Background

Cystic Fibrosis (CF) is a relatively rare disease.  Data registries have proved to be the most effective means for obtaining demographic information and for monitoring disease characteristics, type of care, progress and outcomes.  Registries of high quality exist in many countries, including the United States, Canada, most countries of Western Europe, Australia and New Zealand.  Although registry systems vary, there is sufficient international comparability of core data to make an international repository viable.  Recent movements in this direction include the establishment of a regional CF Data Registry in Europe (1), development by the US Cystic Fibrosis Foundation of a CF data warehouse capable of holding international data (2) and proposals from Cystic Fibrosis Australia for international benchmarking for CF patient outcomes using registry data (3).

An international CF Data Network – what would it look like?

Although national and regional CF data registries publish their results regularly, usually annually, few people would know about all available CF registry information or where it can be found.  Potential users range from those with a casual interest, through the CF community of interest, to important external stakeholders such as governments and national and international NGOs. Interests range from descriptive information to more complex analytical requirements of CF treatment teams.
An international CF Data Network could offer services at several levels, with different degrees of technical and management complexity.  Three levels of service are identified and described below.

Level 1 – a portal for access to existing CF data

At the simplest level, an international CF Data Network would provide a website portal through which users could access published information from national and regional CF registries.  This would assist the general user to know what registry data exists and to find it efficiently.  It could promote knowledge of the disease outside the CF community.  Such a service could be established with minimal effort by someone who is in touch with international CF registry activity.  It can be achieved by providing simple website links from a central website and would require no formal governance arrangement.  Seeking prior agreement from individual registry managers before including a link to their website would be appropriate.  An international website coordinator could request advice from registry managers about new and forthcoming data releases, to keep the portal up to date.

Level 2 – technical and comparative data at national level

At a second level, an international CF data website would extend to include some processed national and regional data, aligning selected data into comparable presentations.  Existing published data (which could be available in a level 1 portal described above) present some problems for users because they encapsulate separate decisions about the format, content and presentation of information.  With the cooperation of national and regional registry managers, an international CF Data Network coordinator could bring the most important demographic and clinical data into a common format, aligned for easy comparison in tables and charts on the website.  Such a service would be supported by technical reference material, including definitions of CF data elements.  Some governance arrangements would be desirable for implementation of a level 2 CF Data Network.   This could take the form of a scientific/technical committee comprising registry managers and data analysts whose role would be to agree on standards for international comparison of CF data and advise on content for a level 2 CF Data Network.

Level 3 – International centre-level benchmarking

Many national registries supply benchmarking information to their contributing centres.  The benefits are limited by the national scope of such services.  A further extension of an international CF Data Network would support growing interest in benchmarking across treatment centres at an international level.  For instance, a bilateral arrangement between the US Cystic Fibrosis Foundation and Cystic Fibrosis Australia has allowed comparison of selected indicators, including lung function and nutrition outcomes, for de-identified centres. 
 Extending such activity to a broader international level could only be achieved by a multilateral agreement between national and regional CF registries. Data would be submitted to an international database at a centre level rather than patient level, so patient confidentiality is not infringed.  However, a much more sophisticated management arrangement would be necessary, including maintenance of a secure database with approved rules for access to data.  Rules may determine that centre data be identifiable within the database only at country level.  However, to obtain the full benefits of benchmarking, a managed arrangement may be required to put centres in touch with each other, to enable secondary analysis to be done.
The governance arrangements for an international CF Data Network that includes benchmarking would require a scientific/technical committee, as for a level 2 resource, and a management committee, whose role would be to determine policy, to recommend and/or appoint a coordinator/manager for the resource and to oversee ongoing management arrangements.

Governance arrangements

Governance requirements have been identified at each level described above.  However, appropriate governance for a multi-lateral agreement should be under the auspice of a trusted third party.  Such a body does not need to have scientific or technical expertise for the conduct of such a role, as this would be provided by the management and scientific/technical committee’s described above.  Cystic Fibrosis Australia, the developer of the proposal, has had discussions with Cystic Fibrosis Worldwide (CFW) about that organisation taking up this role. CFW has responded with in-principle agreement. 

Funding

Funding options have not been explored.  Possibilities include external sponsorship and/or website advertising and shared funding by contributing registries/organizations.   A business case will be prepared following receipt of initial comments on this outline and proposal.

Comments

Comments on this proposal should be directed to:
Terry Stewart
Chief Executive Officer
Cystic Fibrosis Australia

Telephone: +61 2  9878 5250
Email: terry@cysticfibrosisaustralia.org.au

References

1.            European Cystic Fibrosis Society.  ECFS Patient registry described on its website at http://www.ecfs.eu/ecfs_supported_initiatives/european_cf_registry, accessed 6 April 2010.
2.            A planned component of PortCF Version 2, the US Cystic Fibrosis Foundation’s Patient Data Registry to be launched early 2010 – communications between registry managers at Cystic Fibrosis Australia and the United States’ Cystic Fibrosis Foundation 2009.
3.            Geoff Sims. International benchmarking for cystic fibrosis outcomes using registry data.  Poster displayed at the European Cystic Fibrosis Conference, Brest, July 2009.