fda advisory committee meeting: the management of an acceptance test for a new medicine by external...
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FDA Advisory Committee Meeting:
The management of an acceptance test for a new medicine by external stakeholders
Jan W.C.M JansenGlobal Project DirectorAbbott Healthcare ProductsWeesp, The Netherlands
PMI Dutch Chapter Meeting22 Feb 2011
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Who is Jan Jansen?
• Chemistry/biochemistry University of Utrecht
• PhD University of Nijmegen
• Philips Duphar (1979) Solvay Pharmaceuticals Abbott
• Groupleader Vascular Biochemistry
Research Program Manager
Groupleader Biotechnology
Global Project Director since April 2000
leading Project Teams, phase I-II-III studies, Advisory Boards, Advisory Committee, etc
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FDA Advisory Committee Meeting:
The management of an acceptance test for a new medicine by external stakeholders
AGENDA:
- What is an FDA Advisory Committee?
- How to manage the preparation for a FDA AC
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Advisory Committee: focus on safety
An Advisory Committee meeting is normally part
of the review process of a NCE dossier to obtain registration in US
Nowadays for almost every dossier an Advisory Committee
is organized due to the increased focus on safety
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The consequence of submission a dossier
for a new drug application in US
may be to be invited by FDA for an Advisory Committee meeting
This is excellent news:
your drug reached the final review stage.
One hurdle to go!
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Advisory Committee
The Food and Drug Administration (FDA),to assist in its mission to protect and promote the public health,uses 49 committees and panels to obtain independent expert advice on scientific, technical, and policy matters
Advisory committees provide FDA with independent opinionsand recommendations from outside experts on applicationsto market new drugs, and on FDA policies. The marketing applications include data to show the safety and effectiveness of human drugs.The outside experts receive summary information aboutthe applications and copies of FDA's review of the application documents.Based on this information, advisory committees may recommendapproval or disapproval of a drug's marketing application. FDA generally follows an advisory committee's recommendation, but is not bound to do so.
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AC members•Recognized experts
technically qualified experts in their field and have experienceinterpreting complex data. Candidates must be able to analyze
detailed scientific data and understand its public health significance
•Consumera consumer representative must be able to analyze scientific data,understand research design, discuss benefits and risks,and evaluate the safety and efficacy of products under review.It is also essential that the representative have an affiliation withand/or active participation in consumer or community-based organizations
•Industry industry representatives act on behalf of regulated industry at committee meetings.The representative discusses an issue before the committee from the perspectiveof the affected industry and not as an individual from a specific sponsor. Non-voting
•Patient representatives
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AC part of dossier review process
phase IIIclinical studies
several years
dossier completion
6-9 months
Dossiersubmission
Dossier filing
FDA messagefor AC AC
decisionletter
dossierreview process
10—14 months
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What is a dossier?
• A dossier consists of 4 main parts:
• Preclinical part:reports and overall summaryall preclinical studies (pharmacological, toxicological, ADME)
• Pharmaceutical part all required reports on the active ingredient (chemical process) and finished product (pharmaceutical process)
• Clinical partall reports (efficacy and safety) and overall summary
• (proposed) SPC (EU) or TPI (US)describes the wished indication
SmPC: Summary of Product CharacteristicsTPI: Targeted Package Insert
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AC has a fixed agenda
provided by FDA
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AC meeting room
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How to prepare for an (naïve) Advisory Committee?
the company expects an positive result!
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Project Team (P-Team) composition (in most companies)
All compound knowledge
is in this team present
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Givens at the moment of submission
• Project Team (P-Team) - Is responsible for all activities of a certain NCE- Consists of all essential disciples: Clinical, Pharmaceutical, Preclinical, Regulatory Affairs, Marketing, Pharmacovigilance- Reports to Management Governance
• Cooperation with CROs- Clinical and Regulatory Affairs- Pharmacovigilance (risk management plan)
• No in-house experience on ACs
• Between submission and an AC is ca. 12 months
FDA message ca. 3 months before AC!!
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Risk Management Plan Risk Response Response tactics
never tasted the atmosphere
avoid visit an AC at early stage
preparation time too short when waiting for FDA message
avoid start asap after dossier submission
no in house experience with AC
avoid approach a company which is experienced
due to focus on content, no time for organization
transfer idem
due to unknown questions, a lot of slides to be prepared
transfer approach a slide company
in house know-how may be insufficient
accept in house knowledge is far more then external
no adequate well known US clinical expert to assist
avoid select and invite
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First actions
Immediately after submission:
• Contact US based company (PharmApprove™) “PharmApprove teams are helping to prepare for drug and device teams for upcoming Advisory Committee hearings, providing services including strategy development, medical writing, presentation coaching, slide creation, and project management”.
• Compose adequate AC P-Team and Core-Team
• Create clear governance interactions
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US based AC coaching company
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Essential for AC
• How to behave
• What to say
• How to answer
• Which prove/arguments to show
• In fact an AC is a media circus
• Handling the AC-members is key
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P-Team, AC P-Team and AC Core-Team
AC P-Team• Global Project Management• Clinicial• Statistics• Clinical Pharmacology• CRO Clin Pharm• CRO Clin/Reg Affairs• Pharmacovigilance• CRO Pharmacovigilance• Preclinical Toxicology• Preclinical Pharmacology• Preclinical US• Pharmaceutical Dev• Reg Affairs Phar Dev US• Regulatory Affairs US• Marketing• Media
AC Core-Team• Global Project Director• Regulatory Affairs US• Clinical• Marketing
P-Team
• Global Project Director
• Clinical
• Preclinical Toxicology
• Preclinical Pharmacology
• Regulatory Affairs (US)
• Pharmaceutical Dev
• Marketing
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AC Core P-Team/AC P-Team vs. PharmApprove
AC Core P-Team
PharmApproveAC preparation experience
Delivery of:-planning-training-messaging
Delivery of:-knowledge
-kick off- f-t-f meetings
- weekly telecons- ad hoc telecons
organization
AC P-Team
Weekly telecons
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Interaction with Governance Bodies
Executive Committee
decides on strategy
Portfolio Committee
Decides on running activitiesProposals for strategy
P-Teamoperational team
Decides on executionProposals for plan
Involved in strategy
Directinvolvement
ACpreparations
Frequentlyas
presenter
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First key step: KICK-OFF meetingasap after submission
f-t-f ALL members present
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AT Kick-off: Roles and Responsibilities
• Presenters: -Introduction-Unmet medical need-Clinical facts-Risk Management Plan-Risk-benefit-Conclusions-Question handling
• Who in bullpen for answers
• Who instructs slide company for slide projection
• Who is preparing what
• Who interacts with PharmaApprove, Governance Bodies, etc
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Preparation Plan
• Phase A:Period: Feb-JunePrepare for Mock Panel 1Goals:- slide sets- briefing book- presentations- organize mock panel 1
• Phase B:Wait for message from FDAIn case invitation for AC full program towards AC (period 3 months)Goals:- optimized slide sets- final briefing book (ready 2 months before AC)- 2 extra mock panels- additional Q&A training- messaging
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Plan A: preparation until FDA call
December: dossier submission
June: Mock panel 1
January: f-t-f contact with PharmApprove
February: kick off meeting
April: draft Briefing Book ready
March: key messages
May: slide sets ready
FDA call
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Conclusions after Mock Panel 1
Panel: 3 clinical experts, 1 statistician, 1 risk management consultant
adequate panel
Briefing Book too long, to much details decision: rewrite immediately
Presentations too detailed action: focus on messaging
Slides for first round okay; action: be sure to have all aspects covered; more structure required
Key messages too much science action: one-liners
Q&A too detailed action: training
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Plan B: preparation from FDA call until AC
September: - FDA call- restart kick off
Dec (week 2.5): AC
October: - mock panel 2- update Q&A - verbal comments FDA on dossier (end Oct)
November: - early Nov: BB to FDA- mid Nov: mock panel 3- key messages ready
Dec (week 1): - comments FDA (FDA BB)/ adapt presentations if necessary- presentation/answer coaching- adapt slides/ incorporate in Q&A
November (end): - slide sets final/ Q&A ready- know the FDA panel- media plan
Dec (week 2): - adapted slide sets ready
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Conclusions after Mock Panel 2
Panel: 3 clinical experts, 1 statistician, 1 risk management consultant
new adequate panel
Briefing book rewritten: okay, some adaptations
Presentations better however still too detailed; improve messaging
Slides prepare modules
Key messages better; less science
Q&A think out of the box
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Conclusions after Mock Panel 3
Panel: 4 clinical experts, 1 statistician, 1 risk management consultant
new adequate panel
Briefing book okay (was submitted to FDA early Nov)
Presentations okay
Slides okay action: update slide book
Key messages okay
Q&A rehearse, rehearse ….
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Between Mock-Panel 3 and AC ….
• Analyse FDA Briefing Book contains FDA comments on your dossier
- adapt Q&A- incorporate in slide set
• Analyse members AC- what’s their specialism?- what type of questions did they ask previously?- How do they behave, formulate questions?
• Q&A: rehearse, ………
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The AC meeting
• Exciting everyone knows “you have one chance”
• Self-reliance is key
• Correction not possibleIt’s like a soccer team which starts to play the match you hope they act according your instructions
• Voting after 9 hours
• It is an advice but difficult to change!
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Overall conclusions
• To be invited for AC is the route for success!
• Start as soon as possible: always too less time!
• Be clear and fair from start to finish
• Don’t focus on costs but on quality!
• It is a US event!Don’t think you can do the job with Europeans only
• US KOLs are essential
• Focus on AC only for last 4 months (no other activities)
• Believe in your product and show this!
• Stay quiet, be relaxed and convinced.
Thank you!
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Slide Content Area :Slide Content Manager:
Q # Category Issue / Question area Response Responder Slides
Q&A grid
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Know your AC members: complete CV
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AC meeting room
company expertsmax 15 persons
Slide deck
screen
FDAA
C m
embe
rs
AC membersA
C m
embers
Audienceca. 100 seats
presenter
FDA
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