Supporting Innovation through Scientific Advice

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1 Supporting Innovation through Scientific Advice PEARRL Regulatory Science Symposium, 21 st June 2017, University College Cork HPRA Dr Peter Kiely

2 Disclaimer 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.

3 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

4 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

5 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 Regulatory Framework (1/2) Provides Scientific Advice Promotes harmonisation at international level Incentives to stimulate development Development of best practice for medicines evaluation and supervision in Europe European Medicines Agency (EMA) Involvement of patients, healthcare professionals, stakeholders Preparation of scientific guidelines Committees and working parties

7 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

8 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

9 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

10 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

11 Qualifications of Novel Methodologies CHMP Qualification Opinions: The method can apply to nonclinical 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

12 EMA SCIENTIFIC ADVICE AND PROTOCOL ASSISTANCE REQUESTS RECEIVED TOTAL

13 EMA SCIENTIFIC ADVICE REQUESTS BY THERAPEUTIC AREA (2015)

14 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)

15 SCIENTIFIC ADVICE REQUESTS BY AFFILIATION OF REQUESTER

16 CLINICAL-TRIAL PHASES OF SCIENTIFIC ADVICE REQUESTS ( ) EMA

17 EMA Incentives Total or partial fee reduction for Orphan medicinal products. Paediatric development Small and Medium enterprises Prime scheme

18 HPRA Scientific Advice The HPRA provides national scientific and regulatory advice to commercial and noncommercial entities. Further details are provided on the HPRA website:

19 National scientific advice Meeting at HPRA offices Respiratory medicine Rheumatology Obstetrics / Gynaecology Dermatology Radiopharmaceuticals Indications for Botulinum neurotoxins Endocrine and Gastrointestinal conditions Generic medicines and biosimilars areas 30 days after the meeting, written advice is issued HPRA also actively contributes to Scientific Advice at EU level Provides advice on Biopharma products

20 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.

21 Links EMA scientific advice =pages/regulation/general/general_content_ jsp HPRA National scientific and regulatory advice. latory-information/national-scientific-andregulatory-advice

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