International LAM Coordinating Dinner

1
International LAM Coordinating Dinner

• ATS. San Diego. 18 May 2009

2
Agenda

1. Introduction
2. Purpose of the group
3. Clinical trials - review of current status
4. Project - Registry
5. Clinical management task force

3
Purpose of the group

• To ensure non-redundant use of funding for LAM
• To maximize collaboration between LAM clinician
  scientists
• To create community resources
• To align the interests of clinician scientists
  working on LAM
• To create opportunities for advocacy groups to
  support clinician scientists

4
Clinical Trials

• In progress
• Being considered

5
Registry Project

• Do patients diagnosed at the extremes of ages
  have different rates of disease progression?
• International Registry platform
• Commitment from groups in
• Asia
• Australia
• USA
• Europe

6
Registry 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 harmonized clinical data by
  clinicians and researchers around the world
• To create a mechanism to address discrete
  scientific questions that would be un-answerable
  without access to the global patient population
• To provide patients 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 clinician researchers and basic
  scientists
• To accelerate research towards a treatment.

7
Two essential components
Public Homepage

• 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

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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

9
LAMsight Patient Portal
10
Statistics

• Partial launch last week
• 130 users already
• North America
• South America
• Europe
• Asia
• Australia
• New Zealand

11
Two essential components
Public Homepage

• 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

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Data Entry
Surveys and Data Acquisition
13
Data Entry
14
Surveys and Data Acquisition
15
Explore Other Patients Responses
Visualization of data
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International Clinical Management Task Force

• Proposal Clinical Management Working Group
•  
• Overview Proposed creation of a forum where
  physicians seeing LAM patients from anywhere in
  the world can share practices, cases and
  challenging issues in a comfortable environment.
•  
• Context This proposal is advanced in response to
  clinicians expressing the need for a space to
  think through and discuss differences in clinical
  management practices /challenges and with
  awareness of the ongoing LAM guidelines process.

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International Clinical Management Task Force

• Working Group Approach
• Open-ended focus exploratory
• Focused on sharing differences in practices,
  interesting cases, key challenges
• Comfortable environment in which to share
• Globally inclusive - open to interested
  clinicians seeing LAM patients anywhere in the
  world
•  

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International Clinical Management Task Force

• Potential Benefits
• Open-ended focus exploratory
• Potentially identify new areas in need of
  critical evaluation
• Potentially indentifying areas in need of funding
• Potentially identify questions that can be
  addressed through International LAM patient
  registry
• Process fully funded

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International Clinical Management Task Force

• Proposed Meeting Schedule
• Flagship meeting would take place within 5 months
  in an attractive location, LTA funded
• Annually at ERS
• Annually at ATS