as valid scientific evidence to support the regulatory and healthcare decision-making process. By integrating patients’ voices throughout the total product lifecycle, concepts important to patients can be considered in the evaluation and surveillance of medical devices.

PRO instruments allow for collection of certain data as valid scientific evidence of safety and/or effectiveness which is complementary to other evidence of clinical outcomes and/or biomarkers. Use of PRO instruments is generally voluntary but may be specifically recommended in certain standards and guidances. PRO instruments can include patient diaries, visual analog scale (such as measures of pain severity), symptom measures, as well as multi-item, multidomain questionnaires measuring aspects of health-related quality of life (HRQOL). A PRO can be measured by self-report or by an interview, provided that the interviewer records only the patient’s response. Symptoms and unobservable concepts known only to the patient (e.g., pain

intensity and anxiety level) can be measured using PRO instruments. A PRO instrument can be used in clinical studies to measure the effects of a medical intervention or changes in the health status of a patient.

Read more online 

Clinical and Nonclinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential Questions and Answers
On 27 August 2020 the ICH E14/S7B Implementation Working Group (IWG) announced the availability of the ICH E14/S7B Draft Q&As.

ICH S7B and ICH E14 describe nonclinical and clinical risk assessment strategies to inform the potential risk for proarrhythmia of a test substance and contribute to the design of clinical investigations. Emergent data over the past several years demonstrate that different experimental results can arise for the same compound as a function of the study conditions used in nonclinical assays.

The ICH E14/S7B draft Q&A document on “Clinical and Nonclinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential” reached Step 2b of the ICH Process on 27 August 2020 and now enters the consultation period.

On 15 and 16 October 2020, a public webinar (ICH WG Presentations and Live Q&A) will be held to provide an overview of the high-level principles and rationale behind the new interconnected Q&As to ICH E14 and S7B and to answer questions received during the webinar.

Read the pdf 

Quick Interactive Guide to IRIS Registration Process
On 25 August 2020 the EMA announced the availability of the quick interactive guidance.

IRIS is the EMA’s secure online platform to carry out certain regulatory procedures with EMA. It is a core part of EMA’s digital transformation process. EMA plans to extend the platform to cover other regulatory and scientific procedures. IRIS aims to make the handling of product-related regulatory procedures more efficient and user-friendly and to ensure better data quality through integration with other EMA systems such as the substance, product, organisation and referential (SPOR) portal.

Read the pdf 

Evaluating Cancer Drugs in Patients with Central Nervous System Metastases
On 25 August 2020 the FDA announced the availability of the draft guidance for industry.

The purpose of this guidance is to describe FDA’s recommendations for clinical trial designs of cancer drugs or biological products regulated by CDER and CBER that are intended to support product labeling describing the antitumor activity in patients with central nervous system (CNS) metastases from solid tumors originating outside the CNS.

FDA’s current thinking regarding inclusion of patients with brain metastases in clinical trials is addressed in the guidance for industry Cancer Clinical Trial Eligibility Criteria: Brain Metastases.

Read more online 

Manufacturing, Supply Chain, and Drug and Biological Product Inspections During COVID-19 Public Health Emergency Questions and Answers
On 19 August 2020 the FDA announced the availability of the guidance for industry.

FDA plays a critical role in protecting the United States from threats such as emerging infectious diseases, including the Coronavirus Disease 2019 (COVID-19) pandemic. FDA is committed to providing timely guidance to support response efforts to this pandemic.

FDA is issuing this guidance to provide answers to frequently asked questions about regulatory and policy issues related to inspections, pending drug applications, and changes in manufacturing facilities for approved pharmaceutical products.

Read more online 

Compliance Policy for the Quantity of Bioavailability and Bioequivalence Samples Retained Under 21 CFR 320.38(c)
On 18 August 2020 the FDA announced the availability of the guidance for industry.

This guidance describes FDA’s compliance policy related to the retention of reserve samples of the test article and reference standard used in an in vivo bioavailability (BA) and in vivo or in vitro bioequivalence (BE) study. Specifically, this guidance:

  • Addresses the requirement at 21 CFR 320.38(c) to retain reserve samples of sufficient quantity to permit FDA to perform five times all the release tests required in an application or supplemental application.
  • Describes the conditions under which the Agency does not generally intend to take enforcement action against an applicant or contract research organization (CRO) for retaining less than the quantity of reserve samples of the test article and reference standard that were used in the BA or BE study as specified in 21 CFR 320.38(c).

Read more online 

Acute Myeloid Leukemia: Developing Drugs and Biological Products for Treatment
On 17 August 2020 the FDA announced the availability of the draft guidance for industry.

The purpose of this guidance is to assist sponsors in the clinical development of drugs and biological products for the treatment of acute myeloid leukemia (AML). Specifically, this guidance addresses FDA’s current thinking regarding the overall development program and clinical trial designs for the development of drugs to support an indication of treatment of AML, including indications limited to an individual phase of treatment (e.g., maintenance, transplantation preparative regimen, etc.).

Read more online 


You receive this newsletter, because you have subscribed to it.
If you no longer want to receive this newsletter, you can easily leave the list at this Website. You can't view the images in this e-mail? Click here to open it in your browser.

Widler & Schiemann AG - Baarerstrasse 75 - CH 6300 Zug, Switzerland - VAT number: CHE-472.215.777 MWST - Public Limited Company according to Swiss Common Law - Managing Partner: Dr. Beat Widler, Dr. Peter Schiemann - +41 41 558 9193 - -