Where will doctors find subjects for rare disease clinical trials?
Background
Patients with rare diseases face a great challenge to get properly diagnosed, on average waiting 6 years from when symptoms develop. Even with proper diagnosis, currently only about 350 of the 7,000 rare and orphan diseases (those conditions affecting less than 200,000 people nationwide) have treatments.
A decisive step in the development of a therapy is the clinical trial. A clinical trial is where experimental treatments are tested on a small group of patients. However, these diseases affect so few people that many clinical trials are stopped due to inability to identify and recruit patients. If a clinical trial can’t be run, then development of a treatment can’t continue, creating a potentially life-threatening health crisis for those affected by the disease.
The Alexion Challenge
Your challenge is to recommend a “Research Study” strategy for a specific set of disorders: Lysosomal Storage Disorders (LSD). LSDs are a family of conditions that affect the cell’s ability to digest and recycle large molecules, resulting in cell death. Many diseases in this family affect children and can be fatal within a few years of birth, heightening the need for timely diagnosis.
Identify patients with the condition and develop a recruiting and monitoring strategy for a clinical trial. Your strategy should be based on an analysis that includes data regarding (1) the demographics and geographic dispersion of people by country, (2) the demographics of the people the disease affects, and (3) the location of research centers related with the capability to run the clinical trial.
Your analysis should address one or more of the following questions:
- What is the likely distribution of LSD patients across the globe? Are certain areas more likely to contain patients with the disease than others?
- Which disease(s) in the LSD family would you prioritize and why?
- Where should the clinical site be located to provide the greatest possibility of finding and recruiting patients?
- What are the key criteria for clinical site selection?
Data
Additional background about the disease
- Information about rare diseases can be found through these portals for rare diseases and orphan drug information
- LSD-specific information
- LAL-D (one example of a Lysosomal Storage Disorder) sources
- A good overview of the clinical trial challenges for LSDs
Sources of data about the disease
- LSDs incidence/prevalence from the National Institute of Health (refer to Table 1)
- Population statistics
- Healthcare/Research center locations and their specialty
- Research grant recipients are a good way to find expertise centers, such as RareDiseases.org and theNational Tey-Sachs & Allied Diseases Association
- ClinicalTrials.gov (from the NIH) is a great source of past and present trials for any disease.
- You can find useful information under the “contacts and locations” section of each trial description page.
Other Rules
- The project submissions must entirely be the work of the project team. While faculty and other individuals can help review the submission, they should not contribute to the content of the report or the solution.
- Incomplete submissions will not be considered, so make sure you have all of your submission deliverables are in the submission package.
- The contest materials must be submitted by the due dates. Late submissions will not be accepted and no extensions will be given.
- Teams must be members of an AIS Student Chapter.
General Information
Resources
Get more data using the Temple Univerity Paley Library Guides
Paley Library has created an excellent set of guides that highlight where to find even more data for each challenge.The guide also will point you to additional resources about creating effective graphics.
Create graphics with Tableau and PowerBI
All Temple University students can get a free one-year license for Tableau! Tableau is a leading, easy to use visual analytics tool. Sign up on Tableau’s website and you’ll receive an activation key. Once you install the software, start with their Tableau Training and Tutorials.
Microsoft PowerBI also allows you to create visualizations through a user-friendly interface. Their product is alsofree for download. They also have a nice “getting started” tutorial.
Map geospatial data
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Google Fusion Tables is simple. You need a regular – non-Temple – Google account.
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Carto creates great looking maps. A free-tier is available.
Create infographics
- Compose and edit using Piktochart or Infogram.
- Find great examples on Cool Infographics, Daily Infographic, and the Guardian. Updated frequently.
Get public data
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Data.gov is the Federal Government’s open data initiative. Hundreds of data sets.
- The Pew Research Center has a number of data sets on different social and technology topics.
- Socrata is an open data repository with data, mostly from government sources.
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The City of Philadelphia posts their public data to OpenDataPhilly.
Lynda resources
Lynda.com hosts software video tutorials. Acces to the following resources may require an account.
Judging
How entries will be evaluated
All entries will be evaluated by the judges in two categories: visualization and analysis.
The specific criteria for each category are:
Graphic
- Clarity (how well the graphic stands on its own without additional explanation)
- Novelty/creativity (originality of thought; surprising way of approaching the data)
- Insight (graphic aids understanding of the data)
- Utility (ability of the graphic to aid decision making)
Analysis
- Relevance (analysis relates to the problem statement)
- Completeness (degree to which the analysis answers the stated question)
- Depth (sophistication of the analysis)
- Consistency (conclusions consistent with the analysis)
Your entry will be disqualified if…
- It is submitted after the deadline.
- The attachments won’t open or are in the wrong file format.
- Your interactive graphic won’t run.
- You don’t specify the challenge you are addressing.
- Team member names and college name are not on both the graphic and the description.
