Rare Disease Registries

Transcription

1 Rare Disease Registries Dr Marcus Klein, Senior Director, Regional Medical Officer J-APAC Anne Fabry Disease Denmark

2 Discussion Topics Evolution and increasing role of Observational Research Genzyme s Experience with Registries Looking Forward Future Direction 2

3 3 "Registries" posted on clinicaltrials.gov

4 Why have observational programs and registries become so prevalent? They can serve numerous purposes and stakeholders They can fill a critical void that clinical trials often cannot Acceptance of quality and value of registries has grown, resulting in request for more registries Ability to leverage systems, patient base, data, etc. for multiple purposes can be significant For example: regulatory and reimbursement authorities seek out registry data more frequently 4

5 Regulatory and reimbursement authorities use of observational studies & registries As part of product approvals, there is an increased expectation that longterm data are not only important, but are necessary Withdrawn products have demonstrated the need to monitor the safety profile as well as longer-term treatment outcomes in the real world, e.g.: Thalidomide, Terfenadine, Fen-phen Industry-run registry data may be required to support label changes Under-studied / special populations are of particular importance (e.g.: children, pregnant women, etc.) 5

6 Genzyme s Rare Disease Registries 6 MPS I patients

7 Rare Disease Registry Goals and Leadership Research Database Disease Management Goals To significantly advance the medical understanding of these rare diseases To improve the quality of care for patients worldwide through the publication of Registry data analyses and evidence-based disease management recommendations Leadership Scientific support and direction from Board of Advisors who are medical and scientific disease experts Operational and financial support from Genzyme

8 Rare Disease Registries A Global Collaboration

9 Rare Disease Registries: Value and Contributions Scientific: The data that is collected through the registries has helped us to better understand and document the natural course and presentation of a particular disease. This helps to educate the medical and patient communities. Clinical: Even if the data is observational, we have the ability to look at how the drug is being used in the real world, and not just during administered clinical trials. 9

10 Rare Disease Registries: Value and Contributions Regulatory: Regulatory submissions and post-marketing commitments. Policy: For patients with rare diseases, time matters. The ability to influence policy, and therefore access to treatment, is very important to the population we serve. Access: In countries where there is no uniform health coverage, a patient with a rare disease may have difficulty receiving subsidized treatment. Utilizing registry data can influence public policy to ensure treatment for patients and sustainable reimbursement in the country. 10

11 Registry support of Patient Monitoring: Recommended Schedule of Assessments Each Registry should collaborate with Expert Advisors to develop global patient monitoring recommendations

12 Registries - A Collaborative Platform to support, Disease Awareness, Patient Care, Patient Access Physicians and Care Takers Improving patient management Payers, Regulators and Policymakers Maintain market approvals, help to secure reimbursement Patients and Patient Organizations Increasing knowledge about the diseases and treatment outcomes

13 Future direction - One Disease Registry? Joined Governance Consortium governs the pooled database based on an agreed set of rules 1 Disease Registry One pooled database to conduct research on outcomes, comparative effectiveness, disease Separate Governance Companies govern their own database for their own strategic needs GZ Company X Company Y Separate Data Pools per product to manage regulatory commitments Firewalls

14 Summary Registries: A diverse and valuable source of real-life data Add complementary value to clinical trial research Increase the knowledge & awareness of the disease Support patient access to therapy Help to improve patient care: Guidelines Publications

15 Anne Fabry Disease Denmark Thank You

16 Patients are people like us. We all laugh and cry. We hope and dream. We have family and friends who love us. We want to lead normal lives. This is why we do what we do.