Workshop 3 - "Outcome of the RD Task Force and EPPOSI Workshop"

Eurordis Membership Meeting 2011 Amsterdam

13 – 14 May 2011

Summary of outcomes from 2009

EPPOSI Registries Workshop

and the 2009 RDTF Report

Tsveta Schyns

Founder and Secretary General of ENRAH


13 – 14 May 2011

RARE Diseases

Characteristics

• Restricted information from randomized clinical trials (RCT)

• Often conditional regulatory and reimbursement approvals

• Need for real world data

• Low disease awareness, which affects referral and diagnosis

• Need for communication

• Not optimal disease management

• Need for improved treatment guidelines


13 – 14 May 2011

Rare Diseases Task Force Workshop

13 March 2008, Paris, 2009 Report. I

• the scarcity of RD cases imposes a large geographical

coverage of the data collection

→ multiple collaborations and exchanges of data, usually

transnational

• 371 Registries in 20 EU Countries in Orphanet database

→ 13 of which described as European or Global


13 – 14 May 2011

→ Regulations concerning registries are in early stages in

most European countries

→ With the multiplicity of actors and of rules at MS level,

the situation is difficult to comprehend

→ No guidelines are available yet on best practices for

exchanging and sharing data

→ The notion of return of benefits to research subjects or

communities is fairly recent.

Rare Diseases Task Force Workshop

13 March 2008, Paris, Report. II


13 – 14 May 2011

Purposes of PR in the field of RD

RDTF Workshop 2008. III

→ Establish natural history

→ Provide an inventory of patients for future

research

→ Assess and compare therapies’ effectiveness

→ Measure quality of care and services

A tool to assess real life situation and facilitate

research on RD


13 – 14 May 2011

1 st EPPOSI Workshop

on Patient Registries for Rare Disorders

18-19 March 2009 Brussels Belgium

Need for Data Collection to

→ Increase Knowledge on Rare Disorders and

→ Optimize Disease Management and Care


13 – 14 May 2011

Programme First EPPOSI Workshop

→ Current Situation of registries in Europe

→ Challenges for registries from the point of view of stakeholders

→ Issues at stake with registries in Europe

→ Registration of Patient Data: legal and ethical issues

→ Data collection, management and analysis

→ Governance of registries

→ Concrete recommendations


13 – 14 May 2011

Planning and Operating Patient

Registries

1. Setting the scope → strategy

2. Protocol and CRF design

3. Implement and Roll Out

4. Daily Operations

5. Output


13 – 14 May 2011

1. Setting the scope

Why and what to document?

• Each and every registry is different : there is no one recipe

for all

→ Coverage: Local, Regional, National, Global

→ Initiator: Scientist (s), National Health/HTA, Patient

Organizations or Pharmaceutical Company

• Recommendations: To establish a Working Group/Forum

with experience in setting up registries for

→ Best Practice Templates

→ Downloadable Software

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13 – 14 May 2011

1. Setting the scope

Related Issues to be brought up

→The link between the data entry and data output for optimal

design

→ The value of Disease history data for optimal evaluation of

treatment effectiveness

→ Systems at a EU level to ensure collaboration between

different registries

→ Central registration of all patient registries involving

patients from Europe

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13 – 14 May 2011


• Protocol design

→ What data to collect ?

→ Who will report: Clinician or Patient?

• Demands on application used for data entry:

→ User friendliness

→ Report generation on all or a selected patient cohort

→ Operation manuals

• Center Criteria

→ Number of centers

→ Specialist/hospitals/GP

Recommendations: Establish a Working Group/Forum

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13 – 14 May 2011


Related Issues to be brought up → The value of systems for data entry:

• user friendliness

• fexibility to difference in standard of care between countries and to changes in management approaches over time

• data structure to assure flexibility in data analysis

• in build data validation to assure data quality

not just free/cheap systems

→ The fundamental difference between Clinical Trials and Patient Registries- a RD Patient Registry needs to be continuously adapted to varying definitions (e. g. diagnostic criteria) and varying scopes

→ The relationship between a RD Patient Registry and Guidelines for diagnosis, treatment and care

→ RD Patient Registries should be based on a Network of centers of expertise in the RD

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• Protocol and Informed consent (translations)

• Submission to authorities and EC (if applicable)

• Training of centers

Recommendations:

→ Training of ethics committees

→ Reference centers with experience in data

management

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→ Patients with RD are often followed by several specialists

→ Networks of Centers of expertise do not always exist

→ Data providing centers differ and require different approach

and support

→ Being part of registries on rare disease - mandatory for

receiving a new treatment?

→ Are Patients in control of ”their” data?How long should a

RD Patient registry run? Is the IC a life-long commitment?

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4. Daily Operations

• Data management; Data Cleaning off/onsite

• AE monitoring follow up/coding

• Communication/motivation

Recommendations:

→ Dedicated documenters to visit clinics and enter data

(e.g. within the national plans)

→ Credits for Professionals and Reference centers

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13 – 14 May 2011

4. Daily Operations


→ Assure complete accurate data for regulatory purposes, no

monetary or other incentives

→ The value of motivation ” what is in it for me? “

→ Data from patient registries to be used for P&R

→ Networks of centers of expertise on the RD

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5. Output from Patient Registries

• Scientific publications in peer reviewed journals

• Reports to Authorities (regulatory and P&R)

• Presentation at congresses within the field

Recommendations (data are underused):

→ Integrate data from different sources

→ Data mining and review (but who and how was not

discussed)

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13 – 14 May 2011


Related Issues to be brought up.I

→ The value of feed back on output to all stakeholders

→ The use of other statistical approaches -for missing or

biased data

→ Data mining to utilize all collected data hidden in patient

registries

→ Output is needed to increase disease awareness

→Improve diseases management by reviewing, updating and

publishing Guidelines on RD

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Related Issues to be brought up.II

Hollak et al 2011. Orphanet Journal of Rare Diseases:

→ Fragmentation : Disease Registries versus Product Registries;

Compare patient‘s outcomes;One disease Register rather than multiple

registries

→ enhance the Quality and Completeness of the data-systems should be

developed that support physicians to submit data, in line with defined

requirements

→ registries should be supervised and analyses performed by an

Independent body and access to data should be possible on request.

→ registries should be audited at regular intervals.

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13 – 14 May 2011

Conclusions

• 8 years with the European registry for AHC

Patients & the nEUroped registry:

→ support by and support to RD Networks of

Centers of expertise

→ relate to the existing /draft and update/

Guidelines for RD diagnosis, treatment and care

→ register in a centralized (European) and

continuously updated system


13 – 14 May 2011

THANK YOU !

Special thanks to:

→ ENRAH and nEUroped Consortium

→ Elizabeth Hernberg-Ståhl

Founder Late Phase Solutions Europe

& Co-Chair of EPPOSI Workshop 2009

Thank you all!

Health & Medicine

Workshop 3 - "Outcome of the RD Task Force and EPPOSI Workshop"