AIFAAIFAEuropean Conference on European Conference on

Clinical Research for Clinical Research for Decision Making Decision Making

Which Medicines Do We Which Medicines Do We Need ?Need ?

Thomas Lönngren, EMEA Thomas Lönngren, EMEA 30 March 200730 March 2007

ContentContent

• 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

DemographicsDemographics

• 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

DemographicsDemographics

Disease patternsDisease 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 IncidenceTB Incidence

Assessing the needs for medicines Assessing the needs for medicines

• WHO report Priority Medicines for Europe and the World

• Methodology

• Findings

WHO methodologyWHO methodology

Burden of disease rankingEU10, EU25

The world (including EU25)

Cochrane database of systematic reviews

Clinical efficacy

Projectionsand trends

Social solidarity

FINAL REPORT

PRELIMINARY LISTOF PRIORITY DISEASES AND

GAPS

IN DEPTH REVIEWS OF PRELIMINARY LIST OF DISEASES AND GAPS

WHO methodologyWHO methodology

• 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 gapsFindings on the pharmaceutical gaps1. Infections due to antibacterial resistance2. Pandemic influenza3. Cardiovascular disease (secondary prevention)4. Diabetes (Type 1 and Type 2)5. Cancer6. Acute stroke7. HIV/AIDS8. Tuberculosis9. Neglected diseases10. Malaria11. Alzheimer disease12. Osteoarthritis13. Chronic obstructive pulmonary disease14. Alcohol use disorders: alcoholic liver diseases and alcohol dependency15. Depression in the elderly and adolescents16. Postpartum haemorrhage

Submission of MAA through the Centralised Process are likely to come in phases for key indications

Type II Diabetes: novel classes including ‘Gliptins’ and Glucagon-Like Peptide-1 (GLP-1) receptor agonists. Developed as monotherapies and combinations

2010200920082007

Hypertension: multiple submissions per year, third of which could be generic.Large number of combinations

Chronic Obstructive Pulmonary Disease/Asthma: multiple novel mechanisms, many as combinations

Parkinson’s: possible generics, few novel mechanisms

Schizophrenia: compounds targeting combination of Serotinergic and Dopaminergic pathways

Insomnia: compounds targeting Serotinergic, GABA and Melatonin pathways

Pain: multiple novel mechanisms and generic MAA

Rheumatoid arthritis: multiple biological therapies using novel mechanisms

Alzheimer’s disease: extensive development including potential disease modifiers

Obesity: some development targeting both lipid and CNS pathways

Influenza: continued submission of Core Dossier, Pre-pandemic and Seasonal influenza vaccines

Oncology: multiple submissions, multiple indications. Increased number of biologicals including therapeutic antibodies, transfected cells, fusion proteins and DNA vaccines

Who decides on the medicines we need?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 Policy makers, EU institutions and individual governmentsgovernments

• 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 industryResearch-based pharmaceutical industry

• Drivers for decision-taking– 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?

ChallengesChallenges

Regulatory agencies for market approvalRegulatory 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 authoritiesPricing 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

In line with EU Directive

in number of days

Average time delay between registration and effective market access

p7-8 – Situation

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240

220

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105124

175

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380478

488495

PolandGreeceSlovakiaBelgiumFranceCzech Rep.ItalyPortugalNorwaySpainHungaryNetherlandsFinlandSwitzerlandIrelandCyprusSwedenEstoniaAustriaGermanyUKUSA

Note In Poland no new innovative products have been reported as reimbursed for the past six yearsSource: IMS, 2004

Health care systems and prescribersHealth 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

PatientsPatients

• 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 medicinesEMEA 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 2010EMEA 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 Rapid access to new medicinal products and Support to innovationand 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 InitiativesEMEA 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 InitiativeIMI Initiative

IMI InitiativeIMI Initiative

ConclusionConclusion• A shift to a growing elderly population will create a high need for health care in EU and

need for new treatments

• Important to monitor the development of new medicines and identify gaps and needs

• If not market forces will fill the gaps governments or none profit organisations need to intervene

• Decision making and criteria's for regulatory approval is well defined with a long tradition and history

• Post approval decisions on different levels in member state is not harmonised leading to difference in availability

• A priority for EMEA is to support accesses to medicines and stimulate drug development

• Innovative medicine initiative (IMI )will contribute to an improved drug development process