DIA 2022 Global Annual Meeting

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-22-518-L04-P ; CME 1.00; RN 1.00

Many disease-specific animal models have been used to test emergent medicines for rare diseases. Appropriate animal models, based on the knowledge of the molecular pathology of the human disease, are a significant element to support the medical plausibility of an orphan designation during the development of orphan medicines for these rare diseases. There is a body of evidence, however, that there is a substantial difficulty in choosing/accessing an optimal model or choosing measurements which would be truly informative of the product’s efficacy. We intend to present a critical revision of preclinical models that may be used to support orphan drug designations in rare infectious and neurologic diseases, which are validated for each disease and to evaluate assays pertinent to the core features of selected diseases or otherwise relevant from the clinical standpoint. The analysis will help identify models with best predictive value as well as those acceptable based on their face value, highlighting the areas of most unmet need where development of better preclinical tools is necessary. We will present also results from a study that has investigated the nature of data submitted to the Committee Orphan Medicines to support medical plausibility of orphan designations for a group of rare and paediatric neurological diseases, where the use of in vivo nonclinical data was significantly higher than clinical data. We will explore the data for several case studies and discuss the importance of the availability and publication of this information in encouraging sponsors to develop innovative medicines in rare infectious and neurologic conditions. Nonclinical appropriate models, assessing disease relevant endpoints, may contribute to increase the translational value of animal models in rare diseases, to accelerate research and the effective development of treatment options.

Learning Objectives

Discuss the importance and value of nonclinical efficacy animal models in rare diseases (neurological and infectious); Apply real-world experience from available historical data to the development strategy using animal models of disease towards an increase into the translational value of these models in drug development for rare diseases.

Chair

Martine Zimmermann, PharmD

Speaker

The Translational Value of Animal Models in Rare Disease: Regulator View
Dinah Duarte, MSc

Perspectives on Evaluating New Tools for Regulatory Use: Organ on a Chip Technology
Donna L. Mendrick, PhD

The Translational Value of Animal Models in Rare Diseases: Sponsor's Perspective
Melissa Haskell, DVM

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-22-547-L04-P ; CME 1.00; RN 1.00

This session will discuss biomarker and/or model development considerations in ASO development.

Learning Objectives

Describe the opportunity non-linear imaging provides to track ASO distribution.

Chair

Yuri Zamostin

Speaker

Harnessing the Power of Multimodal Imaging to Explore ASO Distribution in Cells, Complex In Vitro Models, and Tissue
Steve Hood, PhD

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-22-554-L04-P ; CME 1.00; RN 1.00

Historically, regulators have required sponsors to collect pregnancy safety information in the post-marketing setting through prospective registries. However, pregnancy registries have practical barriers to enrolling subjects and often suffer from the lack of an adequate control group, inadequate power to achieve study objectives, high operational cost, a lack of generalizability, and long timelines before results are available to support clinical decisions. There is increasing opportunity and ability for real-world data and real-world evidence (RWD/E) from claims and electronic health record (EHR) databases to address some of the challenges seen with registries and more efficiently answer the question of drug safety during pregnancy. Retrospective database studies allow for observation of patients representing a broad population – not just those self-selected for registry enrollment or referred by providers, and the larger sample size enables faster accumulation of data and ultimately insight into patient safety. This session will discuss various fit-for-purpose pregnancy study options to inform a future framework that includes prospective registries, retrospective database studies, single-arm safety studies or enhanced pharmacovigilance with criteria dependent on the patient population in scope. In addition, the recent EMA guideline on good pharmacovigilance practices for pregnancy prevention programs will be discussed.

Learning Objectives

Discuss fit-for-purpose pregnancy safety study options including prospective registries, real-world data retrospective database studies, single-arm safety studies, enhanced pharmacovigilance or a combination of studies to inform a future framework for pregnancy safety studies; Describe industry, FDA, and EMA current thinking of various study types, criteria, and requirements for post-approval pregnancy safety studies.

Chair

Chelsea O'Connell, MS, RAC

Speaker

FDA Perspective: Postapproval Safety Studies in Pregnant Individuals
Leyla Sahin, MD

Assessing Need and Effectiveness of Pregnancy Prevention Programs: A Crucial Piece of the Jigsaw
Priya Bahri, PhD, RPh

Fit-for-Purpose Pregnancy Safety Study Designs: Considerations for Developing a Framework
Rohini Hernandez, PhD, MPH

Barriers and Future Considerations to Generating Meaningful Safety Data from Post-Approval Safety Studies in Pregnancy
Lockwood Taylor, PhD, MPH

Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-22-559-L04-P ; CME 1.00; RN 1.00

This multidisciplinary panel introduces the most pressing novel ethical considerations facing individualized therapeutics (antisense olgionucleotide) for those with ultra-rare genetic diseases, and suggests ways to address these challenges. Join the DIA Patient Engagement Community for a follow up round table discussion (session #237 RT) on Tuesday, June 21, 12:30pm - 1:30pm.

Learning Objectives

Describe the ethical challenges facing the emerging field of individualized anti-sense oglionucleotide (ASO) therapeutics; Discuss the overlapping roles of treatment and research in individualized therapeutics; Identify ways in which individualized therapies require unique regulatory and development considerations.

Chair

Alison Bateman-House, PhD, MA, MPH

Speaker

Panelist
James P. Smith, MD, MS

Panelist
Sarah Glass, PhD

Panelist
Timothy W. Yu, MD, PhD

Panelist
Edward Neilan, MD, PhD

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-22-576-L04-P ; CME 1.00; RN 1.00

This session will discuss complexities in pediatric drug development including identification of appropriate endpoints and biomarker, challenges in extrapolation from adults and across age ranges, and best practices to overcome key development challenges.

Learning Objectives

Identify key challenges that may delay development of products for pediatric diseases; Discuss via case studies of historical development programs; Identify approaches to overcome challenges including the role of developers, regulators and patient groups.

Chair

Adora Ndu, JD, PharmD

Speaker

Industry Update
Andrew Mulberg, MD

FDA Update
Lynne Yao, MD

Component Type: Workshop
Level: Basic
CE: ACPE 1.00 Application UAN: 0286-0000-22-595-L04-P ; CME 1.00; RN 1.00

Gene therapy research in pediatric patients faces a number of ethical concerns. Trial sponsors must ensure that clinical trials are conducted ethically and balance stakeholder needs and priorities. This workshop will include facilitators from a multidisciplinary working group on ethical issues related to pediatric GT research and distribute realistic cases to groups.

Learning Objectives

Examine common ethical issues currently facing pediatric gene therapy research; Identify how different stakeholder communities may differ in their stances on these issues; Evaluate proposed responses on the part of sponsors.

Chair

Lesha D. Shah, MD, FAPA

Speaker

Facilitator
Emma James, PhD

Facilitator
Cara Hunt, MA

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-22-629-L04-P ; CME 1.00; RN 1.00

Rare disease drug development continues to explode. As a result, development stratgies coupled with regulatory science and policy continues to evolve to keep up. Regulators from FDA will describe key trends and opportunities in this arena.

Learning Objectives

Discuss recent trends in the development and review of medical products for rare diseases; Identify new regulatory tools and guidance to support regulatory interactions around orphan drugs; Discuss how to apply unique strategies to overcome the unique challenges faced in clinical trials for rare conditions.

Chair

James Valentine, JD, MHS

Speaker

Panelist
Patrizia Cavazzoni, MD

Panelist
Peter W. Marks, MD, PhD

Rare Disease Patient Community Perspective
Jennifer Farmer, MS

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-22-646-L04-P ; CME 1.00; RN 1.00

A new class of monoclonal antibodies have reached a milestone in elucidating more on the pathology of AD and this pathology can be affected by treatment to alter progression. Current US HC delivery system for mild-cognitive impairment (MCI) AD patients do not have an efficient, well-established care processes to adopt the new MAB technology which requires care steps not presently being done or coordinated across care settings other than centers of excellence (e.g., Diagnostics such as Positron Electron Tomography, Cerebral Spinal Fluid tap, MRI). The MAB class has focused primarily on studying earlier stages in AD disease progression given the predominant belief that treating progressive neurologic diseases earlier with an efficacious treatment will result in better health outcomes than initiating treatment in later stages of AD. There may be a barrier to adoption if the current health care system will require screening of patients for mild MCI. How can a new technology which can affect the disease process overcome the challenge that anyone who is experiencing mild MCI may be early stage of disease versus dismissed as “getting old”? A panel composed of the relevant stakeholders will have a semi-structured discussion on the access challenges and opportunities of adoption of the new and upcoming class of therapeutic agents for the treatment of the underlying pathology in Alzheimer’s Disease.

Learning Objectives

Identify the access challenges and barriers for adoption of a class of monoclonal antibodies (MAB) which treat underlying pathology in Alzheimer’s Disease.

Chair

Phil Sarocco, MPharm, MS

Speaker

Panelist
Yash J. Jalundhwala, PhD, MS

Panelist
Brian S. Mittman, PhD, MA

Panelist
Tony Grillo, PharmD

Panelist
Darren Gitelman, MD