"Pharmaceutical sciences and the challenge for a healthcare focused agenda for pharmacists training" (A interface ensino-profissão no desenvolvimento das Ciências Farmacêuticas) Rogério Gaspar (Presidente da SPCF) 21.SET.2016

1. Rogério Sá Gaspar
Presidente da Sociedade Portuguesa de Ciências Farmacêuticas (SPCF / membro da EUFEPS)
Professor do Departamento de Socio-Farmácia da FFUL
Vice-Reitor da Universidade de Lisboa
rgaspar@ff.ulisboa.pt
Pharmaceutical sciences
and the challenge for a healthcare focused agenda for pharmacists training
(A interface ensino-profissão no desenvolvimento das Ciências Farmacêuticas)
21.SET.2016

2. Recent references
(last 5 years)
2
Reference positions (last 5 years)
• University Governance
• Vice-Rector R&D, University of Lisboa (ULisboa), Portugal (2013-2016 )
• General Council of University of Lisbon (UL, Portugal), 2008-2013
• Vice-Dean, Research & Tech Transfer/ International Affairs (FFUL) 2012-2013
• Head of Department Pharmaceutical Technology, FFUL, 2007-2013
• Research groups Leadership
• Group Leader “Nanomedicine & Drug Delivery Systems” (iMed.UL), 2007-2013
• Group Leader “Intracellular Trafficking Modulation for Advanced Drug Delivery” (iMed.UL), 2013-2014
• Scientific Societies / International bodies
• SPCF, President Portuguese Society for Pharmaceutical Sciences (since 2016) / Vice-President (2011- 2016)
• Foreign member of the Spanish Royal Academy of Pharmacy (RANF, 2016)
• EUFEPS, Executive Committee (2009-2013), VP for Science Policy in 2011/2012, EUFEPS Council representing SPCF (2016)
• EUFEPS, Chair of Regulatory Science Network (2011-2014)
• FIP, Vice-chair Regulatory Science Special Interest Group (2011-2014)
• Advisory positions (international)
• External Advisory Board of EuroNanoMedNet (ERA-NET in Nanomedicine, FP7, EU) (active)
• External Advisory Board of European Doctoral Program in Nanomedicine (NANOFAR) (active)
• Scientific Advisory Board of TRANS-INT (Large FP-7 project) (active)
• Scientific Advisory Board of Z-Cube (Zambon group, Milano, Italy, 2011-2013)
• Editorial Board/International Advisory Board:
Nanomedicine: Nanotechnology, Biology, and Medicine (IF2015= 6.155, Elsevier, 2010-2015), WIRES – Nanomedicine and Nanotechnology (IF2015= 4.494, Wiley, 2009-
today), International Journal of Nanomedicine (IF2015= 4.383, Dove Press, 2010-today), Drug Delivery and Translational Research (Controlled Release Society, Springer, 2011-
today) (…)

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Medicines development
and market determinants
• Societal pressure for healthcare resourcesfacing the impact
of demographics and aging populations
• Drug development process and increased costs

4. Horizon 2020
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5. 5
Health expenditure
& pressure of increased life expectancy
Public Spending on health and long-term care: a new set of projections
(OECD Economic Policy Papers, 06, June 2013)
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6. 6
Human development, aging
& pattern changes in geographicaldistribution
Source: BBSR, Bonn, 2015
Source: EuropeanParliament Research Service, 2013
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Aging European population
Source: EuropeanParliament Research Service, 2013
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8. 8
Trends in new medicinal products
“2015 FDA drug approvals”
Asher Mullard, NRDD, February 2016
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Trends in New Approvals
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New medicinal products (FDA)
Asher Mullard, NRDD, February 2016, “2015 FDA drug approvals”
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Drug Development: increased costs…
How to improve R&D productivity: the pharmaceutical industry’s grand challenge
Steven M. Paul, Daniel S. Mytelka, Christopher T. Dunwiddie, Charles C. Persinger,, Bernard H. Munos, Stacy R. Lindborgand Aaron L. Schacht
Nature Reviews Drug Discovery, 9 (3): 203-214 (2010)
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Primary cause of failure categories
for terminated compounds*
“An analysis of the attrition of drug candidates from four major pharmaceutical companies” - Michael J. Waring, John Arrowsmith, Andrew R. Leach, Paul D. Leeson, Sam Mandrell, Robert M. Owen,
Garry Pairaudeau, William D. Pennie, Stephen D. Pickett, Jibo Wang, Owen Wallace and Alex Weir, Nature Reviews Drug Discovery, 14: 475-486 (July 2015)
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“Pharmaceutical sciences in 2020”
“Pharmaceutical sciences in 2020”
Daan Crommelin, Pieter Stolk, Luc Besançon, Vinod Shah,
Kamal Midha and Hubert Leufkens,
Nature Reviews Drug Discovery,
volume 9 | February 2010 | p.99-100
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14. EUFEPS position paper on Horizon 2020
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European Journal of Pharmaceutical Sciences 47 (2012): 979-987, doi 10.1016/j.ejps.2012.09.020

15. EUFEPS (2012 position paper)
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Systems Approaches: the 5 S
Prediction
Kinetics
Modelling
Systems
Toxicology
Systems
Pharmaco-
logy
Systems
Therapeutics
Systems
Techno-
logies
Complex
Delivery
Systems

16. Innovative agendas (2017)
http://pswc2017.fip.org/files/static/Untitled.png

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Quantitative and Systems Pharmacology
Quantitative and Systems Pharmacology in the Post-genomic Era:
New Approaches to Discovering Drugs and Understanding Therapeutic Mechanisms
An NIH White Paper by the QSP Workshop Group – October, 2011
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18. The global agenda for “medicines research”:
Holistic translational research
Quantitative and Systems Pharmacology in the Post-genomic Era:
New Approaches to Discovering Drugs and Understanding Therapeutic Mechanisms
An NIH White Paper by the QSP Workshop Group – October, 2011
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19. Medicinal Product versus Medical Device
• Medicinal Product
(art.1(2) of DIRECTIVE 2001/83/EC OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL, of 6 November 2001, as amended):
(a) Any substance or combination of substancespresented as having propertiesfor treating or preventing disease in
human beings; or
(b) Any substance or combination of substanceswhich may be used in or administered to human beingseither with a
view to restoring, correcting or modifying physiologicalfunctions by exerting a pharmacological, immunologicalor
metabolic action, or to making a medicaldiagnosis
• Medical Device:
(art.1(2) of COUNCIL DIRECTIVE 93/42/EEC, of 14 June 1993, concerning medical devices (as amended)
any instrument, apparatus, appliance,software, materialorother article, whether used alone or in combination,
including the software intended by its manufacturerto be used specifically for diagnostic and/ortherapeutic purposes
and necessary for its proper application, intended by the manufacturertobe used for human beingsfor the purpose of:
—diagnosis, prevention, monitoring, treatment or alleviation of disease
—diagnosis, monitoring, treatment, alleviation of or compensation for an injury or handicap,
—investigation, replacement or modification of the anatomy or of a physiological process,
—control of conception,
and which does not achieve its principalintended action in or on the human body by pharmacological, immunologicalor
metabolic means, but which may be assisted in its function by such means;
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20. Steps for
Class IIb medical devices compliance
• Classification: ensure the device is a Class IIb medical device.
• Choose Conformity Assessment Route: refer the flow chart below.
• Compile the Technical File.
• Obtain certification from a Notified Body
• Declaration of Conformity.
• Appoint an Authorised Representative.
• (Hold the Tech Files for inspection by the Competent Authority)
• Vigilance and Post Market Surveillance.
• (affix CE marking & market the products)
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22. Class IIb Medical Devices:
Conformity Assessment Routes
• In the case of devicesfalling within Class IIb, other than deviceswhich are custom-made orintended for clinical
investigations, the manufacturershall, in order to affix the CE marking, either: follow the procedure relating to the EC
declaration of conformity set out in Annex II (full quality assurance); in this case, point 4 of Annex II is not applicable;
or
• follow the procedure relating to the EC type-examination set out in Annex III, coupled with:
• (i) the procedure relating to the EC verification set out in Annex IV;
or
• (ii) the procedure relating to the EC declaration of conformity set out in Annex V (production quality assurance);
or
• (iii) the procedure relating to the EC declaration of conformity set out in Annex VI (product quality assurance).
• There are tworoutes: a Notified Body must carry out either an Annex II audit of the full quality assurance system (ISO
13485:2003),
or
• a type-examination (AnnexIII) plus one of the three options given here:
• Examination and testing of each product or homogenous batch of products (Annex IV); or
• Audit of the production quality assurance system (Annex V:) ISO 13485:2003 (excluding Design) or
• Audit of final inspection and testing (Annex VI:) ISO 13485:2003 (excluding Design & Manufacture)
• Once the manufacturerhasreceived certification from the Notified Body he may CE mark his productsand place them on
the market.
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Public and private sector contributions
to medicines R&D (last 25 years)
Source: “Public and Private Sector Contributions to the Research & Development of the Most Transformational Drugs of the Last 25 Years”,
A Tufts Center for the Study of Drug Development White Paper, Ranjana Chakravarthy, Kristina Cotter, Joseph DiMasi, Christopher-Paul Milne, Nils Wendel
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Biomarkers qualification (PSTC)
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Towards research-driven PPPs…
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Innovative Medicines Initiative
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Innovative Medicines Initiative (SRA)
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Innovative Medicines Initiative (IMI2)
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IMI2, Strategic Research Agenda (SRA)
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NCATS
National Center for Advancing Translational Sciences (NIH)
• Bridging Interventional Development Gaps, which makes available resources needed for new
therapeutic agents
• Clinical and Translational Science Awards, which fund a national consortium of medical
research institutions working together to improve the way clinical and translational research
is conducted
• Cures Acceleration Network, which enables NCATS to fund research in new and innovative
ways
• FDA-NIH Regulatory Science, which is an interagency partnership that aims to accelerate the
development and use of better tools, standards and approaches for developing and
evaluating diagnostic and therapeutic products
• Office of Rare Diseases Research, which coordinates and supports rare diseases research
• Components of the Molecular Libraries, which is an initiative that provides researchers with
access to the large-scale screening capacity necessary to identify compounds that can be
used as chemical probes to validate new therapeutic targets
• Therapeutics for Rare and Neglected Diseases, which is a program to encourage and speed
the development of new drugs for rare and neglected diseases.
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31. Diagnostics and Medical Devices
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32. Diagnostics and Medical Devices
Organs-on-a-chip models for cancer research
Organs-on-chips at the frontiers of drug discovery - Eric W. Esch, Anthony Bahinski (Univ. of Pennsylvania) and Dongeun Huh (Harvard),
Nature Reviews in Drug Discovery, April 2015
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33. Organs-on-chips at the frontiers of drug discovery - Eric W. Esch, Anthony Bahinski (Univ. of Pennsylvania) and Dongeun Huh (Harvard),
Nature Reviews in Drug Discovery, April 2015
Body-on-a-chipsystems
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Diagnostics and Medical Devices

34. 34
Where does nanotechnology belong in the map of science?
Alan L. Porter and Jan Youtie, Nature Nanotechnology, (September 2009): 534-536
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35. 35
Outreach Dissemination
GROWTHINNOVATION
CAPABILITIES
JOBS
Business
creation
activities
Higher
education
activities
Innovation-
driven research
activities
Research
actors
Business
actors
Higher
education
actors
Entrepreneurship
Entrepreneurial
talent
Start-ups,
Spin-offs
New products, services
and business models
35
The EIT innovation model
European Institute of Innovation & Technology
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36. Windows into next generation technologies:
from medicines/health technologies research
to innovative platforms and integrative healthcare
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37. Need to look “out of the box”:
the caverne syndrome

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https://www.wilsoncenter.org/sites/default/files/personalized_medicine.jpg
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The Path to Personalized Medicine
Margaret A. Hamburg, M.D., and Francis S. Collins, M.D., Ph.D.,NEJM, June 15, 2010.
The success of personalized medicine dependson having accurate diagnostic
tests that identify patients who can benefit from targeted therapies.
Increasingly, however,the use of therapeutic innovationsfor a specific patient is
contingent on or guided by the results from a diagnostic test that has not been
independently reviewed for accuracy and reliability by the FDA.
The agency's goal is an efficientreview processthat producesdiagnostic–
therapeutic approaches that clinicians can rely on and allows companiesthat
invest in establishing the validity and usefulness of tests to make specific, FDA-
backed claims about benefits.
New EnglandJournalof Medicine. The Path to Personalized Medicine.
http://content.nejm.org/cgi/content/full/NEJMp1006304
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Oncology: SNP analysis (NCI-60 & GWAS)
Pathway analysis of top genes from SNP comparative analysis. (a) Network created using genes common to the NCI-60 and GWAS: gemcitabine +placebo data sets; (b) Network using the significant genes in the NCI-60 data
set; (c) Network using the significant genes in the GWAS: gemcitabine +placebo data set. The significant genes have been indicated using the blue highlight. GWAS, genome-wide association studies; SNP, single-nucleotide
polymorphism. In Pharmacogenomic characterization of gemcitabine response – a framework for data integration to enable personalized medicine, by Harris et al. 2014
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Big Data & Clinical Research
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Personalized medicine: stakeholders
Personalized Medicine in Canada: A Visual Summary, Allie Janson Hazell (Dec 22nd
, 2011)
http://www.thegenoscape.com/2011/12/22/personalized-medicine-in-canada-a-visual-summary/OF_20160921

44. Pharmacists training goals:
science-based practice
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http://pswc2017.fip.org/files/static/Untitled.png
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45. Pharmacists training goals
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46. Pharmacists training goals
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47. Pharmacists training goals
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Pharmacists at The Johns Hopkins Hospital are working to improve HCAHPS scores related to educating patients

48. Pharmacists training goals
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49. Pharmacists training goals
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50. Pharmacists training goals
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“We are in the business of caring for people…” Dean Bob Blouin (Eshelman School of Pharmacy, UNC, Chappel Hill)