International LAM Registry on the LAMsight Portal

1. International LAM Registry on the LAMsight Portal Michael Nurok MD, PhD Division of Surgical Critical Care, Cardiac and Thoracic Anesthesia Brigham and Women’s Hospital Harvard Medical School Boston, MA, USA 3rd International Congress on Pulmonary Rare Diseases and Orphan Drugs Milan, Italy March 20-21, 2009

2. What is the LAM Treatment Alliance (LTA)? (Continued) Our Approach Connecting researchers and resources Mobilizing expertise • Bringing researchers and clinicians across disciplines together • Ad hoc recruitment meetings • Monthly seminar series • International Summits • Ad hoc problem-solving Summits/ fostering collaborations / partnerships • Clinical care that stays on the cutting edge of research developments • Multi-disciplinary disease management • Global patient trial network • Clinical trials Fast-tracking core research Eliminating barriers • Accessing expertise and technologies to develop: • Biomarkers • Genotyping / expression analyses • High-throughput combination drug screens • Prioritizing work to overcome key barriers to accelerating treatment research • Ensuring fast and effective tissue procurement, banking, distribution • Cell isolation, culturing, cell line • Animal Model development

3. LAMsight objectives: • To enhance the understanding of the variability, progression, response to experimental treatments and natural history of LAM through rigorously collecting standardized patient data from the global LAM patient population; • To increase access to clinical data for clinicians, patients and researchers around the world; • To provide patients, clinicians, and researchers with access to clinical trials by cataloging a network of patients and ascertaining their willingness to have their treating clinicians contacted for future clinical trials; • To create, reinforce, and expand a network of cooperating LAM patients, clinicians, and researchers.

4. Collaborators: LAM Clinicians and Researchers LAM Treatment Alliance Ian Eslick, MIT Media Lab Dana Farber Cancer Institute Andrew Shalit, Jeremy Jones, Clozure Associates

5. Two essential components: Patient portal: • Patient entered contact information • Patient entered willingness to be contacted regarding clinical and other trials • Patient self-reported data portal International LAM Registry portal: • Clinician entered prospective and retrospective harmonized data on LAM • Participants represent approximately 1-2000 patients • Automatic and manual data import.

6. Incentives: Publish on your own data at any time Access to data about the global LAM patient population (1-2000+) Cite any publication from the Registry on your CV If anyone uses your Registry data you have the option to become an author or refuse your data use Proportional voting rights based on contributions (up to 10%)

7. Incentives: Generate surveys with instant patient access Easy, secure data management platform Technical support at no cost Address discrete studies that would un-answerable without access to the global patient population because of low numbers or rare occurrences Accelerate research towards a treatment

8. LAMsight Patient Portal

9. Statistics: Partial launch last week 87 users already • North America • South America • Europe • Asia • Australia • New Zealand

10. Public Homepage Two essential components: • Patient portal: • Patient-entered contact information (confidential) • Patient-entered information re: willingness to be contacted regarding clinical trials, other trials, surveys • Patient self-reported data portal • International LAM Registry portal: • Clinician-entered prospective and retrospective globally harmonized data on LAM patients (de-identified) • Clinicians participants represent approximately 1-2000 patients • Automatic and manual data import from clinical centers around the world

11. Data Entry Surveys and Data Acquisition

12. Data Entry

13. Surveys and Data Acquisition

14. Explore Other Patients’ Responses Visualization of data

15. International LAM Registry:Clinician Portal on LAMsight

16. Demonstration: http://newmed-temp.media.mit.edu:8080

17. Concerns and solutions: Security • All information is deidentified • Platform uses state of the art encryption Data quality • Data provenance determined • Different quality data is labeled accordingly Ownership and control • Clinicians control registry • LTA provides funding to support mission

18. Future functions: Patients can initiate linkage of their self and clinician reported Tissue specimen data linked to Registry and LAMsight Import and harmonisation of other Registry data

19. Outstanding questions: Theoretical • What do we want the Registry to do? • Off label drug use • What relationships will be useful to explore? • Data quality • Pulmonary Function Tests • Existing trials?

20. Outstanding questions: Technical • MRN to Derived ID? • Center ID not stored • Entering Center ID will produce unique ID (one way hash) • Automatic Import • Retrospective – what can be easily provided? • Prospective – what is ideal? • Higher data quality • Need templates and format to write adapters

21. Outstanding questions: • Headings and Fields • Organization *end use • Format *end use and analysis • Dates, numbers, text, decimals etc. • yes/ no, set of choices, free form, check boxes • Additional Fields • All foreseeable biomarkers? • Anything else?

22. Outstanding questions: Outreach to other clinicians? Other questions or concerns?