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Scientific AdvicePart of an overall strategy

BiopeopleRegulatory Scientific Advice Seminar

Prof Steffen Thirstrup, MD, PhDDirector, NDA

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Why seek scientific advice?

• Lack of guidance documents– New therapeutic area– Orphan condition (protocol assistance)

• ’Inappropriate’ guidance documents– Different population– Change in scientific opinion

• Endpoints– Etc.

• Potential use of expedited pathways– Conditional approval

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Scientific Advice is not

• A substitute for the responsibility of the company to due proper product development

• A pre-evaluation (pre-assessment) of data for future MAA

• A guarantee for a future positive benefit:risk and approval

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Whom to consult?

• National agencies– Dependent on national expertise/interest– A potential to vet your briefing book– Generate interest in becoming Rapporteur

• EMA– Scientific Advice Working Party (SAWP)– Potential input from other scientific committees, e.g. PDCO, COMP– Option for a pre-submission meeting

• HTA bodies– Parallel Regulatory-HTA advice

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Does it help you?

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Nature Reviews Drug Discovery 2015; 14: 302–3

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Other considerations- Costs

• National Advice– Moderate (~ 5.000 €)

• Centralised Advice> 40.000 €

• PIP related SA free• 75% fee reduction for orphan drugs• 90% fee reduction for SME

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Other considerations- PRIority MEdicine

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By failing to prepare, you are preparing to fail

[Benjamin Franklin (1706 – 1790)]

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https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/european-medicines-agency-guidance-applicants-seeking-scientific-advice-protocol-assistance_en.pdf

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https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/dates-2020-scientific-advice-working-party-sawp-meetings-deadlines-submission-scientific-advice/eunethta-requests_en.pdf

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SA pre-submission meeting is optional

To be requested by Applicant

An opportunity to: Introduce the proposed development program - and get initial feedback Get feedback on questions and draft briefing package Identify additional issues to be included in the request Discuss specific questions regarding the SA procedure Ask regulatory questions Establish contact with the SAWP coordinators and EMA Scientific Officer involved with the

procedure as it proceeds

Consider especially if it is first SA on the particular project It takes an additional month – so needed or not?

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De-risking strategies

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Why engage with regulators?- What’s the benefits?

• Regulatory Agency Interaction Plan– Determine the adequacy of the current development program– Anticipate and address potential issues during development– Determine feasibility for Accelerated Approval Procedures (CMA, PRIME,

etc)– Address important requirements, e.g. PIP, ODD, SME– Payer- and HTA feedback

• Scientific Advice– EU/US/joint?– Joint with HTA?Robust data generation to support B/R (and HTA)

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De-risking strategies- HTA

• Detailed Market Understanding– Payers and payment models– Organisational requirements for implementation– Legal issues

• Agree on valid historical controls

• Agree on endpoints for HTA

• Characterise the uncertainly of future effect

• Map the impact on health care system organisation incl. need for new equipment

• Patient registry as part of development

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Meeting preparation and frequent deficiencies

• Timing in overall development - Setting your strategy• Defining the goal of the meeting - Setting expectations• Content of the information for the meeting

– (data to be shared with the agency / or presented at the agency)

• Questions• Presentation• Agenda • Attendees

– Wrong or too many

• Rehearsal incl moderation of the meeting• Minutes of the meeting• Analysis of outcome / follow up / implementation

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Early dialogue between regulators and HTA-bodies