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Rolling Reviews in SAKIGAKE and Breakthrough Therapy Designation
Toshiyoshi TOMINAGA, Ph.D.
Associate Executive Director
Pharmaceuticals and Medical Devices Agency
2
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Disclaimer
Prior Assessment Consultation as Japan’s Rolling Review
Summary• PMDA reviews data piecemeal as they become ready during developmentAim• Issues are found and addressed early to streamline review and decreaser
burden on the applicant
Doc. Submission
Pre-meeting Response
Draft Evaluation report
FinalE. report
40 days 30 days 35 days 15 days
Inquiry
Total:120 work. days
(6 calendar mo.s)Applicant’s Action
PMDA’s Action
NDA Submi-ssion
2011 2012 2013 2014 2015 Approx. Cost (US$)
Phase I study 3 5 2 5 0 36,000
Phase II study 1 1 0 1 0 46,000
Phase II / III study 1 2 3 6 1 72,000
Pre-clinical: toxicity 4 9 2 7 2 21,000
Pre-C: pharmacology5 7 2 8 2 21,000
Pre-C: pharmacokinetics4 9 3 6 2 21,000
Quality 6 8 3 11 1 31,000
TOTAL 24 41 15 44 8180,000-210,000
Pr. Rev. Consultations made and Cost for each category
5
1. This is “Consultation”; not formal submission or review.
When done, formal submission of ALL data required
2. About 50% of Applications accepted, depending on
Human-resources available in the relevant Review
Team
Whether there are more suitable categories of
consultation
3. Does not ensure quicker review; target review time of 9
months (prioritized) or 12 months (regular) is set
independently.
On Prior Assessment Consultation
Survey Results on Applicants completing Prior Review Consultation and formal review (2009-2010 Pilot)
Q4 Fee for Consultation appropriate?Very Cheap 0
Somewhat Cheap 1
Neutral 5
Somewhat Expensive 5
Very expensive 5
Q1 review time shortened?Very much shortened 2
Shortened 3
Neutral 1
Extended 0
Very much extended 0
Q2 Review became efficient?
Made very much efficient 2
Made somewhat efficient 4
Neutral 0
Made somewhat inefficient 0
Made very much inefficient 0
No duplicated review was conducted
6
Partly duplicated 0
Almost all duplicated 0
Q3 Duplicated review avoided?
Rolling Review in SAKIGAKE (Pioneer) Designation Program
【SAKIGAKE Designation Product】
【Ordinary Review】
Non-clinical
Clinical Research
Phase I/II Consul-tation
Phase III Review
Consultation
12 months
2months
Phase I/II Phase III
Consultation Prior ReviewDesignation as SAKIGAKE
6months
1 month
Review
Approval
Non-clinical
Clinical Research
Expedited PathwaysUS JAPAN
Accelerated Approval
- Approval on a surrogate or intermediate clinical end point
(usual practice)
Priority Review - 6-month review- Designation required
- 9-month review period- Designation required
SAKIGAKE/Breakthrough Therapy
- Rolling Review & 6-month review period
- Designation required
- Rolling Review (SAKIGAKE Compre-hensive Consultation)& 6-month review period
- Designation required
Fast Track - Rolling Review & 6-month review period
- Designation required-
Jinsoku (Rapid) Review
-- Specially designated by MHLW
Breakthrough Therapy vs. SAKIGAKE 1Breakthrough Therapy SAKIGAKE
Launch July 2012 April 2015
Criteria - Unmet Needs, Serious or Life-
threatening Conditions- Substantial improvement in
efficacy
- New Mechanism/Action- Unmet Needs, Serious or Life-
threatening Conditions- Substantial improvement in
efficacy
Project Manager - Cross-disciplinary project lead- Senior manager involved
- Review Partner (Concierge)
Priority Review - Not automatically designated - Review within 6 months (shorter than Priority’s 9 months target)
Rolling Review Eligible Eligible (SAKIGAKE Comprehensive Evaluation Consultation
Breakthrough Therapy vs. SAKIGAKE 2
Breakthrough Therapy SAKIGAKE
First NDA in the world
- Intent to develop and file NDA first in Japan (simultaneously in other countries)
Preferably• First-in-human study in Japan• Proof of Concept Study in Japan
Re-examination period
- Maximum 10 years (standard 8 years)
# of Designated Items
Total 130 items111 (CDER), 19 (CBER)
Total 11 items6 Drugs, 3 Regenerative Products, 2 Medical Devices
Approved items 43 (about a quarter of novel drugs approved)
0
SAKIGAKE Comprehensive Evaluation Consultations (simplified)
ApprovalNDA
Submission
Review
1. CMC
2. Non-Clinical
3. Clinical
4. GCP/GLP Compliance
5. GMP Compliance
ca. 4 months
First submission
I/A
(on-site Inspection)Preparation
On-site InspectionP 3
PreparationOn-site Inspection
(on-site inspection)
= Permission to submit
I/A
Inquiry/Answer
6 months
Monthly Consul.with Concierge
12
1. Mandatory for Sakigake-designated products
2. 5 Tracks as 1 package, basically no separate deals
3. Cost: about $ 200, 000
4. Currently (June 2016), monthly consultation going-on,
not yet reached the initiation of the Sakigake
Consultation.
On SAKIGAKE Consultations’ Operation
Rolling Review in Breakthrough Therapy Designation
ApprovalNDA
Submission
In case designated as Priority Review Products
Review
1. CMC
2. Non-clinical
3. Clinical
Cross-Disciplinary Project Lead
6 months
14
1. All-Hands-on-Deck for applicants and reviewer/inspectors
2. Rolling review (or Sakigake comprehensive consultation) is an
essential mechanism to start review/inspection as early as
possible to achieve the ambitious goal.
3. Enabling partnership between applicant and regulator to deliver
drugs to patients.
4. Strain on data obtained in the late stage of development (e.g.
product quality in scaled-up production). Applicants’ efforts
expected.
Findings
Thank You
Dr. Peter Honig
Mr. Jerry Stewart
PMDA SAKIGAKE Team
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