WSQMS Homepage


Jan 19, 2021

Protecting Participants in Bioequivalence Studies for Abbreviated New Drug Applications During the COVID-19 Public Health Emergency
On 15 January 2021 the FDA published the final guidance for industry.

FDA is issuing this guidance to provide recommendations to prospective applicants of abbreviated new drug applications (ANDAs) on ensuring the protection of participants when resuming or initiating bioequivalence (BE) studies conducted to support the approval of an ANDA that have been disrupted during the COVID-19 public health emergency.

This policy is intended to remain in effect only for the duration of the public health emergency related to COVID-19 declared by the Secretary of Health and Human Services (HHS) on January 31, 2020, effective January 27, 2020, including any renewals made by the HHS Secretary in accordance with section 319(a)(2) of the Public Health Service Act (PHS Act) (42 U.S.C. 247d(a)(2)).

Read more online 

EMA Updated Questions & Answers on Signal Management
On 15 January 2021 the EMA published revision 4 of the Q&A on signal management.

Signal management is defined in article 107h of Directive 2001/83/EC, article 28a of Regulation (EC) No 726/2004 and chapter III of Commission Implementing Regulation (EU) No 520/2012.

This document addresses a number of questions which stakeholders, in particular marketing authorization holders (MAHs), may have on the management of safety signals. This revision includes update of the requirements for the submission of additional data (question 11).

Read the pdf 

EMA Updated Guidance on Obtaining and Maintaining a Scientific Opinion on a Medicine for Use Outside the EU
On 14 January 2021 the EMA updated the guidance on promoting parallel application for EU-M4all opinion and centralized marketing authorization procedure.

The EMA provides guidance on obtaining and maintaining a scientific opinion under the 'EU-M4all' procedure (previously known as Article 58) on high priority human medicines intended for markets outside of the EU.

The pre-submission requirements and evaluation procedure for the EU-M4all procedure are similar to the centralized marketing authorization procedure. Applicants may apply in parallel for an EU marketing authorization under the centralized procedure and an opinion under EU-M4all for their medicine to be used outside the EU.

EU-M4all (EU-Medicines for all) medicines can be evaluated in parallel with a centralized procedure, which will result in independent outcomes of EU-M4all opinion and centralized marketing authorization, and get facilitated worldwide access after approval.

The eligibility criteria are:

  • Active substances must be identical and indications comparable
  • Medicines may have different formulations, forms or routes of administration

Read the pdf 

EMA Procedural Advice for Users of the Centralized Procedure
On 13 January 2021 the EMA updated 3 guidance on pre-/post-authorization procedural advice for users of the centralized procedure or for generic/hybrid applications.

These documents address a number of questions which users of the Centralized Procedure may have. It provides an overview of the EMA position on issues, which are typically addressed during the course of Pre-Submission Meetings.

The Agency emphasizes the importance of Pre-Submission Meetings with applicants. Pre-Submission Meetings (which should take place approximately 7 months prior to the anticipated date of submission of the application) are a vital opportunity for applicants to obtain procedural, regulatory and legal advice from the EMA. The product team is available to address any questions MAHs may have regarding their pre-/post-authorization or for generic/hybrid applications.

Read more online 

COVID-19: Potency Assay Considerations for Monoclonal Antibodies and Other Therapeutic Proteins Targeting SARS-CoV-2 Infectivity
On 13 January 2021 the FDA published the guidance.

Due to the current public health emergency, FDA is issuing this guidance to assist sponsors in the development of monoclonal antibodies (mAbs) and other therapeutic proteins for use as COVID-19 therapeutics. A critical quality control measure for these products is the development and implementation of a potency assay(s) adequate to ensure that each lot is consistently produced with the potency necessary to achieve clinical efficacy and that such potency is maintained over the shelf life of the product.

Read more online 

Florida Medical Doctor Pleads Guilty to Conspiring to Falsify Clinical Trial Data
On 8 January 2021 the FDA released press on a sentence of medical doctor falsifying clinical trial data.

A Florida medical doctor pleaded guilty to conspiring to falsify clinical trial data regarding an asthma medication, the Department of Justice announced today.

Dr. Yvelice Villaman Bencosme, 64, of Miami, Florida, pleaded guilty in U.S. District Court for the Southern District of Florida today to one count of conspiracy to commit wire fraud. Bencosme was a licensed medical doctor who served as the primary investigator for clinical trials purportedly conducted at a medical clinic called Unlimited Medical Research (UM Research) in Miami. In pleading guilty, Bencosme admitted that from approximately 2013 to 2016, she participated in a scheme to defraud an unnamed pharmaceutical company by fabricating the data and participation of subjects in a clinical trial at UM Research. The clinical trial was designed to investigate the safety and efficacy of an asthma medication in children between the ages of four and 11. Bencosme admitted that she falsified medical records to make it appear that pediatric subjects arrived for scheduled visits at UM Research, took study drugs as required, and received checks as payment for site visits.

Read more online 

Requests for Feedback and Meetings for Medical Device Submissions: The Q-Submission Program
On 6 January 2021 the FDA announced the availability of the final guidance for industry and FDA staff.

The purpose of this guidance is to provide an overview of the mechanisms available to submitters through which they can request feedback from or a meeting with the Food and Drug Administration (FDA) regarding potential or planned medical device Investigational Device Exemption (IDE) applications, Premarket Approval (PMA) applications, Humanitarian Device Exemption (HDE) applications, Evaluation of Automatic Class III Designations (De Novo requests), Premarket Notification (510(k)) Submissions, Clinical Laboratory Improvement Amendments (CLIA) Waiver by Applications (CW), Dual 510(k) and CLIA Waiver by Application Submissions (Duals), Accessory Classification Requests, and certain Investigational New Drug Applications (INDs) and Biologics License Applications (BLAs) submitted to the Center for Biologics Evaluation and Research (CBER)) (specifically, INDs and BLAs for devices that are regulated as biological products under section 351 of the Public Health Service (PHS) Act).

Read more online 

Safer Technologies Program for Medical Devices
On 6 January 2021 the FDA announced the availability of the final guidance for industry and FDA staff.

The FDA is introducing a new, voluntary program for certain medical devices and device-led combination products that are reasonably expected to significantly improve the safety of currently available treatments or diagnostics that target an underlying disease or condition associated with morbidities and mortalities less serious than those eligible for the Breakthrough Devices Program; for example, this may include devices treating or diagnosing non-life- threatening or reasonably reversible conditions. Devices and device-led combination products are eligible for this program if they are subject to review under a premarket approval application (PMA), De Novo classification request (“De Novo request”), or premarket notification (510(k)), taking into account the specific eligibility factors described in this document. Consistent with the Agency’s statutory mission to protect and promote public health, FDA believes that this “Safer Technologies Program” or “STeP” will help patients have more timely access to these medical devices and device-led combination products by expediting their development, assessment, and review, while preserving the statutory standards for premarket approval, De Novo marketing authorization, and 510(k) clearance. FDA has modeled STeP on the principles and features of FDA’s Breakthrough Devices Program as mandated in section 515B of the Federal Food, Drug and Cosmetic Act (FD&C Act) (21 U.S.C. 360e-3) and further described in the FDA guidance document entitled “Breakthrough Devices Program” (hereinafter referred to as the “Breakthrough Devices Program guidance document”). As resources permit, FDA intends for STeP to incorporate similar features offered under the Breakthrough Devices Program, such as interactive and timely communications, early engagement on Data Development Plans (DDPs), sprint discussions, and senior management engagement.

Read more online 

Human Gene Therapy for Neurodegenerative Diseases
On 5 January 2021 the FDA announced the availability of the draft guidance for industry.

This guidance provides recommendations to sponsors developing human gene therapy (GT) products for neurodegenerative diseases affecting adult and pediatric patients. Neurodegenerative diseases are a heterogeneous group of disorders characterized by progressive degeneration of the structure and function of the central nervous system or peripheral nervous system. These diseases vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases. This guidance focuses on considerations for product development, preclinical testing, and clinical trial design. The guidance also discusses marketing approval pathways for investigational gene therapy products.

Read more online 

IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations
On 4 January 2021 the FDA announced the availability of the draft guidance for sponsor-investigators. Comments may be submitted until 4 March 2021.

This guidance is intended for sponsor-investigators (hereafter referred to as sponsors) developing individualized investigational antisense oligonucleotide (ASO) drug products for a severely debilitating or life-threatening genetic disease. Most often, individuals with such diseases will not have FDA-approved treatment options and their diseases will be rapidly progressing, resulting in early death and/or devastating irreversible morbidity within a short time frame. In these situations, drug development targeted at a larger number of patients with the ASO is not anticipated because of the specificity of the mechanism of action of the ASO drug product combined with the rarity of the treatment-amenable patient population.

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 - -