Through the creation of a Canada-wide network of academic clinics, integrating lipid specialists, endocrinologists and cardiologists, the FH Canada registry leads to significant benefits for FH patients, clinicians and researchers, biopharmaceutical industry and government. The registry is available for clinicians to manage patient care, identify relatives for screening and treatment (cascade screening), to provide advice to general practitioners and to support collaborative studies in biomedical, clinical, health outcomes and health economics research. The data extracted for the provincial portion of the database allows administrative database research that will provide important information to key stakeholders and permit allocation of resources, and will also allow a sound and uniform rationale for the use of novel therapeutic agents and provide expert advice to regulatory agencies. At the Canadian level, the database is useful for clinicians and researchers to determine the burden of disease and the long-term effects of treatment.
Benefits for patients
Patients entered in the FH Canada registry have access to expert care in the field of lipoprotein disorders and cardiovascular disease prevention. In many cases, optimal treatment of severe hypercholesterolemia may be best provided by experts in the field. They have access to on-going clinical trials and to novel therapies. Furthermore, individual patients are offered family screening (cascade screening) to identify affected members who would benefit from early diagnosis and treatment. The identification of new FH cases by cascade screening proves to be both cost-effective and aligns with the aim of providing personalized medicine for patients with severe lipoprotein disorders.
Benefits for clinicians and researchers
For clinicians, this initiative allows a standardized approach to the screening and treatment of FH and, with the SMASH (Systems and Molecular Approach of Severe Hyperlipidemia) initiative, the ability to share information and research material on rare lipoprotein disorders (orphan diseases) between members of the FH Canada registry. The registry provides the local (i.e. clinic or hospital) framework to perform cascade screening (first, second and third-degree relatives), a cost-effective strategy to screen potentially affected subjects. The registry also links clinicians into a network of specialized clinics that favor collaborative investigator-initiated research and participation in multi-center studies. The local biobanking allows the storage of plasma/serum and DNA for future research, after patient’s approval.
For biomedical researchers, the FH Canada registry will help identifying novel genes for FH and other rare lipoprotein disorders that may lead to a better understanding of metabolic pathways and eventually the potential for novel therapeutic targets. In addition, gene-gene and gene-environment interactions research may shed light on mechanisms of disease. The FH Canada registry will allow the clinical-scientists to develop investigator-initiated research, using the pan-Canadian registry and biobanking resources as a platform. For health outcomes and population health researchers, this database will be invaluable to determine the epidemiology, regional disparities, gender effects, ethnic distribution and the effect of therapeutic modalities on outcomes. It will also allow resource allocation according to the prevalence of FH.
Benefits for biopharmaceutical industry
Pharmaceutical and biotechnology industry partners will have access to a group of lipid experts across Canada, with links internationally, and to a harmonized database for subjects with FH and rare lipoprotein disorders. With access to a network of established clinics with a unified database, clinical trials can be rapidly implemented. Access to the registry database will be provided after review of the intended research. This data will be important in determining health economics.
Benefits for government
This initiative will allow the development of clinical practice guidelines, to determine resource allocation according to need, the identification of rare (orphan disease) lipoprotein disorders requiring specific techniques, such as extracorporeal LDL filtration for homozygous FH. The initiative will allow the framework to set-up collaborative research under the peer-review systems (HSFC, CIHR). Health outcomes and health economic studies will allow resource allocation and quality control. Finally centralized genetic testing would provide molecular diagnostics in a cost-effective manner, when required. This initiative will dovetail with the CIHR C-CHANGE initiative concerning the harmonization of cardiovascular preventive guidelines in Canada. To date, we are the only country with harmonized cardiovascular disease prevention guidelines.
Benefits from knowledge translation and training
Individual academic institutions with a strong biomedical research commitment will offer undergraduate and graduate-level training in the field of lipid and lipoproteins within a country-wide collaborative research environment. Several centers will offer post-doctoral fellowships for clinicians and PhDs in lipoprotein metabolism, health outcomes and health economics research. This training of highly qualified personnel is expected to contribute to the academic and economic engine of the country. A formal training course, aligned with the National Lipid Association (NLA-US http://www.lipid.org/), will provide certification on the treatment of lipoprotein disorders. The establishment of clinical practice guidelines for adult and pediatric patients will enable clinicians to hone their clinical practice to the highest standard (Canadian Cardiovascular Society). The creation of patient- and physician-oriented websites will provide the opportunity to disseminate knowledge on FH diagnosis, treatment and research. By using well-established conferences, the aim will be to integrate novel knowledge into the yearly conference programs across Canada and to disseminate this information to practicing physicians and health care professionals.