Organizing Observational Studies in the Region Conducting Observational Studies in Latin America

Transcription

1 Organizing Observational Studies in the Region Conducting Observational Studies in Latin America Soledad Suarez Ordoñez,MD. Registry Associate. Chiltern Latin America

2 Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to Drug Information Association, Inc. ( DIA ), its directors, officers, employees, volunteers, members, chapters, councils, Special Interest Area Communities or affiliates, or any organization with which the presenter is employed or affiliated. These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. Drug Information Association, DIA and DIA logo are registered trademarks or trademarks of Drug Information Association Inc. All other trademarks are the property of their respective owners. The speaker works as a contractor registry associate for Genzyme. Drug Information Association

3 Definition The Agency for Healthcare Research and Quality defines a patient registries as an organized system that uses observational study methods to collect uniform data to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves a predetermined scientific, clinical, or policy purpose(s).

4 Why we create a registry? Registries are being used to fill important gaps in evidence and contribute to understanding how trial results can be applied in practice Data from registries are also used to support timely decisions by regulatory agencies about safety and about coverage (payment). The FDA has also used observational data to expand labeled indications. Registries for Robust Evidence. Nancy A. Dreyer; Sarah Garner. JAMA. 2009;302(7):

5 Observational studies - Genzyme Gaucher Registry Initiated in 1991 Currently over 5,900 patients worldwide enrolled Fabry Registry Launched in 2001 Currently over 3,700 patients worldwide enrolled MPS I Registry Initiated in 2003 Currently over 900 patients worldwide enrolled Pompe Registry Launched in 2004 Currently over 800 patients worldwide enrolled

6 LSDs registries They are ongoing observational databases that track outcomes of routine clinical practice for patients with Gaucher, Fabry, MPS I & Pompe disease. Conditioned only on informed consent from patient, regardless of treatment status or type of treatment, is eligible for inclusion. Each Registry is supported logistically and financially by Genzyme Corporation. However, its governance and scientific direction is in the hands of an independent International Board

7 LSDs registries in Latin America patients. 17 countries More than 200 physicians. 5-6 coordinaros per registry

8 Registry enrollment Gaucher: 5,883 Fabry: 3,637 MPS I: 932 Pompe: 860 Average increase in patients enrolled 11% per year since Patients Enrolled , , , , , , , , July ,312 Gaucher Fabry MPS I Pompe

9 Enrollment in Latin America

10 Registry objectives Enhance understanding of the natural course of LSD s Increase disease awareness Basis for defining impact of intervention Identify prognostic indicators Develop guidelines for monitoring patients Individual patient reports Evaluate long-term effectiveness & safety of treatment options Define causes of morbidity & mortality Defining most efficient strategies for impact of therapy Registry programmes; to answer the unanswered questions

11 Stakeholders Global report Physicians Medical knowledge New insights of disease Therapeutically response Support for guidelines Country report Patients Standards of care Patient survey data SF36 Guidelines Opportunity for clinical studies Registry Rare diseases Health Authorities Cost/effectiveness Health authority reports Reimbursement Burden of disease Pharmaceutical Company Research database Treatment patterns Product utilization Support for product label extensions

12 Registry Program - Benefits Research Largest Gaucher, Fabry, MPS I & Pompe datasets in the world The primary source for lisosomal deseases manuscripts. Data quality is high. Relationship with patients >11,000 patients enrolled by >1,000 physicians from >70 countries. Improvement in patient s adherence to treatment and close follow-up.

13 Registry Program - Benefits Clinical Data Example - Gaucher Registry used as possible comparator group in Eliglustat trial Approvals, PMCs Expanded label indications. Treatment reimbursement expenses are directly tied to Registry data in some countries

14 Regulatory and Reimbursement commitments Agency / Country EMA FDA Canada Australia Belgium Phillipines Commitment Annual Report, Safety and effectiveness and quality of data Annual Report, Safety and effectiveness and immunogenicity Annual report and long term safety of therapy Information to support evaluation for reimbursement Long term safety and effectiveness of own patients Efficacy and safety post-approval

15 Quality Assurance Of Data CRFs are designed by an extensive review process, including input and review by Data Management, Registry, Medical, and Regulatory Affairs, and Registry Board of Advisors. Registries, where appropriate, use FDA clinical data standards for the content of CRFs, the technical formats for the data and standards and controlled terminology. Data collected through this Registry are entered and reviewed within a secure web-based system. All reports generated from registries data undergo quality assurance review for statistical accuracy. All data management and analysis occur in a validated computing environment.

16 Credibility - Quality of Data Source data verification (in process to be published) Accuracy of the data entered into the MPS 1 Registry, 6 sites around the world Data verified: demographic, informed consent, co/morbidities, lab data and therapy Global error rate 4,9%, mainly data entry errors, no systematic errors (9,4% of source to database error from other registries and clinical trials). Nahm ML, Pieper CF, Cunningham MM. Quantifying data quality for clinical trials using electronic data capture. PLoS ONE. 2008;3(8):e3049.doi: /journal.pone Accessed on line 17 June 2009.

17 Publications based on Registries Andersson H, Kaplan, P, Kacena K, Yee J. Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1. Pediatrics 2008;122: Sims K, Politei J, Banikazemi M, Lee P. Stroke in Fabry Disease Frequently Occurs Before Diagnosis and in the absence of Other Clinical Events: Natural History Data from the Fabry Registry. Stroke. 2009; 40: Arn P, Wraith J, Underhill L. Characterization of surgical procedures in patients with mucopolysaccharidosis I: findings from the MPS I Registry. J Pediatr. 2009;154:64 e3. Nicolino M, Byrne B, Wraith JE, et al. Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. Genet Med. 2009;11(3):

18 Challenges in the region Lack of specific regulation. Not standard common clinical practice. Difficulties in producing registry outcomes materials. Budget. Complex logistics.

19 Challenges in the region. Cont. Lost of follow up of patients in common clinical practice Understanding of the registries from physicians / regulatory agencies/irbs Awareness of rare diseases Lack of Publications policies for non English publications

20 Some initiatives in the region Newsletters quarterly to physicians Annual LatAm registry meetings Focus data collection Medical writers

21 Some initiatives in the region Patient s Materials Data review follow-up & completeness of data Data entry support in in many countries Support in regulatory presentations to EC

22 Conclusions A great tool to complement scientific information collected during clinical trials A real world scientific approach to raise awareness of orphan diseases & treatments A cost effective approach to comply with long term regulatory commitments

23 Thank you! Obrigada! Gracias!