improving access to orphan drugs_eu_feb'10

8/8/2019 Improving Access to Orphan Drugs_EU_feb'10

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Andras Fehervary

Head Market Access & External Affairs, Novartis Oncology

ERDC, May 14th

Krakow

Improving Access to Orphan DrugsAn Industry Perspective


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Agenda

So far, so good. Significant number of ODs are approved

Approval is not enough

Access to orphan drugs is a multi-causal case

Orphan drugs have special challenges

What has been done to tackle access issues so far?

Examples of access issues and potential options

We are in this together: It needs good will and actions from allstakeholders

| Improving access to ODs - industry's perspective | Andras Fehervary | 14 May 2010 - ERDC Krakow | Business Use Only2


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Evolution of Orphan Drugs in the EUWhere are we now? Where do we go?


2000

How to approve ODs?

Since 2000:

62 ODs approved

> 720 OD designations

Fair & Equitable Access

2010

Approval

How to ensure access?

?

Optimized Diagnosis & Treatment

How to improve diagnosis& treatment?

?


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Patients do not have timely and equitable accessto approved orphan medicines across the EU


Data from EURORDIS survey 2007

Approval is not enough:

Number of available ODsdiffers significantly

between EU Member

States

# of orphan drugs

available

Countries that place a high value on social equity (e.g., France,

Sweden and Germany) also provide best access to orphan drugs


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Access to Orphan Drugs: a multicausal case


Access toOrphanDrugs

Affordability /reimbursement

Diagnostics

MedicalExpertise

Compassionateuse

Fundallocation

Infrastructure &

Information

Incentives forDevelopment

ApprovedODsMSs acceptanceof value of ODs


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The affordability issue Opportunity cost Budget impact (= price x number of patients)

What is the elephant in the room?

2009 budget impact of ODs*

Germany 2.5%

France 2.4%

Italy 2.5%

Spain 2.5%

UK 1.8%

Poland 1%* 2009 OD sales compared to total pharmaceutical sales; IMS data

6 | Improving access to ODs - industry's perspective | Andras Fehervary | 14 May 2010 - ERDC Krakow | Business Use Only

However, budget impact oforphan drugs is rather small:


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Access to Orphan Drugs: a multicausal case


Access toOrphanDrugs

Affordability/reimbursement

Diagnostics

MedicalExpertise

Compassionateuse

Fundallocation

Infrastructure &

Information

Incentives forDevelopment

ApprovedODsMSs acceptanceof value of ODs


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Challenges of drug discovery: Hard and long Selection


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The special case of orphan drugs

Before approval: Limited data set to establish significant benefit

In most cases no comparator or reference available

Risky development, often done by SMEs

Small number of patients for clinical trials; randomized clinical trialsusually not possible

After approval: Expansion of limited data set relies on use of product

Limited clinical data set often used to deny reimbursement

However, expansion of clinical efficacy data only possible by use ofthe orphan drug

Patients need access to OD| Improving access to ODs - industry's perspective | Andras Fehervary | 14 May 2010 - ERDC Krakow | Business Use Only9


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Evolution of Orphan Drugs in the EUProposals to address access issues


Approval Fair & Equitable Access Optimized Diagnosis & Treatment

2000 2010

How to ensure access?

Acknowledgement of ODchallenges

Proposals for solutionse.g. expertise gathering,CAVOD proposals, pilotsto assess drugeffectiveness etc.

How to improve diagnosis& treatment?

Relies largely onsuccessful solution ofaccess issues

Endorsement of newtechnologies


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General agreement that a comprehensiveapproach is necessary


HolisticCollaborative

Innovative

39 principles in 7categories

10th EPPOSI WSDeveloped 8

Recommendations

Industry

Roundtables

Pilot projects onpatient registriese.g. EUTOS


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Some of the still remaining Issues

Acceptance of OD Value by member states

Price/value/affordability

Infrastructure & Information



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Issue and Options: Acceptance of OD Value

Centralized approval (EMA):

Either unique or demonstration ofsignificant benefit over existingtherapy

Assessment takes small number of

patients in clinical trials into account

National reimbursement :

Agencies often re-assess clinicalefficacy ( delay)

Agencies might consider data notenough to decide on reimbursement( no access)


Options

Issue

Immediate reimbursement of any approved OD in all Member States

Clinical-Added Value of ODs (CAVOD) EMA: Use of all data available at time of approval to provide clinical added value to

inform and facilitate national pricing & reimbursement decisions; no delays

National authorities need to accept the assessment

First step of an improved EPAR (May 2010): review of orphan designation publicand linked to EPAR, including discussion on the justification for significant benefit over

other authorized treatments

National reimbursement :

Agencies often re-assess clinicalefficacy ( delay)

Agencies might consider data notenough to decide on reimbursement

( no access)


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Issue and Options: Price/Affordability

International Reference pricing (IRP) limits flexibility of companies

IRP does not necessarily lead to lower prices *

Most countries not willing to accept to pay according to their

capability to pay


Options

Issue description

Exploring innovative pricing approaches to address affordability

and treatment gaps

Holistic and flexible approaches could include:

Performance-based agreements (outcomes guarantee)

Financial agreements (capping/rebates, portfolio agreements)

Consumer oriented agreements (services, differential prices)

*OECD Study on Pharmaceutical Pricing Policies and Danzon/Towse: Differential Pricing for Pharmaceuticals


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Most Common Types of Innovative Pricing Models

Outcomes based / treatment response (individual

level)

Value based pricing (sub populations)

Achievement of overall treatment target (population)Performanc

e

Outcomes

Guarantee

Value added servicesOt

her

Services

Price & volume agreements

Capitation agreements (on patient or population basis)

Portfolio agreements (on patient or population basis)

Portfolio trade-offs

Capping / Rebates

PortfolioFinancia

l

Differential pricing

A

ffordability

Consumer

Oriented


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Issue and Options: Infrastructure & Information

Lack of information on rare disease therapies

Patient organisations have varying impact in individual countries

Limited medical expertise


Options

Issue description

Support for patient organizations

Implementation of national rare disease plans

Establishment of centers of excellence

Foster R&D into rare diseases

Gather expertise on EU-wide level


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Issue and Options: Others

Need for holistic collaborative and innovative approaches to acomplex set of issues


Options

Issue description

ESMO 39 Recommendations and EAARC Call to Action (Nov2009)

Swedish Presidency and 5 Projects (incl EUTOS)

EPPOSI 10th Workshop on Partnering for Rare Disease TherapyDevelopment Recommendations

Etc.


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All stakeholders need to work together

Create awareness for medical need

and patient rights Provide and request information Establish networks and communities$ Share knowledge

Patients &

Medicalcommunity

Pharma/Biotech &

Scientificcommuntiy

Authorities &Regulators

Identification of new pathways/targets

Development of new, safe & effective drugs Targeted therapies Create competitive environment Compassionate use programs

Provide incentives foster innovation Create framework for R&D Grant timely and equal access to treatment Adapt regulations to high and different needs

Ensure sustainable healthcare systems18 | Improving access to ODs - industry's perspective | Andras Fehervary | 14 May 2010 - ERDC Krakow | Business Use Only

improving access to orphan drugs_eu_feb'10

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