0. Drug development in Parkinson’s disease
The regulatory perspective, and how can we help coping with the bottlenecks
Presented by: Pavel Balabanov, MD PhD
Human Medicines Evaluation Division,
Scientific and regulatory management department,
CNS and ophtalmology

1. Main topics
Who are we at EMA and what is our regulatory experience in Parkinson’s disease (PD)
Initiatives available at EMA to stimulate and support new drug development in PD:
What can we offer?
When and what you should use the different initiatives for?
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2. The European Medicines Agency –who are we?
Responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union.
Main task – the centralized procedure
but also:
A significant role in stimulating innovation and research in the pharmaceutical sector:
Publishing guidelines on the requirements for the quality, safety and efficacy testing of medicines
Providing scientific advice to companies on the development of new medicines;
special assistance to micro, small and medium-sized enterprises (SMEs);
Issue opinions on orphan designation for medicines for rare diseases;
Innovation task Force (ITF) -a group that provides a forum for early dialogue with applicants.
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3. Regulatory experience in PD
25 October 2017
Number of licenced products since 1997: 25
Dopamine agonists – 8 (pramipexole -6, rotigotine -2)
Entacapone/tolcapone – 5
levodopa / carbidopa / entacapone – 2
MAO inhibitors – 1
ioflupane (123l) – 1 (diagnostic agent)
Rivastigmine – 8 (symptomatic treatment of dementia in PD)
Number of PD products that received scientific advice in the last five years: 15 (mainly dopamine agonists, L-DOPA/carbidopa combinations, adenosin 2a agonists, mGluR5 antagonists, and a couple of diagnostic tools)
Only a couple PD products were seen in Innovation task force (ITF) platform
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4. Regulatory? Scientific? Both? How can we help tackle them?
Bottlenecks?
Regulatory? Scientific? Both? How can we help tackle them?
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5. EMA initiatives to support drug development
What do we provide?
Scientific guidelines
designed to help applicants prepare marketing-authorisation applications for human medicines. Guidelines provide a basis for practical harmonisation of how the EU Member States and the Agency interpret and apply the detailed requirements for the demonstration of quality, safety and efficacy.
PD guideline last updated in June WC pdf
What can stakeholders do?
Comments on the guideline can be sent during the consultation phase or at any other point in time.
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6. EMA initiatives to support drug development
25 October 2017
What do we provide?
2. Innovation Task Force (ITF) platform and meetings
meetings are free and provide an open environment for a dialogue with experts
a multidisciplinary group that includes scientific, regulatory and legal competences.
provide regulatory advice to applicants on the eligibility to Agency procedures as a medicinal product
increase awareness and learning in emerging therapies and technologies at the Agency
What can it be used for?
a discussion platform for early dialogue with applicants to proactively identify scientific, legal and regulatory issues of emerging therapies and technologies (e.g. growth factors, gene and cell therapy, any significant therapeutic innovation in PD;)
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7. EMA initiatives to support drug development
25 October 2017
What do we provide?
3. Qualification of novel methodologies procedure
CHMP Qualification Opinion on the acceptability of a specific use of the proposed method (e.g. use of a biomarker) in a research and development context (non-clinical or clinical studies), based on the assessment of submitted data.
CHMP Qualification Advice - for further method development towards qualification;
What can it be used for?
To qualify for the intended use (and make acceptable in a regulatory sense) different:
- Biomarkers Models Diagnostic tools Scales, PROs etc.
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8. EMA initiatives to support drug development
25 October 2017
What do we provide?
4. Scientific advice (SA) and protocol assistance (PA)
Scientific Advice can be given on ANY scientific question on quality, non-clinical and clinical development
at any time point of the development (post-marketing advice is also available)
Strictly confidential
What should it be used for?
to present and justify a new strategic scientific approach in drug development especially when:
no guideline is available OR when development plans to go against the guideline recommendations (for a scientific reason)
Treatments are developed for rare diseases and conditions as well as in the case of advanced therapies
In the clinical development - SA on primary endpoints, statistics and comparator should be sought – analysis shows that when these have been agreed upon in SA, the success rate for a positive opinion at marketing authorization stage increases
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9. Scientific advice and protocol assistance
25 October 2017
Scientific advice and protocol assistance
Incentives and benefits
The financial side:
For small- and medium-sized-enterprises (SMEs) fee waiver is available
PA for orphan drugs – reduced fee (no fee if applicant is a SME)
Paediatric only developments – no fee
The scientific and regulatory side:
Saves time and resource by providing solutions to difficult issues in the early stages (before too much time and resources have been committed)
Advices are peer reviewed by the CHMP (for advanced therapies CAT is involved) – same experts that will review the marketing authorisation application
Complying with SA/PA is significantly associated with positive outcome – around 97% of applications compliant with received SA have been granted a positive opinion at marketing authorization stage;
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10. Take home messages
25 October 2017
The European Medicines Agency offers a number of initiatives to stimulate and support new drug development, that can help tackle important scientific and regulatory issues
The initiation of early dialogue is essential and recommended.
The active and targeted scientific dialogue with the regulator increases the chances for positive results at marketing authorization stage
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11. Come and talk to us! Drug development
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12. Other useful links and more information:
Small and medium sized enterprises: l/general_content_ jsp&mid=WC0b01ac cc Orphan drugs: l/general_content_ jsp&mid=WC0b01ac ce EMA scientific guidelines: l/general_content_ jsp&mid=WC0b01ac cb Advanced therapies: l/general_content_ jsp&mid=WC0b01ac e0
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