EyeForPharma, Philadelphia – Mark Baglin, Vice President of Global Marketing at Alnylam Pharmaceuticals, works in ultra ultra rare diseases, like Hereditary ATTR Amyloidosis. It impacts 1:100,000 people – making finding them very much like hunting needles in haystacks.
Baglin is a bit of a rare disease detective specializing in finding and supporting those patients. Prior to join Alnylam, he worked in the HAE franchise at Shire.
What the two rare diseases have in common is long, complicated journeys, that often leave the patients wandering and in the dark.
Baglin showed us 45 patient journeys, many of which seemed almost random in their connections and care points. He said that for every 45 on identified journeys, there are probably another 450 still early on in finding a diagnosis.
As a rare disease company, Alnylam see clear roles for both advocacy organizations and big data applications.
Advocacy partnerships have four big impacts:
- Support and guide R&D, like the CF Foundation has done for Vertex and Kalydeco
- Inform and guide patient service models, including Shire’s One Path program
- Influence regulator and payer policy, including having patient representation and FDA advisory panels
- Identify and education patients through collaboration and synergy
Data steps in to help rare disease innovators understand patient pathways. At Alnyla, they call it an Integrated Customer-Patient Data Driven Approach.
Their system works like this:
- 250 million patient lives worth of claims data are updated daily
- They add lab results data that shows which practices have tested for rare diseases
- AI integrates and investigates that data to identify possible diagnoses or disease accelerations
- That intelligence can direct field force and non-person activity
- As well as refine the resourcing around those
- Then, the teams close that loop by feeding field and channel data back into the system to help it get smarter and more efficient over time
For patients, the results can be life changing. They can be diagnosed more quickly and get product to treat more effectively.