[Federal Register Volume 83, Number 22 (Thursday, February 1, 2018)]
[Notices]
[Pages 4660-4661]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-01992]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-N-0001]


Best Practices in Modeling and Simulation for Oncology Products; 
Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration's (FDA, the Agency, or we) 
Center for Drug Evaluation and Research (CDER), in co-sponsorship with 
the International Society of Pharmacometrics (ISoP), is announcing a 
public workshop entitled ``Best Practices in Modeling and Simulation 
for Oncology Products.'' The purpose of the meeting is to discuss 
``best practices'' in integrating pharmacokinetic, pharmacodynamic, 
efficacy, and safety data into models to best inform oncology drug 
development, evaluate disease- and mechanism-specific early endpoints 
to predict long-term efficacy, and discuss potential regulatory 
implications of model-informed decisions in drug development. This 
workshop is also being conducted to satisfy one of FDA's performance 
goals included in the sixth reauthorization of the Prescription Drug 
User Fee Act (PDUFA VI), part of the FDA Reauthorization Act of 2017 
(FDARA), to hold a series of workshops related to model-informed drug 
development (MIDD).

DATES: The public workshop will be held on February 1, 2018, from 8 
a.m. to 5 p.m., Eastern Time. See the SUPPLEMENTARY INFORMATION section 
for registration date and information.

ADDRESSES: The public workshop will be held at the FDA White Oak 
Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great 
Room (Rm. 1503, B and C), Silver Spring, MD 20993-0002. Entrance for 
public workshop participants (non-FDA employees) is through Building 1 
where routine security procedures will be performed. For parking and 
security information, please refer to: http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.

FOR FURTHER INFORMATION CONTACT: Jeannette Dinin, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2108, Silver Spring, MD 20993-0002, 240-
402-4978, email: [email protected]; or Yvonne Knight, Center 
for Drug Evaluation and Research, Food and Drug Administration, 10903 
New Hampshire Ave., Bldg. 51, Rm. 2142, Silver Spring, MD 20993-0002, 
301-796-2133, email: [email protected].

SUPPLEMENTARY INFORMATION: 

I. Background

    Under FDARA, FDA agreed, in accordance with section I of the PDUFA 
VI Performance Goals, Ensuring the Effectiveness of the Human Drug 
Review, part J, Enhancing Regulatory Decision Tools to Support Drug 
Development and Review, to convene a series of workshops to identify 
best practices for MIDD (https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM511438.pdf). FDA is conducting this 
workshop as part of the MIDD workshop series.
    Over the past few decades, there has been extensive investment in 
oncology drug discovery and development. Despite greater understanding 
of disease biology and drug mechanisms of action, further progress in 
model-informed strategies is needed to continue advancements in 
oncology drug development. Innovations in clinical trial design 
utilizing more informative endpoints could help bring more effective 
treatment options to cancer patients faster by accelerating development 
of effective new drugs and reducing failure rates in expensive late-
phase development.
    As more effective and complex combination strategies and novel 
targets for cancer treatment evolve, exploring more informative and 
predictive endpoints to assess treatment response

[[Page 4661]]

(e.g., response evaluation criteria in solid tumors-based endpoints 
(RECIST)) has become an active area of research. Alternative metrics 
that require shorter periods of observation or provide more precise 
assessment of treatment effects could lead to more rapid completion of 
clinical trials and require fewer patients. Promising among these 
alternative metrics are model-based metrics, such as those based on 
longitudinal continuous tumor size measurements. Additionally, model-
informed approaches can help satisfy a need to optimize dosing regimens 
for patients. Investigations to refine dosing regimens often occur 
after new drug approval and/or are driven by pharmacometric modeling 
approaches. There is growing interest in using model-informed 
approaches to help balance the risks and benefits of oncology products 
by identifying optimal dosing regimens, and broad stakeholder 
engagement and discussion around this topic can be beneficial.

II. Objectives

    The objectives of the workshop are to:
    1. Discuss ``best practices'' in integrating human pharmacokinetic, 
pharmacodynamic, efficacy, and safety data into models that best inform 
oncology drug development.
    2. Describe novel imaging techniques and diagnostic and predictive 
biomarkers that may be utilized in oncology drug development.
    3. Describe disease- and mechanism-specific early endpoints to 
predict long-term efficacy.
    4. Evaluate the potential to shift from traditional RECIST-based 
endpoints such as Overall Response Rate (ORR) and Progression Free 
Survival (PFS) to modified RECIST approaches (e.g., imRECIST for 
immunotherapies) as well as to other (model-based) tumor kinetic 
metrics to support early decision making in Phase 1/2 as well as in 
confirmatory trials.
    5. Discuss potential regulatory implications of model-informed 
decisions in drug development, including, model-based target 
identification, dose/exposure justification based on preclinical 
evidence, dose selection for first-in-human trials, quality by design, 
early clinical study design, dose finding/titration, confirmatory 
trials, product labeling, and post-marketing studies.
    A detailed agenda will be posted on the following website in 
advance of the workshop: https://www.fda.gov/downloads/Drugs/NewsEvents/UCM589458.pdf.

III. Registration and Accommodations

    Registration: Persons interested in attending this public workshop 
must register online by January 31, 2018, at https://fdaoce.formstack.com/forms/isop. Please provide complete contact 
information for each attendee, including name, title, affiliation, 
address, email, and telephone number.
    Registration is free and based on space availability, with priority 
given to early registrants. Early registration is recommended because 
seating is limited; therefore, FDA may limit the number of participants 
from each organization. Registrants will receive confirmation when they 
have been accepted. If time and space permit, onsite registration on 
the day of the public workshop will be provided beginning at 8 a.m.
    If you need special accommodations due to a disability, please 
contact Yvonne Knight (see FOR FURTHER INFORMATION CONTACT) no later 
than January 24, 2018.
    Streaming Webcast of the Public Workshop: The meeting will also be 
webcast. A live webcast of this workshop will be available at https://collaboration.fda.gov/fdaisop/ on the day of the workshop. If you have 
never attended a Connect Pro event before, test your connection at 
https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. 
To get a quick overview of the Connect Pro program, visit https://www.adobe.com/go/connectpro_overview. FDA has verified the website 
addresses in this document, as of the date this document publishes in 
the Federal Register, but websites are subject to change over time.
    Transcripts: Please be advised that as soon as a transcript of the 
public workshop is available, it will be accessible at https://
FDAOCE.formstack.com/forms/isop. It may be viewed at the Dockets 
Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers 
Lane, Rm. 1061, Rockville, MD 20852.

    Dated: January 29, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-01992 Filed 1-31-18; 8:45 am]
 BILLING CODE 4164-01-P