Drug Development Breakthrough

Therapy

Accelerated Approval

Regulatory Policy

Expedited Review

Priority Review

January 29-30, 2018 // Hilton Crystal City // Arlington, VA

Innovative Regulatory PathwaysRegulatory Strategies to Expedite Patient Care

SUMMIT

Jennifer Dudinak, Pharm.D., Vice President, Global Regulatory Affairs, Head, Pharma Regulatory Therapeutic Team, GLAXOSMITHKLINE

Stephen Hoffman,CEO, SANARIA INC

John Billington, Director, U.S. Public Policy, Vaccines, GLAXOSMITHKLINE PLC

Jayne C. Gershkowitz, Senior Vice President and Chief Patient, Advocate, AMICUS THERAPEUTICS

Mark Stewart, Ph.D., Senior Science Policy Analyst, FRIENDS OF CANCER RESEARCH

Dr. Henrietta Ukwu, Senior Vice President, Head of Global Regulatory Affairs, OTSUKA PHARMACEUTICAL COMPANIES

Alejandro D. Ricart, M.D., Executive Medical Director, Oncology, Early Development Strategy and Innovation, NOVARTIS

Andrew Storey, Vice President, Global Regulatory Strategy, ABBVIE

Conference Chair: Keynote Speaker:

WHO SHOULD ATTENDThis conference is designed for representatives from pharmaceutical, biotechnology, and medical device companies and advocacy groups with responsibilities in the following areas:

• Regulatory Affairs/Strategy/Operations• Regulatory Policy• Business Development/Strategy• Clinical Operations/Development/Affairs• Product Development• R&D/Drug Development• Patient Advocacy• Federal Government Relations• HEOR and Outcomes Research• Strategic Planning• Competitive Intelligence• Oncology Program Management• Rare Disease Program Management• Pipeline/Portfolio Management• Pharmacovigilance• Medical Information/Affairs

This conference is also of interest to:• Regulatory Advisors/Service Providers/

Consultants • Clinical/Contract Research

Organizations• Drug Development Service Providers• Law Firms• Preclinical/Analytical Research

Organizations• Strategic/Management Consultants

VENUEHilton Crystal City at Washington Reagan National Airport2399 Jefferson Davis Hwy Arlington, VA 22202To make reservations, please call 1-800-695-7551 and request the negotiated rate for ExL’s January meetings. You may also make reservations online using the following weblink: http://bit.ly/2ulR93E. The group rate is available until January 9, 2018. Please book your room early, as rooms available at this rate are limited. *ExL Events, Inc. is not affiliated with Exhibition Housing Management (EHM)/ Exhibitors HousingServices (EHS) or any third-party booking agencies, housing bureaus, or travel and events companies. In the event that an outside party contacts you for any type of hotel or travel arrangements, please disregard these solicitations and kindly email us. ExL has not authorized these companies to contact you and we do not verify the legitimacy of the services or rates offered. Please book your guest rooms through ExL’s reserved guest room block using the details provided.

SPONSORSHIP AND EXHIBITION OPPORTUNITIESDo you want to spread the word about your organization’s solutions and services to potential clients attending this event? Take advantage of the opportunity to exhibit, underwrite an educational session, host a networking event or distribute promotional items to attendees. ExL Events will work closely with you to customize a package that will suit all of your needs.

Dear Colleague,

There are regulatory mechanisms in place to accelerate the review and/or approval of treatments for patients. In the United States, Accelerated Approval, Breakthrough Therapy Designation, Fast Track Designation, Priority Review, and the Orphan Drug Act are designed to expedite treatment availability for medications that provide an increased benefit over existing treatments.

Breakthrough Therapy Designations and other expedited pathways encourage sponsors to increase communication and collaboration with the FDA, to enable efficient drug development. Accelerated drug development options are also available globally in ICH countries through programs like PRIME and Sakigake. Understanding how to best utilize these regulatory options is imperative to make an informed decision about the best ways to accelerate drug development.

ExL’s Innovative Regulatory Pathways Summit will bring together leading regulatory experts and thought leaders to discuss breakthrough therapy designations, accelerated approvals, and global regulatory strategies to expedite review/approval of drugs that have promise in treating serious or life–threatening conditions.

Sincerely,

Dario Dario CavaliereConference Production Director ExL Events

Drug Development

Breakthrough Therapy Expedited

Review

8:00 Open Registration and Continental Breakfast

8:50 Conference Chair Opening Remarks Alejandro D. Ricart, M.D., Executive Medical Director, Oncology, Early Development Strategy and Innovation, NOVARTIS

9:00 AbbVie’s Experience With Breakthrough Therapy Designation• Understand the type of data required for BTD eligibility• Optimize the application process and communication with the

FDA• Utilize increased communication to effectively expedite drug

developmentAndrew Storey, Vice President, Global Regulatory Strategy, ABBVIE

9:45 Breakthrough Therapy Designations in Oncology• Provide safe and effective novel oncology drugs to market

quickly for life-threatening diseases and unmet needs• Identify the importance of clinical trial design and clinical

setting: Pivotal study vs. confirmatory study• Discuss surrogate endpoints, well-established surrogate

endpoints, and endpoints that reflect patient benefit• Understand concern over the timely conduct of postmarketing–

study commitmentsAlejandro D. Ricart, M.D., Executive Medical Director, Oncology, Early Development Strategy and Innovation, NOVARTIS

10:30 Networking Break

11:00 Impact of Breakthrough Therapy Designation on Clinical Trial Design • Understand the implications of receiving a designation on

Phase II and Phase III• Collaborate with the FDA to obtain the best clinical data• Clinical data management for expedited drug development• Find the pathway for generating evidence needed about safety

and efficacy in a narrow populationJoseph A. Sliman, M.D., MPH, Chief Medical Officer, SYNTHETIC BIOLOGICS

11:45 Harmonize Manufacture Readiness Plans With Accelerate Drug Development • Discuss the benefits and impacts of increased communication

with the FDA on clinical trial design and CMC plans• Implement and review CMC plans during early clinical

development• Accelerate manufacturing plans in pace with expedited clinical

trial design• Discuss safety and regulatory considerations for accelerated

manufacturingUsha Ramesh, Ph.D., PMP, Executive Director, Regulatory Affairs, CMC, PHARMACYCLICS, AN ABBVIE COMPANY

12:30 Luncheon

1:30 Sakigake Designation System: A Japanese Strategy for Expedited Drug Development• Review a brief history of why the Sakigake Designation System

was derived• Understand the benefits of receiving Sakigake designation• Discuss global development timelines for Sakigake submission• Implement regulatory strategies to obtain clinical data for

Sakigake application• Incorporate Sakigake/PMDA in global development:

engagement, clinical studies, and submission• Regulatory considerations for applying and maintaining

Sakigake designationColin Vechery, Manager, U.S. Regulatory Affairs–Strategy, BIOGEN

2:15 Global Regulatory Options for Accelerated Drug Development • Discuss the challenges and benefits of utilizing FDA and EMA

expedited review pathways• Review the expedited regulatory options in non–ICH regions• Compare the eligibility requirements for breakthrough therapies

and PRIMEPanelists: Jennifer Dudinak, Pharm.D., Vice President, Global Regulatory Affairs, Head, Pharma Regulatory Therapeutic Team, GLAXOSMITHKLINEDr. Henrietta Ukwu, Senior Vice President, Head of Global Regulatory Affairs, OTSUKA PHARMACEUTICAL COMPANIESJerry Stewart, JD, MS, RPh, Deputy Vice President, Scientific & Regulatory Advocacy, PHARMACEUTICAL RESEARCH AND MANUFACTURERS OF AMERICA (PHRMA)Lawrence Liberti, MSc, Executive Director, CENTER FOR INNOVATION IN REGULATORY SCIENCE (CIRS)

3:15 Networking Break

3:45 Coordinating Global Submissions: How to Reuse Content for Emerging Markets• Harmonize regulatory operations to submit to multiple global

regulatory bodies simultaneously• Discuss Eisai’s experience reducing delays by implementing

global strategies• Learn timesaving options to maximize submission efficiency to

the FDASophia A. Kourliouros, Senior Manager, Global Regulatory Operations, EISAI INC.

4:30 Interact Effectively With the FDA to Accelerate Development of Novel Malaria Vaccine Candidates for a Global Unmet Need• Discuss the global impact of a whole-parasite malaria vaccine• Collaborate with regulatory bodies to enable clinical

development in the developing world• Communicate with the FDA regarding global health

considerationsStephen Hoffman, CEO, SANARIA INC

5:15 Day One Concludes

KEY

NO

TE

CA

SE S

TUD

Y

EISA

I C

ASE

STU

DY

CA

SE S

TUD

Y

PAN

EL

Day One Monday, January 29, 2018

12:30 Luncheon

1:30 Integrate Patient Advocacy Groups Into Drug Development Programs to Navigate the Expedited Regulatory Options• Compare eligibility requirements across available expedited

pathways• Examine the benefits and challenges of creating patient-

focused product development plans• Discuss best practices for engaging advocacy groups in early

stages of development• Consider the impact of BTD on the FDA resource allocationBrian E. Harvey, M.D., Ph.D., Executive Vice President, Scientific and Regulatory Affairs, GLOBAL LIVER INSTITUTE; Former Vice President U.S. Regulatory Strategy, PFIZER

2:15 Effective Collaboration Between Drug Developers and Patient Advocates • Utilize data-driven insights to understand the impact of the

patient perspective on regulatory strategies• Establish endpoints that are both clinically significant and

meaningful for the patient• Maximize clinical development efficiency through patient-

focused drug developmentModerator: Brian E. Harvey, M.D., Ph.D., Executive Vice President, Scientific and Regulatory Affairs, GLOBAL LIVER INSTITUTE; Former Vice President U.S. Regulatory Strategy, PFIZER Panelists: Jayne C. Gershkowitz, Senior Vice President and Chief Patient Advocate, AMICUS THERAPEUTICSRon Bartek, President and Co-Founder, FRIEDREICH’S ATAXIA RESEARCH ALLIANCE (FARA)Stacy Woeppel, Advocate in Science, SUSAN G. KOMEN

3:15 Networking Break

3:45 Utilize Expedited Review Pathways in Precision Medicine• Identify clinical endpoints based on available treatments and

disease progression• Demonstrate non–progression of the disease to gain BTD

eligibility• Discuss regulatory strategies to earn conditional approval to

expedite treatment availability• Understand the options for expedited review for targeted

oncology therapiesChristopher Adams, CEO, ANDARIX PHARMACEUTICALS

4:30 Implications of Personalized Medicine on Clinical Trial Design and Expedited Review Pathways • Understand the potential role of personalized medicine in

clinical development• Discuss accelerated solutions for patients with unmet needs

using personalized methodologies• Empathize the impact of personalized medicine on regulatory

strategiesChitra Edwin, Ph.D., RAC, Senior Vice President of Regulatory Affairs and Compliance, SPOTLIGHT INNOVATION, INC

5:15 Conference Concludes

PAN

EL

8:00 Open Registration and Continental Breakfast

8:50 Conference Chair Opening Remarks Alejandro D. Ricart, M.D., Executive Medical Director, Oncology, Early Development Strategy and Innovation, NOVARTIS

9:00 Development and Implementation of Whole-Parasite Malaria Vaccine • Discuss the benefits and challenges of working with U.S. and

foreign regulatory officials• Consider the impact of fast-track designation on the review

process• Analyze the eligibility requirements for breakthrough therapy

designationTooba Murshedkar, Senior Director, Regulatory Affairs, SANARIA INC.

9:45 A Public Policy Perspective on Regulatory Policies and Market-Shaping Considerations for Emerging Infectious Diseases • Compare the protocols in place for expedited access to

vaccines during a public health emergency• Discuss the impact of ACIP recommendations on portfolio

decisions and market access for vaccines• Evaluate existing policies to maximize market access in

uncertain markets for infectious disease products• Consider innovative business model proposals for emerging

infectious disease and how they might interact with existingregulatory policies

John Billington, Director, U.S. Public Policy, Vaccines, GLAXOSMITHKLINE

10:30 Networking Break

11:00 Expedited Options for Competitive Generic Therapies • Consider the impact of GDUFA II on earlier access and

increased competition between generic therapies• Understand updated fee structures and effects on small

businesses• Identify the Impact on prioritization and approval of First

Generics• Discuss the effectiveness of the updated submission protocols

and impact on market dynamicsPanelists: Lisa Parks, R.Ph., Vice President, Sciences and Regulatory Affairs, ASSOCIATION FOR ACCESSIBLE MEDICINESNicholas Tantillo, Head, Policy and Regulatory Strategy, SANDOZ INC

11:45 Utilize Patient–Focused Drug Development to Build Clinical Trial Frameworks for Breakthrough Therapy Applications• Discuss the current and potential applications of breakthrough

therapies in oncology and other disease spaces• Understand the role of patients and advocacy groups in

informing effective clinical development programs• Pathways for patient engagement in clinical development

programsMark Stewart, Ph.D., Senior Science Policy Analyst, FRIENDS OF CANCER RESEARCH

Day Two Tuesday, January 30, 2018PA

NEL

EARLY BIRD PRICINGRegister by Friday, December 15, 2017

Conference $1,895

STANDARD PRICINGRegister After Friday, December 15, 2017

Conference $2,095

ONSITE PRICING

Conference $2,295

TERMS AND CONDITIONS: By registering for an ExL Events (“ExL”) event, you agree to the following set of terms and conditions listed below:

REGISTRATION FEE: The fee includes the conference‚ all program materials‚ and designated continental breakfasts‚ lunches and refreshments.

PAYMENT: Make checks payable to ExL Events and write C1010 on your check. You may also use Visa, MasterCard, Discover or American Express. Payments must be received in full by the conference date. Any discount applied cannot be combined with any other offer and must be paid in full at the time of order. Parties must be employed by the same organization and register simultaneously to realize group discount pricing options.

**Please Note: There will be an administrative charge of $300 to substitute, exchange and/or replace attendance badges with a colleague within five business days of any ExL conference.**

CANCELLATION AND REFUND POLICY: If you cancel your registration for an upcoming ExL event, the following policies apply, derived from the Start Date of the event:

• Four weeks or more: A full refund (minus a $295 processing fee) or a voucher to another ExL event valid for 12 months from the voucher issue date.

• Less than four weeks: A voucher to another ExL event valid for 12 months from the voucher issue date.

• Five days or less: A voucher (minus a $395 processing and documentation fee) to another ExL event valid for 12 months from the voucher issue date.

CREDIT VOUCHERS: Credit vouchers are valid for 12 months from date of issue. Credit vouchers are valid toward one (1) ExL event of equal or lesser value. If the full amount of said voucher is not used at time of registration, any remaining balance is not applicable now or in the future. Once a credit voucher has been applied toward a future event, changes cannot be made. In the event of cancellation on the attendees’ behalf, the credit voucher will no longer be valid.

ExL Events does not and is not obligated to provide a credit voucher to registered attendee(s) who do not attend the event they registered for unless written notice of intent to cancel is received and confirmed prior to the commencement of the event.

SUBSTITUTION CHARGES: There will be an administrative charge of $300 to substitute, exchange and/or replace attendee badges with a colleague occurring within five business days of the conference.

ExL Events reserves the right to cancel any conference it deems necessary and will not be responsible for airfare‚ hotel or any other expenses incurred by registrants.

ExL Events’ liability is limited to the conference registration fee in the event of a cancellation and does not include changes in program date‚ content‚ speakers and/or venue.

*The opinions of ExL’s conference speakers do not necessarily reflect those of the companies they represent, nor ExL Events.

Please Note: Speakers and agenda are subject to change without notice. In the event of a speaker cancellation, significant effort to find a suitable replacement will be made. The content in ExL slide presentations, including news, data, advertisements and other information, is provided by ExL’s designated speakers and is designed for informational purposes for its attendees. It is NOT INTENDED for purposes of copywriting or redistribution to other outlets without the express written permission of ExL’s designated speaking parties. Neither ExL nor its content providers and/or speakers and attendees shall be liable for any errors, inaccuracies or delays in content, or for any actions taken in reliance thereon. EXL EVENTS EXPRESSLY DISCLAIMS ALL WARRANTIES, EXPRESSED OR IMPLIED, AS TO THE ACCURACY OF ANY CONTENT PROVIDED, OR AS TO THE FITNESS OF THE INFORMATION FOR ANY PURPOSE. Although ExL makes reasonable efforts to obtain reliable content from third parties, ExL does not guarantee the accuracy of, or endorse the views or opinions given by any third-party content provider. ExL presentations may point to other websites that may be of interest to you, however ExL does not endorse or take responsibility for the content on such other sites.

Group Discount ProgramSAVE 25% PER PERSON WHEN REGISTERING FOUR For every three simultaneous registrations from your company, you will receive a fourth complimentary registration to the program (must register four at one time). This is a savings of 25% per person.

SAVE 15% PER PERSON WHEN REGISTERING THREE Can only send three? You can still save 15% off every registration.

Offers may not be combined. Early Bird rates do not apply.

36OPMTHE FULL SPECTRUM OF PRODUCT MANAGEMENT

36O

Media Partners

q YES! Register me for this conference!

Name: ______________________________________________________________

Title: ________________________________________________________________

Company: ___________________________________________________________

Dept.: _______________________________________________________________

Address: ____________________________________________________________

City: ______________________________________ State:_____ Zip:___________

Email: _______________________________________________________________

Phone: ______________________________________________________________

Fax: _________________________________________________________________

Please contact me:

q I’m interested in marketing opportunities at this event.

q I wish to receive email updates on ExL Events’ upcoming events.

CONFERENCE CODE: C1010

Method of Payment: q Check q Credit Card

Make checks payable to ExL Events.

Card Type: q MasterCard q Visa q Discover q AMEX

Card Number: _______________________________________________________

Exp. Date: _______________________________________ CVV: ______________

Name on Card: ______________________________________________________

Signature: ___________________________________________________________

January 29-30, 2018 // Hilton Crystal City // Arlington, VA

SUMMIT

20+ Accelerated Approvals

50+ Breakthrough

Therapy Designations

24 Organizations

585+ Years of

Experience

22 Speakers

Combined With

and

Representing

Regulatory Strategies to Expedite Patient Care

Innovative Regulatory Pathways