Supporting Innovation through Scientific Advice · 2019. 11. 18. · • EMA parallel HTA, FDA...
Transcript of Supporting Innovation through Scientific Advice · 2019. 11. 18. · • EMA parallel HTA, FDA...
Supporting Innovation through Scientific Advice
PEARRL Regulatory Science Symposium,
21st June 2017, University College Cork
HPRA
Dr Peter Kiely
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Disclaimer
6/21/2017
• All views expressed in this presentation are my own and do not necessary represent the opinions of HPRA or any entity which I have been, currently am or will be affiliated to.
• I declare I have no conflicts of interest.
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Overview
• Challenges for Drug Development/Access
• Scientific Advice (SA) - what is it & when
should it be sought
• EMA SA -figures and incentives.
• National SA
• Conclusion
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
Challenges in Access to Novel Medicines in
2017 & Beyond (1/2)
• Ageing population
• Increasing burden of chronic illnesses
• Increasing complexity novel medicines/supply chain
• Significant developments in medicine and manufacturing
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Challenges in Access to Novel Medicines in
2017 & Beyond (2/2)
• High cost of development of new technologies
• High and rising pharmaceutical expenses
• Effects of the economic crisis on healthcare spending
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
Regulatory Framework (1/2)
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Incentives to stimulate
development
Involvement of patients,
healthcare professionals, stakeholders
Committees and working parties
Promotes harmonisation at international level
European Medicines Agency (EMA)
Provides Scientific Advice
Development of best practice for
medicines evaluation and supervision in
Europe
Preparation of scientific
guidelines
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Regulatory Framework (2/2)Health Products Regulatory Authority (HPRA)
• National interface with the EMA / participate in EMA procedures
• Representatives on Committees and Working Parties – CHMP, COMP, CAT, PDCO, PRAC, HPMC
• National Innovation Office
• National Scientific Advice
• Clinical Trials
• HPRA Strategy
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
Scientific Advice (what is it?)
• Aim: to assist applicants in the development of new or existing
medicinal products.
• Taking into account the current knowledge of a given condition,
targeted patient population, existing treatment modalities and
specificities of the product being developed.
• Directive 2001/83: In assembling the dossier: applicants shall also
take into account the scientific guidelines relating to the quality,
safety and efficacy.
• Guideline Deviations, Clarification or Absence
• Guidelines of limited scope: Provision of tailored product specific
advice
• Novel drug development
• Unforeseen problems
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
Types of scientific advice
• Product-Specific Scientific Advice - should be about
one medicinal product (or drug-device combination) only
(majority of meetings are in this category)
• Broad Scope Scientific Advice - not product-specific,
e.g. use of a new study design that is applicable in a
disease area, a new manufacturing process to be used
across a range of products, general discussion of clinical
development in a particular area
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
Types of Scientific Advice (2/2)
• Joint Regulator and Payer Scientific Advice
• Regulator: enables product access to market.• Payer: enables patient access to product.
• National Centre for Pharmacoeconomics (NCPE in IE)• National Institute for Health and Care Excellence (NICE
in UK)• German Institute of Medical Documentation and
Information (DIMDI)
• EMA parallel HTA, FDA meetings• EMA tailored scientific advice pilot project in February
2017 to support the development of new biosimilar medicines
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
Qualifications of Novel Methodologies
• CHMP Qualification Opinions: The method can apply to non-
clinical or to clinical studies, such as the use of a novel biomarker.
open for public consultation by the scientific community before
adoption.
• CHMP Qualification Advice: advice on protocols and methods that
are intended to develop a novel method with the aim of moving
towards qualification.
• Letter of Intent: drug biomarker qualification or clinical outcome
assessment to EMA and FDA.
• Letters of Support: when the novel methodology under evaluation
cannot yet be qualified but is shown to be promising based on
preliminary data - aims to encourage data-sharing and to facilitate
eventual qualification
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
EMA SCIENTIFIC ADVICE AND PROTOCOL ASSISTANCE
REQUESTS RECEIVED – TOTAL
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
EMA SCIENTIFIC ADVICE REQUESTS BY THERAPEUTIC AREA (2015)
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Scientific Advice: When and Who?
Interactions with Regulators
• Innovation Offices (EMA and NCA): novel medicine, device or manufacture
• Scientific Advice (EMA and NCA); any stage of development - can be broad scope ; not legally binding
• Clinical Trial Authorisation Applications (NCA): investigational medicinal products
• Marketing Authorisation Applications (EMA and NCA): centralised (biologicals, vaccines, certain chemicals, orphan), decentralised, national (chemicals)
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
SCIENTIFIC ADVICE REQUESTS BY AFFILIATION OF REQUESTER
presented at the PEARRL Regulatory symposium 2017 – for personal use only
CLINICAL-TRIAL PHASES OF SCIENTIFIC ADVICE REQUESTS (2011-2015) EMA
presented at the PEARRL Regulatory symposium 2017 – for personal use only
EMA Incentives
• Total or partial fee reduction for Orphan medicinal products.
• Paediatric development
• Small and Medium enterprises
• Prime scheme
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
HPRA Scientific Advice
• The HPRA provides national scientific and
regulatory advice to commercial and non-
commercial entities. Further details are
provided on the HPRA website:
www.hpra.ie
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
National scientific advice
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• Respiratory medicine
• Rheumatology
• Obstetrics / Gynaecology
• Dermatology
• Radiopharmaceuticals
• Indications for Botulinum neurotoxins
• Endocrine and Gastrointestinal conditions
• Generic medicines and biosimilars areas
• Meeting at HPRA offices
• 30 days after the meeting,
written advice is issued
• HPRA also actively contributes to
Scientific Advice at EU level
• Provides advice on Biopharma
products
presented at the PEARRL Regulatory symposium 2017 – for personal use only
Conclusions
• Development of new medicines that address unmet medical needs and to meet challenges are essential
• Increase interaction at earlier development.
• Improve efficiencies– ensuring data generated by sponsors is relevant for the needs of regulatory authorities/HTA’s .
6/21/2017 presented at the PEARRL Regulatory symposium 2017 – for personal use only
LinksEMA scientific advicehttp://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000049.jsp
HPRA National scientific and regulatory advice.
http://www.hpra.ie/homepage/medicines/regulatory-information/national-scientific-and-regulatory-advice
presented at the PEARRL Regulatory symposium 2017 – for personal use only