Optimising the guidance on significant benefit demonstration in the context of protocol assistance Recent developments stimulated through the work of both COMP and SAWP

Industry stakeholder platform on research and development support

Presented by Matthias Hofer on 25 April 2017 Scientific Officer, Orphan Medicines

Glossary: • SB…significant benefit • PA…EMA protocol assistance • SA… EMA scientific advice • PA+SB… EMA protocol assistance procedure with COMP SB question • MA… marketing authorisation • MAA… marketing authorisation application

An agency of the European Union

Orphan environment after 16 years of EU orphan legislation



Success of EU orphan legislation



128 orphan MAs until 2016 and rising



~ 75% require demonstration of SB



Crowded areas, e.g. oncology, haematology, pulmonology



1

Recent developments stimulated through the work of both COMP and SAWP

Importance of adequate data to support SB

Orphan environment after 16 years of EU orphan legislation •

Observations on orphan MAAs in 2016 •

Products that received previous PA+SB kept orphan status





Rise in appeal procedures on COMP SB assessment



More important role for PA+SB

The number of PA+ SB procedures not rising over the last years

2

Recent developments stimulated through the work of both COMP and SAWP

COMP priority is to foster early dialogue on SB via EMA PA procedure Kind invitation for sponsors to use the tool of PA+SB

COMP raises their hands for Rare Disease Day 2017

3

Recent developments stimulated through the work of both COMP and SAWP

When should SB question be asked?

Clinical development Pharm

Non-clinical

I

II

III

MAA

Orphan designation and re-assessment (COMP) SA

4

PA

PA+SB on initial and subsequent indications

Post-mkt

Iterative PA

5



Cumulative body of evidence over time



Updates to clinical development



Changes in regulatory environment, e.g. EC notice 2016/C 424/03



Changes in authorised competitors



PA has reduced fee structure to allow for iterative PA process



PA fee incentives can be combined with SME fee reductions



SB question has to be part of PA procedure with other questions



Combination with other initiatives, e.g. PRIME, HTA parallel advice

Recent developments stimulated through the work of both COMP and SAWP

SB question and COMP answer • Provide clear methodology for demonstration of SB, e.g. indirect/direct comparisons, details on quality of life tools (validation, disease specific, reduction of treatment burden)

• Efforts of the COMP to provide better guidance  Improved SB answer template with a clear conclusive statement  Proactive discussions on hot topics with network support, i.e. indirect comparisons, major contribution to patient care

• If uncertainties remain, or updates are necessary: clarification request or follow up PA 6

Recent developments stimulated through the work of both COMP and SAWP

Who provides COMP SB answer?

COMP-SAWP coordinator as link to EU network

COMP peer review

COMP PA working group

more expertise, better peer-review and more time for critical discussion 7

Recent developments stimulated through the work of both COMP and SAWP

COMP

EMA support •

EMA SA officer: one single point of contact for PA



EMA orphan office





Support for PA presubmission meeting/ PA validation process



General requests

Scientific publication on the scientific assessment and grounds of significant benefit (due to be published in 2017)



Scientific publication on orphan marketing authorisation and protocol assistance (due to be published on 2017)

8

Recent developments stimulated through the work of both COMP and SAWP

Thank you for your attention [email protected] European Medicines Agency

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact

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Presentation - Optimising the guidance on significant benefit ...

Apr 25, 2017 - Industry stakeholder platform on research and development support. Presented by Matthias Hofer on 25 ... authorisation application. Page 2. Orphan environment after 16 years of EU orphan legislation. Recent developments ...

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