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)

6/21/2017

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

6/21/2017

• 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