2022 Round Table Details

Table # Speaker & bio Topic & Description
1

Clare Matti, Associate Director, Regulatory Affairs, The FlexPro Group

 

 

EU Clinical Trial Regulation

A profound change was finally realized in January 2022 when the European Union’s Clinical Trial Regulation (CTR) entered into application with the “go live” of the Clinical Trials Information System (CTIS). The regulation repeals the Clinical Trials Directive and has long been anticipated: since 2014, in fact.  
 
Why the hold up? As you may be aware, the European Medicines Agency, EMA, had been stalled with the attempt to roll out the CTIS online submission platform for clinical trial applications. This, along with an extension from the originally-planned date of May 2020 due to the pandemic, has brought us all the way into the new decade with the Directive intact.
 
And the EU didn’t just flip a switch and say “lights out” on the Directive: there’s a three year period for member states and sponsors to transition systems and procedures for full implementation.
 
Working through the specifics on which clinical trials are impacted, and the lengthy transition period, can be tricky.  While most sponsors are maneuvering through CTIS for the first time in 2022, those with ongoing trials don’t have to change procedures until 2025.  On top of this, changes brought by the CTR may impact current trial requirements and make maintaining these projects daunting.

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3

Cynthia Nolte, RAC, Director Regulatory Affairs
, Strategic Regulatory Service, ICON, plc.

 Cindy has 25 years of experience in medical device regulatory affairs, with a focus on drug delivery systems.  She leads the strategic regulatory affairs team, engaging with regulatory authorities to define regulatory strategies for medical devices and combination products.  Cindy has authored PMAs and 510(k) premarket notifications for a range of complex medical device products and systems.  Cindy also leads teams in MDR and IVDR assessment  projects,  working  with  customers  to  develop  and  execute  compliance  plans  for  their  medical devices  and  in  vitro  diagnostic  products. 

Prior  to  joining  the  company  in  1995,  she  was  a  staff  scientist  at Organogenesis,  Inc.,  where  she  was  involved  in  the  design  and  development  of  cellular  tissue  substitutes.  Cindy is a member of the Regulatory Affairs Professionals Society and the Association for the Advancement of Medical Instrumentation.  She  holds a  B.A. in  biology  from Wheaton College and  a Ph.D. in  biochemistry from Boston University.

MDR/IVDR Technical Documentation Audits – War Stories from the Audit Trenches

 

 

4

Charles Jagun, Regulatory Senior Manager, Merz Aesthetics

Dr Jagun has over 12 years of US, EU, and International experience in medical device regulation. Charles worked as a regulatory professional with ConvaTec and Bioventus in Ostomy, Wounds, Catheters, orthobiologic, and dermal fillers. He graduated with MS in Pharmaceutical Sciences from BRITE-NCCU and a doctorate from Walden with a dissertation on regulation in pharmaceutical companies.

Regulatory Intelligence – Medical Devices

 As product designs, development, and manufacturing change get more innovative, regulatory authorities have also become proactive and alert to guarantee medical device safety and effectiveness. Manufacturers and those involved in the medical device value chain must comply with the dynamic global regulatory landscape, increasing the burden on drug and medical device manufacturers. To effectively keep up to date in product development and lifecycle management requirements, it is incumbent on industry stakeholders to keep internal stakeholders informed with current information on various regulatory changes and competitive intelligence and provide input on submissions and comments on new guidance documents.

Regulatory Intelligence (RI) is the act of processing targeted information and data from multiple sources, analyzing the data in its relevant context, and generating a meaningful output, such as outlining risks and opportunities to regulatory strategy. RI is critical to the overall strategy of medical product organizations as it helps them anticipate and manage global regulatory developments through a dedicated process. The RI function removes the burden of conflicting responsibilities, priorities, and uncoordinated and disconnected regulatory information from different sources.
In this discussion, I will provide insight into regulatory intelligence to address the challenges of the steady flux of information in the medical device sector, which is critical to meet these previously highlighted challenges. I will demonstrate that despite recognizing the essential role of RI in the survival of medical products companies, many companies find it challenging to allocate much-needed resources to RI or even make provision for one. The talk will center on critically understanding and developing RI function and process.

5

Shilpa Gampa, Delivery Manager, Medical Devices and IVD's Division, Freyr Inc

Shilpa Gampa has been working as a regulatory professional for over 10+ years with rich experience in wide variety of medical device product categories. She has supported organizations ranging from small start-ups to multi national companies for various regulatory requirements.

FDA medical device regulations and Submissions 

 

6

Cheng Li, PhD; Senior Principal Engineer at Aerie Pharmaceuticals

Cheng Li  currently focuses on developing and commercializing innovative devices and combination products in eye care.  She is passionate about integrating her technical expertise in device design and materials with 15 years of experience in product development, CMC, and RA/QA roles to bring innovative devices and combination products to patients.

Combination Product Design Controls/QbD—Does it apply to you?

 Do you work in the pharmaceutical or biotech industry? Do you or your organization play a key role in new or sustaining combination product development projects? Are you a follower, influencer (or both) of the evolving US and global regulatory landscape re: drug/biologics/medical device/combination products? If your answer is yes to any of the above questions, join me in an important discussion of Quality by Design and Design Controls, and how these principles play into development of combination products that lead to successful programs. We will raise urgent questions, share challenges, and collectively discuss lessons learned and best practices to brainstorm potential solutions and pathways.  

7
Drusilla Scott, PhD, RAC, VP Regulatory Affairs,

Sobi, Inc.

Drusilla Scott, PhD, RAC, has more than 30 years of experience in drug regulatory affairs, and is currently at Sobi, Inc., a specialty pharmaceutical company that is building a portfolio of differentiated medicines for patients with rare conditions.  She has worked in a number of companies as a principal in constructing clinical regulatory strategies and submissions for the development of products across a range of therapeutic areas, with a focus on anti-infective drugs. She is also a certified paralegal with primary interests in administrative law and intellectual property.

Manna from Heaven - It's PDUFA VII

On September 30, 2022, Congress enacted legislation to reauthorize the FDA user fee program for prescription drugs and biologics (PDUFA), as well as the programs for medical devices, generic drugs and biosimilars. These reauthorizations were included in the Continuing Resolution that funded the federal government until December 16, 2022.
While the legislation included short-term extensions for some FDA programs that were set to expire, it did not incorporate any of the substantive policy riders,such as accelerated approval reforms, that had advanced in discussions. These proposals may be revisited in the congressional lame duck session.
We will review the “skinny” user fee reauthorization legislation and discuss the new activities that are part of PDUFA’s seventh rendition.

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9

Amy Mihalchik, PhD, DABT, RAC
Senior Scientist II, ToxStrategies

Kirsten Mease, BS Senior Managing Scientist, ToxStrategies

Dr. Amy Mihalchik is a board-certified toxicologist with 7 years of expertise in risk assessment of pharmaceuticals, medical devices, and consumer products. She is a Senior Scientist II at ToxStrategies, a multidisciplinary scientific consulting firm, where she uses quantitative structure-activity relationship (QSAR) modeling to predict compound mutagenicity and target organ toxicities of drug candidates and impurities. She also has expertise in analyzing leachables and extractables data from medical devices and drug container closure systems, and she is familiar with applicable ISO-10993 guidance to support biocompatibility assessments of medical devices.

Ms. Mease is a Senior Managing Scientist with ToxStrategies with more than 20 years of experience in the pharmaceutical industry, focusing on nonclinical biopharmaceutical and pharmaceutical development. During her career, she has accrued extensive experience in the overall drug development process, particularly the nonclinical safety assessment studies required for the development of biopharmaceutical and pharmaceutical products.

Beyond Study Design – Supporting Roles of Toxicologists in Drug Development

 In this discussion, a team of toxicologists will present several case studies highlighting their roles in providing support beyond study design on project teams by focusing on the assessment of drug impurities and formulation excipients throughout development. This conversation will relay the approaches toxicologists take to ensure these components of a drug formulation are given due diligence. We will discuss computational toxicology methods (i.e., QSAR and read-across) used in the evaluation of drug impurities per ICH M7 guidance to emphasize the ways in which these tools may be used to evaluate the safety of data-poor drug impurities in a cost-effective manner, and also examine the role toxicologists play in addressing formulation changes, including identification of risks and possible alternatives to proposed excipients.

 

 

10

Kirsten Messmer, PhD, RAC, Senior Research Analyst, Agency IQ

Kirsten Messmer contributes to the research and development of content for Agency IQ. Prior to joining Agency IQ, she was a Principal Regulatory Affairs Specialist at PPD providing global regulatory intelligence to support efficient, compliant and successful clinical research and drug development for biopharmaceuticals and advanced therapies. 

Advanced Therapies – Lessons learned from regulatory challenges

 Advanced therapies are one of the fastest growth sectors in the biopharmaceutical industry. Several factors contribute or pose unique challenges for the development of these highly innovative products including: a) technologies developed at the bench need translation into clinical research for approval, b) limited patient pool due to aim of treating rare and ultra-rare diseases and personalization, c) special operational and regulatory challenges throughout product development due to evolving knowledge. Regulatory authorities have shown increasing interest and highlighted support mechanisms for these promising products.
This roundtable will discuss some of the challenges and recent issues for developers as well as recent regulatory developments that may have future implications for sponsors developing advanced therapies. Discussion topics include recent clinical holds, regulatory approval decisions, last year’s FDA advisory committee meeting on the adeno-associated virus-based gene therapies and the proposed E.U. regulation on substances of human origin. But feel free to bring your own discussion topics.

11

Mike Lance, Senior Regulatory Affairs Associate, CMC & Labeling; United Therapeutics Corporation

Mike Lance previously had similar Regulatory Affairs roles at ANI Pharmaceuticals, Inc. and Nephron Pharmaceuticals Corp. while simultaneously managing the Drug Safety operations at each company. 

Premarket Safety Analysis with FDA Medical Queries (FMQs)

 Industry professionals often rely on medical queries to characterize adverse events for premarket safety assessment and for postmarket surveillance.  In the premarket setting, investigators utilize Preferred Terms and the over-arching Standardized MedDRA Queries (SMQ) to translate adverse event terms reported by clinical trial subjects into standardized terms; furthermore, MedDRA, the Medical Dictionary for Regulatory Activities, is the standard dictionary for regulators and industry professionals.  These Preferred Terms are grouped within SMQs and are analyzed to determine the frequency of adverse events and, more importantly, the presence of safety signals.  Differences in how terms are reported, how terms relate, and how terms are grouped create potential risks for missed safety signals.  In response, FDA has created the FDA Medical Queries (FMQ) to provide a more standardized grouping of related Preferred Terms to improve safety signal detection in clinical trial datasets.  
With any potential major FDA proposal, industry professionals must weigh the anticipated benefits and costs.  Discussion points at the roundtable will include, but are not limited to, the impact on current adverse event grouping practices and safety signal detection, the impact on labeling for marketing within the US compared to marketing internationally, opportunities for emerging technologies in safety analytics, and negotiating with the FDA regarding divergences in data using SMQs versus FMQs.  FMQs are not currently mandated for clinical development; however, the FDA’s interest in using FMQs clearly indicates that sponsors should strongly consider its adoption to maintain a competitive edge and ensure the timely review of clinical data.

12

Demetrius Carter, MBA, PMP, RAC-US, CLSSGB
,
SVP Services Operations & Regulatory Services; Certara Synchrogenix
;

Making the Switch: Transitioning your career into Regulatory Writing

Are you at a career crossroads? Have you ever considered Regulatory Writing? Very few people enter the field on a direct path but generally transition from other scientific disciplines. Let’s discuss how you can use transferrable technical skills and a passion for writing to develop a new career path.

13

Marissa Berry, Senior Manager of Regulatory Strategy, Hyloris Pharmaceuticals

Marissa Berry is currently also the owner of her own consulting company, Embee Regulatory Consulting, LLC. As part of her work agreement with Hyloris (which is based in Belgium), Marissa started her LLC in 2022 and has fresh knowledge of the LLC startup process! Marissa obtained her Ph.D. in Pharmacology from the University of North Carolina at Chapel Hill.

Scott Kelly, President, Scott Kelly Consulting, Inc.

 

Scott Kelly is the Principal Consultant and President of Scott Kelly Consulting, Inc. established in 2013. Scott obtained his M.A. in Clinical Psychology from East Carolina University, and maintains his licensure with the North Carolina Psychology Board. He transitioned from clinical practice in early 2002 to clinical research where he intentionally set out to be a CRA to gain site level perspective at a global CRO. Since 2006 he has been on the Sponsor side, and has been on 4 NDA teams that have obtained approval by Health Authorities. Since establishing his S-Corp. he has taken on clients of various sizes, indications and phases of drug development & scope of work for both domestic and global studies.

 

The Logistics of Starting Your Own LLC/Consulting Company

 In this discussion, we will talk about the steps necessary for starting an LLC in North Carolina, the potential benefits of filing your LLC as an S corporation, and some of the pros and cons of an LLC vs. being a full-time employee. Marissa will share her experiences since starting her LLC in early 2022, as well as a few tips she's learned along the way.

14

Devin Rosenthal, VP NovaQuest Capital Management

Dr. Rosenthal has broad operational and strategic experience across a range of therapeutic areas, stages of development, and company types within the biopharmaceutical industry. As a member of the NovaQuest Product Finance investment team, he is responsible for investment due diligence, deal sourcing, and transaction structuring.
 
Prior to NovaQuest, Devin was a Senior Consultant at Triangle Insights Group, a boutique life sciences strategy consulting firm, where he focused on corporate strategy, due diligence, mergers and acquisitions, and commercial forecasting. His earlier industry experience includes work in integrated product development and regulatory affairs at Rho;  companion diagnostics at Novartis Oncology; and clinical development at Boston Biomedical Inc, all following postdoctoral training at Tufts University’s Molecular Oncology Research Institute.

Regulatory Affairs Influence in Investment Decision-making

Some may think of regulatory affairs as just a box-checking requirement or a paperwork-heavy burden, but did you know that it’s also foundational for the multimillion-dollar financings that fuel biopharma companies? We’ll discuss how investors view a company’s regulatory aspects when conducting due diligence to determine whether or not to invest. The importance of well-crafted meeting questions, reaching key agreements with regulators, and distinguishing a “review issue” from a review issue will all feature in the conversation. 

15

Kevin B Johnson, Chief Regulatory Officer, Ring Therapeutics

Ring Therapeutics is an emerging biotechnology company focused on harnessing novel anelloviruses for therapies that address critical shortcomings of current gene therapy modalities.

Kevin has 30 years of experience in the implementation of global regulatory and development strategies for a variety of drugs, biologics, combination products, and cell/gene therapies. He has led global project teams in early, mid- and late-stage development programs across a variety of therapeutic areas; directed the preparation and maintenance of INDs, CTAs, BLAs, NDAs, and MAAs; and orchestrated a variety of meetings and communications with regulatory authorities on a global basis.


Storytelling, Hypnosis, and Vulcan Mind Melds:  The Art and Science of Persuasion in Regulatory Interactions

 Influence and persuasion are key skills for successful interactions and negotiations with regulatory authorities. While extensively data-driven, regulatory documents are inherently persuasive in objective and function. Narrative structure allows sponsors to ‘tell the story’ of a therapeutic in development with respect to the perceived benefits and risks to address a target indication. Rapport building, personal connection, and mutual understanding are key aspects of regulatory interactions via both written documents and direct communication channels. This session will build on the mutual experience of the audience to discuss methods and techniques for regulatory professionals to ensure regulatory submissions and meetings are as productive as possible and ideally meet the mutual goals of both sponsor and regulatory authority to successfully develop new therapies for unmet medical needs.