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

Eurordis Membership Meeting 2011 Amsterdam13 – 14 May 2011

Summary of outcomes from 2009 EPPOSI Registries Workshopand the 2009 RDTF Report

Tsveta Schyns

Founder and Secretary General of ENRAH


RARE DiseasesCharacteristics

• 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


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


→ 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


Purposes of PR in the field of RDRDTF 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


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


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


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


1. Setting the scopeWhy 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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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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2. Protocol and CRF design

• 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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2. Protocol and CRF design 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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3. Implement and Roll Out

• 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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3. Implement and Roll Out Related Issues to be brought up

→ 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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4. Daily Operations Related Issues to be brought up

→ 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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4. Output from Patient RegistriesRelated 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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4. Output from Patient RegistriesRelated 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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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


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!

Documents

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