An agency of the European Union

Adaptive Pathways to accelerate Patient access/medicines development

The European Medicines Agency approach

Presented by: Prof Guido Rasi,

EMA Executive Director

Key factors in R & D

• Medical need and available option

• Business model and clinical trial design

• Complexity of MA process

• Predictability of assessment outcomes

• Trust & Risk perception

• Economic & Social Environment (NHSs,insurances, solidarity, etc.)

• Patent protection status

Adaptive Licencing / Adaptive pathways

…..”The adaptive licencing process is based on a

prospectively-planned process. It starts with the early

authorisation of a medicine in a restricted patient population,

followed by iterative phases of evidence-gathering and the

adaptation of the marketing authorisation to allow broader

patient populations to access medicine…”

Presentation title (to edit, click View > Header and Footer) 2

“Adaptive licencing concept”

Final target indication in

blue, patient group with

highest need in red

the sponsor

could follow

two strategies

1st approval 2nd approval

1st approval

Time

Drivers and enablers of Adaptive

Licensing (Adaptive pathways)

Drivers

•Patient expectations: demand for timely access and

emphasis on unmet medical need

•Emerging science: fragmentation of treatment

populations and early disease interception

•Healthcare systems under pressure: sustainability and

rise of payer influence

•Pharma/investors under pressure: sustainability of

drug development

Drivers and enablers of Adaptive

Licensing (Adaptive pathways)

Enablers

•Improved understanding of disease processes, better

knowledge management

•Innovative clinical trial designs

•Rapid learning systems in the healthcare environment

•Bringing patients to the table: understanding acceptable

uncertainty

•From prediction to monitoring

•Targeted prescribing

Who are the players?

What is their main concern?

• Patients/Regulators:

• Investors/Industry:

• Payers/HTA:

Enhanced risk

Reduced revenues

Expanded use

Evidence

Access

• New methodology to generate evidence

• Redefine benefit/risk

• Adaptive trials?

• Managed entry

• Adaptive prices?

How can we address the access vs. evidence

trade-off?

• Allow timely access for patients to address unmet medical need: “the safest drug that arrives too late is of no benefit to a patient”

• Provide an environment supportive of innovation

• Provide ‘complete’ information on benefits, risks, relative effectiveness

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Competing objectives

What needs to be in place to enable adaptive

pathways? 1/2

• Culture of collaboration with patients and physicians to agree on level of unmet need and acceptable uncertainty

• Collaboration of sponsor, regulators, payers/HTA bodies throughout the life-span of a product

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What needs to be in place to enable adaptive

pathways? 2/2

• Rapid learning systems for data generation across whole life-span to minimise realised risk (as opposed to inherent risk)

• Tools to provide reasonable assurance of appropriate Rx

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Product on the market

HTA / Payer

Assessment

Regulatory

Assessment

Clinical

Development

A systems approach

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Marketing

Authorisation

Coverage

Decision

Regulatory

Submission

Comprises the entire life-span:

Development licensing coverage utilization

monitoring

•Adaptive Licensing

•Adaptive Pathways

What will change with adaptive pathways?

Transition from …

Magic moment life-span management

Prediction monitoring

RCT only toolkit for evidence generation

Big populations small populations

Focus on licensing focus on patient access

Open utilisation managed utilisation

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Launch of the Adaptive Licencing Project

On March 19 the Agency launched the project:

What is this concept about?

•early access for patients starting from a niche indication

with a high unmet medical need,

•continued collection of data (both on efficacy & safety) in

this niche indication and extension to broader patient

groups.

•Involve HTAs, patient representatives and health care

professionals throughout.

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Adaptive Licencing Project: An experiment

Advantages: Early approval and access to patients with

real need, with involvement of all stakeholders and

prospective planning to collect data

Risks: Increased number of withdrawals; uncertainties

may be higher in the initial licencing

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Conclusion

• We are on a trajectory to more adaptive pathways

• The speed of change will depend on how fast preconditions can be met

• Adaptive pathways are likely the best (only?) way to cope access with evidence

• EMA is currently running the ‘Adaptive Licensing Pilots Project’; to date: 26 products submitted, 7 selected for pilot

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Thank you

European Medicines Agency

30 Churchill Place

London E14 5EU

www.ema.europa.eu

info@ema.europa.eu