DIA 2016 Annual Meeting

Component Type: Forum
Level: Advanced
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-523-L01-P; CME 1.25; IACET 1.25; PDU 1.25 PMI 2166-000186; RN 1.25

This forum will provide an overview of traditional small molecule drug development, and highlight special considerations, opportunities, requirements, and risks for specialized programs, such as oncology, vaccines, biologics, or breakthrough therapies.

Learning Objectives

Explain how oncology, vaccine, and biologic drug development are similar to, or different from, a traditional small molecule program; Compare and contrast oncology, vaccine, and biologic drug development with respect to project leadership and project management strategies, approaches, and resource investment decisions; Discuss the impact of breakthrough therapy designation on each of these.

Chair

Eva M. Finney, PhD, PMP

Speaker

Panelist
Susan Uptain, PhD

Panelist
David Robinson, PhD

Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-551-L04-P; CME 1.25; IACET 1.25; RN 1.25

Recently we have seen increased efforts of Asian regulatory harmonization. Hear speakers from Asian agencies and industry and learn how they try to collaborate and harmonize regulations to enable more efficient drug development in this emerging region.

Learning Objectives

Discuss recent changes in drug development regulations in Asia; Describe the Asian regulation trend relating to harmonization with ICH and other global initiatives; Identify how the industry and regulatory agencies collaborate with each other for better and faster drug development by reviewing recent examples of global drug development utilizing multiregional clinical trials including Asia.

Chair

Akio Uemura, PhD

Speaker

Update on Clinical and Regulatory Environment for Drug Development in Korea
Min Soo Park

Rapidly Changing Regulatory Landscape in China
Dan Zhang

Impacts of ICH E17 Guideline in Asian Drug Development
Yoshiaki Uyama, PhD

Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-543-L04-P; CME 1.25; IACET 1.25; RN 1.25

The Breakthrough Designation (US), PRIME (Europe), and Sakigake (Japan) aim to reduce overall drug development times for certain products. This session shows you how to take advantage of these and other global expedited development programs.

Learning Objectives

Compare and contrast global expedited development pathways; Discuss ways to adapt global development programs to take advantage of all expedited pathways.

Chair

Khyati Roberts, RPh

Speaker

The New Japanese “Sakigake” Strategy for Accelerated Development and Approval: Status, Procedure, and Prospects for Industry
Alberto Grignolo, PhD

Strategies for Using the New European PRIME Pathway
Sharon N. Olmstead

Component Type: Symposium
Level: Intermediate
CE: CME 1.25; IACET 1.25; RN 1.25

Over the past decade, several policy strategies have emerged that promise to promote drug development in areas of unmet need, such as exclusivity previsions and advanced market commitments. Recently, one of these policy strategies, the priority review voucher (PRV), has garnered new attention and has re-emerged as a possible driver in R&D for rare pediatric diseases, neglected tropical diseases, and public health emergencies. The PRV program is a regulatory incentive administered by the FDA to promote drug development for certain underserved patient populations. Under the original PRV program, signed into law in 2007, FDA awards a voucher to sponsors for the regulatory approval of a novel drug targeting a particular neglected tropical disease; sponsors that redeem a PRV are subsequently allowed to designate any one NDA/BLA, regardless of indication, as priority review, reducing the target regulatory review period by four months. Since 2007, nine vouchers have been issued by the FDA, and reported sales of the voucher have continued to increase to as much as $350 million. This panel takes a closer look at the value of the PRV as an incentive, focusing on past experiences related to the PRV program, current use, legislative trends, potential value, and its role in encouraging much needed R&D for rare pediatric and neglected tropical diseases.

Learning Objectives

Explain the history and mechanics of the FDA priority review voucher (PRV) program; Describe current policy initiatives regarding the PRV program and industry response to the PRV incentive; Assess the value of the PRV as an incentive for rare and neglected diseases.

Chair

Andrew S. Robertson, JD, PhD

Speaker

Priority Review Vouchers: A New Paradigm for Funding Research?
Patricia R Anderson, RAC

The Priority Review Voucher: The Value, the Pipeline, and the Opportunities for R&D
Andrew S. Robertson, JD, PhD

Priority Review Vouchers: Legislation and History - What You Need to Know
Alexander Varond, JD

Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-16-548-L04-P; CME 1.00; IACET 1.00; RN 1.00

Innovative tools/methods acceptable to drug developers and regulators require focused development strategies for necessary evidence. This session presents case studies illustrating what has been learned to achieve success based on real examples.

Learning Objectives

Discuss the importance of consortia in advancing biomarkers, modeling, and simulation tools that inform drug development; Describe through a lessons learned approach on reaching consensus on the science (e.g. data sharing, governance procedures); Identify and help scientists understand the competencies and expectations involved in development of novel tools and methodologies.

Chair

Martha Ann Brumfield, PhD

Speaker

FDA Perspective
ShaAvhree Y. Buckman-Garner, MD, PhD

Component Type: Session
Level: Intermediate
CE: CME 1.00; IACET 1.00; RN 1.00

In August 2015, the Chinese Food and Drug Administration (CFDA) issued extensive changes to drug reviews and approvals. It has effectively redefined the definition of a new drug and the approval process for any new drug development as well as generics. While China is becoming the second largest pharmaceutical market, it would benefit all firms to carefully review and digest the impact of such a change to their global drug development planning; in turn, this allows them to take advantage of this change and properly adapt. This session will highlight the changes that have a direct impact on the clinical development strategy and share case studies with the audience to illustrate the best approach in handling these regulatory changes.

Learning Objectives

Recognize changes made by the Chinese Food and Drug Administration to drug reviews and approvals; Identify how to best leverage these changes for your new drug developments and generics.

Chair

Dan Zhang

Speaker

Innovative Biopharmaceutical Drug Development in China: Trends and Transformations
Helena Zhang

Regulatory Changes in China
Feng Yi

Outline of Reforming IND/NDA Practice in China
Fudong An

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-16-590-L01-P; CME 1.00; IACET 1.00; RN 1.00

Early consideration of the issues unique to rare disease development allows sponsors to proactively and adequately identify/address them during development and to have productive interactions with Health Authorities (HA). Unique program elements will be compared and contrasted, focusing on regulatory aspects of rare/orphan development and early HA consult to create successful programs that address the particular challenges. We will also discuss how to strategically take advantage of the various US and EU incentives such as market exclusivity, tax reduction, and expedited review process, as well as grants to facilitate the development of orphan drugs, to enhance orphan drug development.

Learning Objectives

Discuss the regulatory requirements unique to the global development of rare disease and orphan drugs, including the regulatory implications; Explain the regulatory considerations to create efficient, feasible, and sufficient rare disease and orphan drug programs; Summarize regulations regarding orphan drug designations in the US and EU; Discuss and demonstrate how drug developers can take advantage of the regulators' incentives to optimize their investment in orphan drug development.

Chair

Maureen Smith

Speaker

Maximizing the Advantages of US and EU Orphan Drug Designation Incentives
Irene Pan

Global Regulatory Considerations in Rare Disease and Orphan Drug Development: When Patients Are Waiting and Everyday Counts
Lauren Peterson Tornetta

Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-545-L01-P; CME 1.25; IACET 1.25; RN 1.25

Expanded access is the use of an investigational drug outside of a clinical trial or study. This forum will provide an opportunity to hear perspectives from regulators, industry, and patients on the use of expanded access in drug development.

Learning Objectives

Describe the different types of expanded access investigational new drugs; Explain why a sponsor might consider expanded access in their drug development programs; Recognize the patient's role and perspective on expanded access programs.

Chair

Kevin Bugin, MS, RAC

Speaker

Introduction and FDA Regulator Perspective
Jonathan P. Jarow, PhD

Industry Perspective on Expanded Access
Kenneth I. Moch, MBA

Patient Advocacy and Expanded Access
Robert Erwin

Industry Perspective on Expanded Access
Anne B. Cropp, PharmD

Component Type: Forum
Level: Intermediate
CE: ACPE 1.50 Knowledge UAN: 0286-0000-16-610-L04-P; CME 1.50; IACET 1.50; RN 1.50

Europe and the US are experimenting with ways to develop therapies based on their actual ‘real world’ performance. This session will discuss the benefits and challenges of outcomes-based health care, as well as the remaining barriers to implementation.

Learning Objectives

Describe the concept of outcomes-based health care; Define real world evidence, Adaptive Pathways, and Precision Medicine; Identify how we can use health data to improve the efficiency of clinical trials and research (better targeting, smaller trials, flexible models); Recognize the links between failures rates, trial sizes, and investments into new therapies.

Chair

Duane Schulthess, MBA

Speaker

European Approaches to Outcomes-Based Health Care
Hans-Georg Eichler

Engineering Outcomes: Driven Biomedical Innovation
Gigi Hirsch, MD

Big Data for Better Outcomes: Innovative Medicines Initiative - IMI Taking the Lead
Richard Bergström, MS

Measuring Outcomes and Performance
Steve Rosenberg

Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-550-L01-P; CME 1.25; IACET 1.25; RN 1.25

Patients with unmet medical needs want early access to medicines. Regulators need to feel confident that the the benefit-risk balance of a medicine they are licensing is positive. Regulators from the EU, Japan, and the US will discuss how access to medicines can be speeded up and the use of postmarketing data to reduce the uncertainties.

Learning Objectives

Recognize the concepts and current progress with speeding up access to medicines in different regions.

Chair

Stella C.F. Blackburn

Speaker

Sooner or Later? Cost and Benefit of Utilizing Rolling Reviews in US and Japan
Toshiyoshi Tominaga

EU Adaptive Pathways Process
Hans-Georg Eichler

Expedited Reviews and Using Postmarketing Data to Supplement Clinical Trials
Gerald J. Dal Pan, MD