Mihaela Ianosev – European Medical Director CDBU
Industry Expectations:
Discovery, Research, Development ,
Access to medicines
Pharmaceutical R&D Ecosystem – radical changes
■ Change in demographics
■ R&D productivity
■ Rapid escalation of the costs of R&D
■ Higher risks of failure associated with greater innovation
■ Global tightening and conservatism of regulatory policy
■ Diminishing advantages of being first to market
■ Changes in social expectations
■ Patent expiration
■ Science & Technology
■ Economical crisis
■ HTA, pricing & reimbursement
1. Priorities for Research
2. Value based development and access
to medicines
3. Modalities of Discovery
Un
sa
tisfie
dN
ee
d
Priorities of Research
important
moderate
modest
Added Benefit
Level of Innovation
Diseases with mostly
satisfied needsUn
sa
tisfie
dN
ee
d
Added Benefit
Priorities of Research
Orphan Diseases
Diseases with residual needs
Diseases with off
patent drugs
Priorities of Research
Rate of Orphan Product Approvals Remain Flat
All Orphan Designations
and Approvals (USA)
0
20
40
60
80
100
120
140
y 2001
y 2002
y 2003
y 2004
y 2005
y 2006
y 2007
y 2008
y 2009
y 2010
All Orphan Designations
and Approvals (EU)
0
50
100
150
200
250
y 2000y 2001y 2002 y 2003y 2004y 2005y 2006y 2007y 2008y 2009y 2010
Orphan Designations
Approvals
Orphan Designations
Approvals
Rare Diseases Drug Development
a Challenging Reality
Substantial heterogeneity of patient population
Difficulty in clearly defining the patient population – clinical
presentation, disease subtype
Small patient populations
Difficulty in demonstrating statistical significance
Geographically dispersed patients – recruitment
Limited clinical experiences
Common problems for medical sites, industry and agency
Challenge of defining practical clinical endpoints
Traditional study designs often not feasible
Randomization of trials and inclusion of control arms can be untenable
Double-blind design with placebo or standard of care is often difficult
to apply
Regulatory expectations established primarily for more common
diseases => big, long clinical studies
8
Access to Rare Diseases Treatments
Opportunities for Policy Optimisation
10 solutions to accelerate access to treatments in rare diseases
Nature Reviews
Drug Discovery
1. Importance of continued flexible orphan incentives
2. Role of Patients’ disease registries & post-approval studies
3. Global Simplification-Harmonization of regulatory requirements
9
Patient Timely Access to Rare Diseases Treatments
Development Process as a Continuum
1. Accelerated and Conditional
approval should become the
default pathway in this priority
population provided sufficient
dialogue has taken place
between patients, physicians,
drug developers and HTA.
2. Progressive assessment
and approval mechanism
as a standard practice
April 2012
Compelling arguments:
- small patient population
-Concentration of clinical
research activities in CoE
- High level of specialization of
the treating physician
- Wealth of scientific knowledge
among patients & their families
= high quality interaction with
the clinician
10
Value & Specificity of the Rare Diseases
Model the Policy Equation
Industrial policy
Health policy
Unmet need
Patient care & access
Equity & solidarity
R&D innovation
Economic ROI
Industry competitiveness
Health Budgets
11
4. Moderate
improvement
1. Small
improvement
7. Major
improvement
Rare Disease Unmet NeedMedicine Therapeutic
Benefit
Medicine Therapeutic
Value
Treatment
available
Treatment
Social
outcome
Disease
Clinical
severity
Treatment
Clinical
Benefit
and outcome
Disease
Social Impact
Value attributes
Value criteria definition
Treatment
innovation
Define Medicine Therapeutic Value
0
20
40
60
80
100
120
Germany Taiwan Malaysia Brazil China
Monthly Price Index
Illustrative case studyGlobal patient reach
Patient Access & Global Reach
Tiered pricing based on payers’ willingness/ability to pay
Germany
Taiwan
Malaysia
BrazilChina
Tier 4 Tier 5Tier 3Tier 2Tier 1
1
2High income
3Upper Middle Income
4Middle Income
5
Low Middle Income
USA +EU + Japan
Modalities of Discovery
Coopetion & Collaboration
1. Among Companies
Pre-competitive research
2. Knowledge Chain
Universities, Charities, Foundations, No profit
Start Up, Micropharma
Coopetion & Collaboration
TransCelerate BioPharma Inc.
Scope: identify and solve common drug development challenges with
the end goals of improving the quality of clinical studies and
bringing new medicines to patients faster
Initial area of focus = clinical study execution
1. development of a shared user interface for investigator site
portals
2. Mutual recognition of study site qualification and training
3. Development of risk-based site monitoring approach and
standards
4. Development of clinical data standards
5. Establishment of a comparator drug supply model
As shared solutions will be developed – will involve industry alliances
(ex. Clinical Data Interchange Consortium, Critical Path Institute,
Clinical trials Transformation Initiative), regulatory bodies (FDA,
EMA) and CROs.
Coopetion & Collaboration
1. Among Companies
Pre-competitive research
2. Knowledge Chain
Academia, Charities, Foundations
Start Up, Micropharma
Modalities of Discovery – The “How”
Preliminary Experience
20
GSK Rare Diseases
Small molecule pharmacological
chaperones
Oligonucleotides
Gene therapy
Enzyme replacement therapy
Rare Diseases Investment Dynamics
Alliances & Partnerships
Our mission
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