L'intervento di Luca Pani (professore Ordinario di Psichiatria Clinica, Università di Miami) in occasione dell'evento "Il valore della ricerca biomedica" che si è tenuto a Pula (CA) il 3 aprile 2019.

1. Il processo di innovazione in atto: dalle strategie di brevettazione
al fundraising fino alle collaborazioni con le big pharma e le
organizzazioni di pazienti
Ricerca e Sviluppo Farmaceutico 4.0
Luca Pani, MD
Professor of Clinical Psychiatry, University of Miami, USA
Professor of Pharmacology, University of Modena and Reggio Emilia, Italy
VP for Regulatory Strategy and Market Access Innovation, VeraSci, USA
Former Director General Italian Medicines Agency (AIFA), Rome
Former CHMP and SAWP Member, European Medicines Agency (EMA), London
Lpani@miami.edu @Luca__Pani

2. The opinions expressed in this presentation are my personal views and may not be understood or
quoted as being made on behalf of or reflecting the position of any of the Institutions or Companies
for which I have worked or I collaborate with.
The mention of commercial products, their sources, or their use in connection with material reported
herein is not to be constructed as either an actual or implied endorsement of such products of any
Public Department or Health and/or Payer Services.
Apart from my Academic roles, I am the Chief Scientific Officer of EDRA-LSWR Publishing Company
and of Inpeco SA Total Lab Automation Company. In the last year I have been a scientific consultant to
Acadia USA, BCG, Switzerland, Dialectica UK-Greece, Ferrer Spain, Guidepoint UK, Johnson &
Johnson USA, VeraSci USA, Otsuka USA, Pfizer Global USA, PharmaMar Spain, Takeda USA.
I do not bear any direct or indirect financial interest in products quoted in this talk.
These slides are both original or have been modified from presentations/videos at other meetings.
Acknowledging: V. Mantua, MD, PhD
This presentation is updated to March 29th 2019.
Disclaimer and Disclosure

3. NEEDED
• Timing
• Risk Taking Abilities
• Ownership & Control
• Ecosystem Facilitation
• Revolutionary Technologies
• People of Outstanding Talent
NOT REALLY NEEDED
• Money
• Isolation
• Feasibility Projects
• Administrative burdens
• Bureaucratic complications
• ”Democratic” sharing of ideas
• “Negative” people around you
Components of Innovative Spin-offs

4. The long and winding road in the realm of precision medicine

5. • Make sure you understand the pathophysiology
• Find informative animal models
• Translate effects from health to disease
• Paradoxically diagnostic entities in precision
medicines such as gene therapies could be more
heterogeneous and have different underlying
biology than previously thought
• Real life data and drug usage could be different
(sometimes radically different) from those
collected in registration clinical trials
Pharma R&D Simple Rules and Further Issues in Predictability
• Safety
• Efficacy
• Quality
• Population
• Endpoints
• Comparator
• Duration of Response
• Outcome Value and Costs

6. Extract and enable precision medicine from human genetics
Laura Nisenbaum, National Academies of Sciences, Engineering, and Medicine workshop presentation, 3/8/17

7. Use the molecular diversity of genetic diseases in Pharma R&D
Michael Pacanowski, National Academies of Sciences, Engineering, and Medicine workshop presentation, 3/8/17
https://www.fda.gov/ScienceResearch/SpecialTopics/PrecisionMedicine/default.htm (accessed April 26, 2017).
e.g. ADA-Scid
e.g. Cystic Fibrosis
e.g. Fabry disease
No biomarkers
Complex Diseases

8. 15-24 years
55-64 years
Absolute size in millions for young and old age groups for EU 25, 1995-2030
Millions
40
45
50
55
60
65
70
1995 2000 2005 2010 2015 2020 2025 2030
Where to go? Well, the population in the EU is ageing rapidly
8
% of > 65 y.

9. Where to go? Fifty Possible Therapeutic Areas
Patients
Member State
policy makers
Regulators
Academia
Research based
pharmaceutical
Industry
EU Policy Makers
Physicians /
healthcare
professionals
SMEs

10. But using innovative drug discovery methods (after IMI-EFPIA).
1. Safety: Making medicines safer
2. Efficacy: Making Medicines more effective
3. The 5 areas with high scientific challenges and opportunities:
a) Cancer
b) Brain disorders
c) Infectious diseases
d) Immune and Autoimmune Disorders
e) Metabolic Syndrome and Inflammation
4. Education and Training: addressing expertise gaps in Europe
5. Knowledge Management: using new technologies to manage and
organize data

11. Pharmaceuticals objectives European regulators long for
Provide for more, more efficient and safe targeted therapies
Push the strategies towards diseases modifiers
Provide new tools for screening, monitoring progression or
relapse of disease, patients treatment selection and monitoring
response to drugs
Increase the rate of success of new development
Expedite the development of drugs

12. Talk early to the EMA European Innovation Network
Facilitate the development of innovative medicines by addressing gaps in early
regulatory support to innovation
─ Make the support more visible and attractive
to innovators;
─ Provide a platform for regulators to share and
improve the knowledge flow
─ Reinforce dialogue with innovators with a
wider EU exposure of identified issues;
─ Encourage sponsors of promising drug
development projects to move into the next
appropriate regulatory level
─ Actively contribute to and integrate into
relevant EU initiatives enabling innovative
medicines development and access to
patients.

13. If a new pharma product breaks out of the marginal value mould of traditional
pharmaceuticals with curative properties in indications with limited treatment
options1 we need to have long term data
Assuming that the effects are at least long term, if not curative, has 2 key
implications:
1. Show that long lasting curative effects are likely to reduce ongoing costs of
patient support and managing chronic comorbidities2.
2. Show even more that early cures or substantial benefits at a young age
could help produce significant gains in work productivity for patients
compared to treatments that bring marginal gains over many years.
Mod. from 1Bubela et al., 2016; 2Abou-El-Enein et al., 2016
Keeping in mind a target that, I am afraid, is also economical

14. Per quelli di voi che erano qui 9 anni fa…
…ho solo aggiornato le cose che avevo già detto !"