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

Summary of outcomes from 2009
EPPOSI Registries Workshop
and the 2009 RDTF Report

Tsveta Schyns
Founder and Secretary General of ENRAH

Eurordis Membership Meeting 2011 Amsterdam
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

Rare Diseases Task Force Workshop
13 March 2008, Paris, Report. II

→ 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.

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

13 – 14 May 2011
9

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

13 – 14 May 2011
10

• 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

13 – 14 May 2011
11

→ 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

13 – 14 May 2011
12

• 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

13 – 14 May 2011
13

→ 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?

13 – 14 May 2011
14

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

13 – 14 May 2011
15

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

13 – 14 May 2011
16

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)

13 – 14 May 2011
17

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

13 – 14 May 2011
18

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.

13 – 14 May 2011
19

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!

13 – 14 May 2011

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

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