Webinar: The Value of Orphan Drug Designation in your Drug Development Program

child-rare-disease-orphan-drugsAs a drug developer you may wonder if you have a portfolio product or development candidate that qualifies as an orphan drug and thus provides options unique to orphan drug development.

There are approximately 7000 known rare diseases and more expected to be identified in the future and your drug may be effective against a rare disease. A number of these orphan diseases are well known, and the epidemiology well characterized, however, there are still numerous rare diseases still in need of an effective treatment.

This webinar will provide an overview of Orphan Drug Designation and the benefits from both a development and commercial viewpoint. It will explore the benefits of early planning and early engagement with the FDA.

A panel of CATO SMS experts will review the key issues encountered during the development of a product receiving Orphan Designation and explore what challenges and options for sponsors may be seen in the coming months.

Webinar Key Learning Objectives:

  • What unique development options does Orphan Drug Designation offer?
  • What are the typical timelines and milestones in an orphan development program?
  • How can FDA’s development designations (Fast Track, Breakthrough, Accelerated Review, Priority Review) be applied in an Orphan program?
  • What are the regulatory nuances of Orphan Drug Designation as it relates to PREA?
  • Case studies on what has and has not been an effective strategy to gain Orphan Drug Designation

Download this Webinar

Original Air Date:  Tuesday, July 28, 2020
Time:  11:00 AM EST/4:00 PM London

Download Now

Featured Speakers

 
greg-hilemanGregory A. Hileman, Ph.D.,
Vice President, Regulatory Affairs

Dr. Hileman more than 35 years’ research and industrial experience, including 25 years in strategic regulatory affairs. He has led drug development meetings with most FDA divisions and contributed to submission of multiple fast track designation requests, breakthrough therapy designation requests, FDA meeting packages, INDs, NDAs, and BLAs. Dr. Hileman currently leads Cato’s regulatory strategy group and serves as primary FDA contact for multiple sponsors. Dr. Hileman has clinical and product development experience in neurology, psychiatry, oncology, gastroenterology, cardiology and women’s health.

 

 
Will-LeeWilliam Lee, Ph.D., R.A.C.,
Vice President, Regulatory Affiars

Dr. Lee has more than 25 years’ research and industrial experience in gene therapy, cellular immunology, and therapeutic vaccines, including more than 15 years in regulatory affairs. Dr. Lee was involved in the writing of patents and the design and execution of preclinical studies. Dr. Lee has been involved in studies for experimental medications for oncology, peripheral arterial disease, dermatology, and myocardial ischemia. He has also been involved in combination drug/device products. He has been involved in the design of Phase 1 and Phase 2 clinical studies for devices, small-molecule drugs, and biologics. He has also participated in the preparation and submission of various INDs, New Drug Applications, and Food and Drug Administration meeting packages. Dr. Lee has experience in pharmacogenetics and the therapeutic areas of infectious diseases and oncology.

 

Nicholas-Osborne
 
Nicholas Osborne, Ph.D., R.A.C.,
Clinical Writing Scientist, Medical Writing

Dr. Osborne has more than 24 years’ experience in biological and clinical research, with extensive experience in molecular biology, biochemistry, embryology, genetics, epigenetics, and stem cell biology. He has extensive drug and biologic development experience in a variety of therapeutic areas, with particular emphasis on rare diseases.

 
 

On Demand Now – Download Form for CATO SMS Webinar: The Value of Orphan Drug Designation in your Drug Development Program

Confirmation: By checking "Yes", you agree to us contacting you by email and storing your contact details for future contact. Your contact details will be accessible by employees of CATO SMS but will never be shared with or sold to third parties. Also, you may receive infrequent and relevant marketing communications from CATO SMS, but you can unsubscribe within the emails at any time if these communications are not of interest to you.

Webinar: The Value of Orphan Drug Designation in your Drug Development Program

child-rare-disease-orphan-drugsAs a drug developer you may wonder if you have a portfolio product or development candidate that qualifies as an orphan drug and thus provides options unique to orphan drug development.

There are approximately 7000 known rare diseases and more expected to be identified in the future and your drug may be effective against a rare disease. A number of these orphan diseases are well known, and the epidemiology well characterized, however, there are still numerous rare diseases still in need of an effective treatment.

This webinar will provide an overview of Orphan Drug Designation and the benefits from both a development and commercial viewpoint. It will explore the benefits of early planning and early engagement with the FDA.

A panel of CATO SMS experts will review the key issues encountered during the development of a product receiving Orphan Designation and explore what challenges and options for sponsors may be seen in the coming months.

Webinar Key Learning Objectives:

  • What unique development options does Orphan Drug Designation offer?
  • What are the typical timelines and milestones in an orphan development program?
  • How can FDA’s development designations (Fast Track, Breakthrough, Accelerated Review, Priority Review) be applied in an Orphan program?
  • What are the regulatory nuances of Orphan Drug Designation as it relates to PREA?
  • Case studies on what has and has not been an effective strategy to gain Orphan Drug Designation

Register Now for this Webinar

Date:  Tuesday, July 28, 2020
Time:  11:00 AM EST/4:00 PM London

Register Now

 

Featured Speakers

 
greg-hilemanGregory A. Hileman, Ph.D.,
Vice President, Regulatory Affairs

Dr. Hileman more than 35 years’ research and industrial experience, including 25 years in strategic regulatory affairs. He has led drug development meetings with most FDA divisions and contributed to submission of multiple fast track designation requests, breakthrough therapy designation requests, FDA meeting packages, INDs, NDAs, and BLAs. Dr. Hileman currently leads Cato’s regulatory strategy group and serves as primary FDA contact for multiple sponsors. Dr. Hileman has clinical and product development experience in neurology, psychiatry, oncology, gastroenterology, cardiology and women’s health.

 

 
Will-LeeWilliam Lee, Ph.D., R.A.C.,
Vice President, Regulatory Affiars

Dr. Lee has more than 25 years’ research and industrial experience in gene therapy, cellular immunology, and therapeutic vaccines, including more than 15 years in regulatory affairs. Dr. Lee was involved in the writing of patents and the design and execution of preclinical studies. Dr. Lee has been involved in studies for experimental medications for oncology, peripheral arterial disease, dermatology, and myocardial ischemia. He has also been involved in combination drug/device products. He has been involved in the design of Phase 1 and Phase 2 clinical studies for devices, small-molecule drugs, and biologics. He has also participated in the preparation and submission of various INDs, New Drug Applications, and Food and Drug Administration meeting packages. Dr. Lee has experience in pharmacogenetics and the therapeutic areas of infectious diseases and oncology.

 

Nicholas-Osborne
 
Nicholas Osborne, Ph.D., R.A.C.,
Clinical Writing Scientist, Medical Writing

Dr. Osborne has more than 24 years’ experience in biological and clinical research, with extensive experience in molecular biology, biochemistry, embryology, genetics, epigenetics, and stem cell biology. He has extensive drug and biologic development experience in a variety of therapeutic areas, with particular emphasis on rare diseases.

 
 

On Demand Now – CATO SMS Presentation Participation: PCT Virtual Global Summit Presentation Participation

Featured Speakers/Panelist  – (~ 5 hrs and 49 mins into recording)

 
Jack Snyder, M.D., J.D., Ph.D.
Assistant Managing Director, CATO SMS Washington

Dr. Jack Snyder, BS, MD (Northwestern), JD (Georgetown), PhD (MCV), MFS (GeoWash), MPH-MBA-MSIS (Johns Hopkins) currently directs the Washington office of CATO-SMS Research, serves on the Board of Directors of the American Board of Toxicology, holds a New York Certificate of Qualification to direct clinical laboratories, maintains board-certifications in Addiction Medicine, Medical Toxicology, General Toxicology, Clinical Informatics, Occupational Medicine, General Preventive Medicine, Pathology (Anatomic, Clinical & Chemical), Chemistry (Toxicological & Clinical), Quality Assurance & Utilization Review, Legal Medicine, Public Health, and Regulatory Affairs (US, Europe, Canada, Global), and has been certified as a Project Management Professional, Physician Investigator, Physician Executive, and Medical Review Officer.

Listen to Live Recordinghttps://us02web.zoom.us/rec/share/uvQkAr3srE5LG6Pn9UXfCq0BAYLAT6a81CgZrvBbxRlLUcugdJxUgImKEQ-26vNF

 

PCT Virtual Global Summit was a one day journey into the insightful world of clinical trials. A gathering of experts uncover practical advice from industry-leading researchers, peers, and executives. This virtual event was designed to deliver real value-packed insights, connections with fellow professionals and learn strategies you can put to use the very next day.

PEG is excited to share thier first global virtual Precision in Clinical Trials summit series on June 24th, 2020. The Precision in Clinical Trials virtual global summit was a unique platform for users to network and discuss collaboration in order to achieve their outsourcing and operational strategies.

The virtual summit was tailor-made to address the concerns of trial sponsors who are conducting clinical trials at a local and global level. With an innovation-focused agenda and a host of reliable solution providers, the virtual summit was designed to help the online attendees find solutions to their most pressing clinical trial challenges in 2020 and beyond.

PEG’s motto is “Helping create Precision for Medicine by connecting Companies around the Globe”.

 

On Demand Now – CATO SMS Webinar: Future Proofing Inflammatory Bowel Disease Diagnosis & Clinical Trials

Inflammatory Bowel Disease (IBD) encompasses a cluster of disorders that cause chronic inflammation of the gastrointestinal tract. Crohn’s Disease (CD) and Ulcerative Colitis (UC) are the most common forms of this prototypically complex disorder that presents multiple diagnostic and therapeutic challenges. The advent of machine learning, big data, and artificial intelligence has enabled modeling of huge amounts of clinical trial data to enhance development of novel biomarkers and quantitative histopathologic image processing techniques that may, in turn, improve delivery of targeted treatments, monitoring of disease progression, and detection of meaningful clinical outcomes. Pragmatic trial designs focused on effectiveness, rather than traditional trial designs focused on efficacy, are emerging in the deliberations of regulatory bodies as sponsors push for recognition of the value of new investigative tools and approaches.

In this webinar, experts from Perspectum, CATO SMS, and ProPath will review past and current complexities of IBD clinical trials, and will explore how currently available, cutting edge solutions can change the regulatory landscape and future-proof IBD investigations in the not-too-distant future.

Webinar Key Learning Objectives will focus on improved understanding of:

  • Past and present challenges in IBD clinical trials
  • Present and future aspects of imaging and histopathologic diagnosis in IBD trials
  • Current traditional IBD trial designs and future pragmatic IBD trial designs
  • Evolution of FDA expectations regarding applications of machine learning, big data, and AI in IBD product development

Original Air Date:  Tuesday, June 23, 2020
Time:  12:00 PM EST

Download Now

 

Featured Speakers

 
Jack Snyder, M.D., J.D., Ph.D.
Assistant Managing Director, CATO SMS Washington

Dr. Jack Snyder, BS, MD (Northwestern), JD (Georgetown), PhD (MCV), MFS (GeoWash), MPH-MBA-MSIS (Johns Hopkins) currently directs the Washington office of CATO-SMS Research, serves on the Board of Directors of the American Board of Toxicology, holds a New York Certificate of Qualification to direct clinical laboratories, maintains board-certifications in Addiction Medicine, Medical Toxicology, General Toxicology, Clinical Informatics, Occupational Medicine, General Preventive Medicine, Pathology (Anatomic, Clinical & Chemical), Chemistry (Toxicological & Clinical), Quality Assurance & Utilization Review, Legal Medicine, Public Health, and Regulatory Affairs (US, Europe, Canada, Global), and has been certified as a Project Management Professional, Physician Investigator, Physician Executive, and Medical Review Officer.

 

 
Cory Roberts, MD, MBA
CEO, ProPath

Dr. Roberts has keen knowledge of gastrointestinal and hepatic pathology; a skill honed during his decades of practice in clinical and anatomic pathology. As a well-respected public speaker and author, Dr. Roberts has published numerous books, book chapters and peer-reviewed articles on topics such as celiac disease, metastatic hepatocellular carcinoma, eosinophilic esophagitis, and atrophic gastritis and endocrine cell hyperplasia. Other areas of interest include liver/small bowel transplantation, sessile serrated polyps and molecular diagnostics of colorectal cancer.

 

 
Marc Goldfinger, PhD
Product Specialist, Perspectum

Dr. Goldfinger has worked in the field of diagnostic pathology for over 9 years, in both neuroscience and gastrointestinal disease. Dr. Goldfinger has recently focused on integrating artificial intelligence into MR applications and digitized histopathology to better understand hepatobiliary disease and bowel disease in order to identify novel biomarkers that may better stratify and monitor bowel disorders.

 
 

On Demand Now – Download Form for CATO SMS Webinar: Future Proofing Inflammatory Bowel Disease Diagnosis & Clinical Trials

Confirmation: By checking "Yes", you agree to us contacting you by email and storing your contact details for future contact. Your contact details will be accessible by employees of CATO SMS but will never be shared with or sold to third parties. Also, you may receive infrequent and relevant marketing communications from CATO SMS, but you can unsubscribe within the emails at any time if these communications are not of interest to you.

Webinar: Future Proofing Inflammatory Bowel Disease Diagnosis & Clinical Trials

Inflammatory Bowel Disease (IBD) encompasses a cluster of disorders that cause chronic inflammation of the gastrointestinal tract. Crohn’s Disease (CD) and Ulcerative Colitis (UC) are the most common forms of this prototypically complex disorder that presents multiple diagnostic and therapeutic challenges. The advent of machine learning, big data, and artificial intelligence has enabled modeling of huge amounts of clinical trial data to enhance development of novel biomarkers and quantitative histopathologic image processing techniques that may, in turn, improve delivery of targeted treatments, monitoring of disease progression, and detection of meaningful clinical outcomes. Pragmatic trial designs focused on effectiveness, rather than traditional trial designs focused on efficacy, are emerging in the deliberations of regulatory bodies as sponsors push for recognition of the value of new investigative tools and approaches.

In this webinar, experts from Perspectum, CATO SMS, and ProPath will review past and current complexities of IBD clinical trials, and will explore how currently available, cutting edge solutions can change the regulatory landscape and future-proof IBD investigations in the not-too-distant future.

Webinar Key Learning Objectives will focus on improved understanding of:

  • Past and present challenges in IBD clinical trials
  • Present and future aspects of imaging and histopathologic diagnosis in IBD trials
  • Current traditional IBD trial designs and future pragmatic IBD trial designs
  • Evolution of FDA expectations regarding applications of machine learning, big data, and AI in IBD product development

Register Now for this Webinar

Date:  Tuesday, June 23, 2020
Time:  12:00 PM EST

Register Now

 

Featured Speakers

 
Jack Snyder, M.D., J.D., Ph.D.
Assistant Managing Director, CATO SMS Washington

Dr. Jack Snyder, BS, MD (Northwestern), JD (Georgetown), PhD (MCV), MFS (GeoWash), MPH-MBA-MSIS (Johns Hopkins) currently directs the Washington office of CATO-SMS Research, serves on the Board of Directors of the American Board of Toxicology, holds a New York Certificate of Qualification to direct clinical laboratories, maintains board-certifications in Addiction Medicine, Medical Toxicology, General Toxicology, Clinical Informatics, Occupational Medicine, General Preventive Medicine, Pathology (Anatomic, Clinical & Chemical), Chemistry (Toxicological & Clinical), Quality Assurance & Utilization Review, Legal Medicine, Public Health, and Regulatory Affairs (US, Europe, Canada, Global), and has been certified as a Project Management Professional, Physician Investigator, Physician Executive, and Medical Review Officer.

 

 
Cory Roberts, MD, MBA
CEO, ProPath

Dr. Roberts has keen knowledge of gastrointestinal and hepatic pathology; a skill honed during his decades of practice in clinical and anatomic pathology. As a well-respected public speaker and author, Dr. Roberts has published numerous books, book chapters and peer-reviewed articles on topics such as celiac disease, metastatic hepatocellular carcinoma, eosinophilic esophagitis, and atrophic gastritis and endocrine cell hyperplasia. Other areas of interest include liver/small bowel transplantation, sessile serrated polyps and molecular diagnostics of colorectal cancer.

 

 
Marc Goldfinger, PhD
Product Specialist, Perspectum

Dr. Goldfinger has worked in the field of diagnostic pathology for over 9 years, in both neuroscience and gastrointestinal disease. Dr. Goldfinger has recently focused on integrating artificial intelligence into MR applications and digitized histopathology to better understand hepatobiliary disease and bowel disease in order to identify novel biomarkers that may better stratify and monitor bowel disorders.