Transcript Criteria for Orphan designation

AIFA
European Conference on
Clinical Research for
Decision Making
Which Medicines Do We
Need ?
Thomas Lönngren, EMEA
30 March 2007
Content
• What are the health trends in EU ?
– Demographics
– Disease patterns
• Assessing the needs for medicines
– WHO report Priority Medicines for Europe and the world
• How will decide on the medicines we need ?
• EMEA role in the need of medicines
• Conclusion
Demographics
• We are getting older
• Number of over 80 year olds in EU 15 will
increase dramatically over next 15 years
• Difference between EU 15 and EU 10
• Ratio of pensioners to workers will have
economic and social consequences
• Higher cost of health care and medicines
is to be expected
Demographics
Disease patterns
• Burden of disease
– Mental 23%,CVD 17%,Cancer 16.7 ,Injuries
11%, Respiratory 7.8
• Mortality
– CVD 42%, Cancer 27%, Injuries 5.2, Mental
4.2%
• Difference between EU countries
– Example Tuberculosis
TB Incidence
Assessing the needs for medicines
• WHO report Priority Medicines for Europe
and the World
• Methodology
• Findings
WHO methodology
Burden of disease ranking
EU10, EU25
The world (including EU25)
Projections
and trends
Cochrane database of
systematic reviews
Clinical efficacy
PRELIMINARY LIST
OF PRIORITY DISEASES AND
GAPS
IN DEPTH REVIEWS OF PRELIMINARY LIST OF DISEASES AND GAPS
FINAL REPORT
Social solidarity
WHO methodology
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What is the size and nature of the disease burden?
What is the control strategy?
Why does the disease burden persist?
What wan be learnt from past/current research into pharmaceutical
interventions for this condition?
What is the current “pipeline” of products that are to be used for this
particular condition?
What are the opportunities for research into new pharmaceutical interventions?
What are the gaps between current research and potential research issues
which could make a difference, are affordable and could be carried out in
a) five years or b) in the longer term?
For which of these gaps are there opportunities for pharmaceutical research?
Findings on the pharmaceutical gaps
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Infections due to antibacterial resistance
Pandemic influenza
Cardiovascular disease (secondary prevention)
Diabetes (Type 1 and Type 2)
Cancer
Acute stroke
HIV/AIDS
Tuberculosis
Neglected diseases
Malaria
Alzheimer disease
Osteoarthritis
Chronic obstructive pulmonary disease
Alcohol use disorders: alcoholic liver diseases and alcohol dependency
Depression in the elderly and adolescents
Postpartum haemorrhage
Submission of MAA through the Centralised Process are likely to come
in phases for key indications
Obesity: some development targeting both lipid and CNS pathways
Alzheimer’s disease: extensive development including potential disease modifiers
Influenza: continued submission of Core Dossier, Pre-pandemic and Seasonal influenza vaccines
Insomnia: compounds targeting Serotinergic, GABA and
Melatonin pathways
Rheumatoid arthritis: multiple biological therapies using novel
mechanisms
Schizophrenia: compounds targeting combination of Serotinergic
and Dopaminergic pathways
Chronic Obstructive Pulmonary Disease/Asthma: multiple novel
mechanisms, many as combinations
Parkinson’s: possible generics, few novel mechanisms
Pain: multiple novel mechanisms and generic MAA
Oncology: multiple submissions, multiple indications. Increased number of biologicals including therapeutic antibodies, transfected cells,
fusion proteins and DNA vaccines
Hypertension: multiple submissions per year, third of which could be generic.
Large number of combinations
Type II Diabetes: novel classes including ‘Gliptins’ and Glucagon-Like Peptide-1 (GLP-1) receptor agonists.
Developed as monotherapies and combinations
2007
2008
2009
2010
Who decides on the medicines we need?
• Policy makers, EU institutions and
individual governments
• Research-based pharmaceutical industry
• Regulatory agencies for market approval
• Pricing and reimbursement authorities
• Health care systems and prescribers
• Patients
Policy makers, EU institutions and individual
governments
• They are setting the overall scene
• In EU pharmaceutical policy proposed by the European
Commission and decided by European Parliament and
Council, e.g. DG SANCO public health programme
• Do not cover healthcare and pricing and reimbursements
- national responsibility
• EU regulation is based on presumption that commercial
forces will supply the medicines we need
• Where the market does not deliver, then targeted action
is taken, e.g. orphan drugs, paediatrics and Article 58
• DG Research 7th framework programme gives directions
on priority areas of research
Research-based pharmaceutical industry
• Drivers for decision-taking
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Scientific opportunities
Market assessment and return on investment
Availability and required resources
Medical need
• Industry dilemma and challenge
– Failure/attrition rate
– High price for new medicines
– Reward for innovation and price pressure
• Will the industry deliver the medicines we need?
Challenges
Regulatory agencies for market approval
• Decision on market approval based on
quality, safety and efficacy
• Relative efficacy (comparative clinical
trials)
• 20-25% of applications will not get market
approval
• Are the approved medicines the medicines
we need?
Pricing and reimbursement authorities
• Different systems and criteria in EU
Member States
• No EU harmonisation/competence
• Decision based on ? and relative
effectiveness and cost effectiveness
• Will their decisions secure access to
medicines we need
• (Un)Equal access in EU
p7-8 – Situation
Average time delay between registration and effective market access
in number of days
Poland
Greece
Slovakia
Belgium
France
Czech Rep.
Italy
Portugal
Norway
Spain
Hungary
Netherlands
Finland
Switzerland
Ireland
Cyprus
Sweden
Estonia
Austria
Germany
UK
USA
521
495
488
478
380
357
317
315
249
240
227
220
175
124
105
92
90
In line with
EU Directive
52
0
0
0
Note In Poland no new innovative products have been reported as reimbursed for the past six years
Source: IMS, 2004
Health care systems and prescribers
• Health technology assessments
– Example NICE in UK
– Recommendation of use within NHS
• Regional and local drug committees
– Recommendation on list of medicines for use
– Local responsibilities for budget
– Forcing hard priorities
• What is the criteria's for decision making?
• Prescribers have the final decision on witch
medicines we need
Patients
• Historical patients passive in decision on therapy
• Probably a dramatic change in the future
• Budget constrains and priorities will make
patients and there organisation more active
• Publication of clinical trials will create a demand
for early access to new medicines
• Availability of information (Internet)
• Patients participate in benefit risk assessment
• Patients will move where they could receive the
treatment
EMEA role in the need of medicines
Research-based industry, not regulators (incl. EMEA)
select therapeutic areas of interest and make
individual drug development decisions
However, regulators (and legislators) proactively
facilitate and influence drug development by:
• Setting regulatory and scientific standards, making
regulatory decision more predictable
• Introduce incentives (e.g. orphan drugs, paediatric
drugs, SMEs)
• Specific collaborative programs: EMEA Road map
2010 ,US FDA critical path initiative, EU commission
and industries initiative IMI
EMEA roadmap 2010
– Safety of medicinal products
– Earlier availability of new medicinal products
– Support to innovation
– Transparency, communication and provision of
information
– Reinforcing the EU medicines network
Rapid access to new medicinal products
and Support to innovation
• New regulatory tools for access
– Conditional MA and accelerate assessment
– Risk management plans
– Benefit risk assessment more consistency and
predictability
• Support to innovation
– How to met the demand from a rapid scientific
development
– Scientific advice in drug development
• Urgent public health needs
– Pandemic influenza vaccines
EMEA support to IMI Initiatives
• The Innovative Medicine Initiative (IMI)
– Part of the 7th Research framework program
– Promote development innovative therapies
– Partners: EU Commission, Academia, Patients
Associations, EU Industry (including SMEs), Regulatory
Authorities
– Objectives: to make development process faster, efficient
(reduce attrition), predictable and cheaper
IMI Initiative
IMI Initiative
Conclusion
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A shift to a growing elderly population will create a high need for health care in EU
and need for new treatments
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Important to monitor the development of new medicines and identify gaps and
needs
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If not market forces will fill the gaps governments or none profit organisations
need to intervene
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Decision making and criteria's for regulatory approval is well defined with a long
tradition and history
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Post approval decisions on different levels in member state is not harmonised
leading to difference in availability
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A priority for EMEA is to support accesses to medicines and stimulate drug
development
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Innovative medicine initiative (IMI )will contribute to an improved drug
development process