Where will doctors find subjects for rare disease clinical trials?


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?


Additional background about the disease

Sources of data about the disease

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

  • Submission Instructions

    What you should submit

    • The story: A graphic that reflects the major findings of your data analysis.
      • It can be a static image or interactive (such as JavaScript).
      • It can be in the form of charts, diagrams, or an infographic.
    • The background: A brief description, one single-spaced page or less, describing what’s great about your analysis and your graphic.
      • Highlight how your analysis helped you arrive at your conclusions.
      • Highlight how your graphic illustrates your key findings.

    How you should submit

    • Email your entry to analyticschallenge@temple.edu by 11:59 P.M. on March 1, 2017.
      • Make sure it’s clear which of the three challenges you’re addressing.
    • Attach the graphic and the description as separate files to a single email message with the subject “Entry for Analytics Challenge”.
    • The following must be clearly displayed on the graphic and on the one page-description: (1) the name of the challenge (Alexion, Amerisourcebergen, or Merck), (2) each team member’s name and college/university name.
    • Convert static graphics to PDF format. The one-page description must also be in PDF format. There are free tools that do this, such as PDF Creator.
    • Interactive graphics must be self-contained and run without special software on any Windows 10 PC. Web-based graphics must run using the Chrome browser.

    What happens after you submit

    • You will receive a confirmation email acknowledging your entry.
    • Finalists will be notified by email on or around March 6, 2017.
    • Finalists will present their work in front of the judging panel at the AIS SCLC April 13-15, 2017.
  • Timeline

    • All preliminary submissions must be received no later than March 1, 2017
    • Finalists will be announced on or around March 6, 2017
    • The winner will be revealed at the Student Chapters Leadership Conference between April 13-15, 2017
  • Prizes

    There will be awards in two categories: graphic and analysis. The judges will evaluate each entry on both categories. Prizes will be awarded for each category separately and an entry can only win in a single category.


    First place – $2,000
    Second place – $1,000
    Third place – $500


    First place – $2,000
    Second place – $1,000
    Third place – $500


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

  • Google Fusion Tables is simple. You need a regular – non-Temple – Google account.
  • Carto creates great looking maps. A free-tier is available.

Create infographics

Get public data

Lynda resources

Lynda.com hosts software video tutorials. Acces to the following resources may require an account.


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:


  • 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)


  • 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.