22 of 100 Ultra-Rare Disease Patients Found

Amplity Medical

Nurse Teams Rare Disease

22 of 100 Ultra-Rare Disease Patients Found

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Nurse outreach led to finding 22 patients, of an estimated 100 in the United States, who live with an ultra-rare disease, far exceeding client expectations.

OUR TASK

For an ultra-rare disease investigational therapy, Amplity’s nurses train HCPs on diagnostic criteria, find patients, and educate testing labs to place the gene on their panels. We research the landscape, identify stakeholder information gaps, and gather insights to inform medical and commercial strategy.

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The Power of This Partnership

60k

Emails Sent

1k

HCPs Spoken To

22%

Of Known Patients Found

The Ask

CLIENT

Global

Large BioPharma

Educate Laboratories

Identify genetic testing labs, discover and analyze their tests, and get the gene for the ultra-rare disease placed on their panels.

Gather Insights

Discover barriers and facilitators to HCP knowledge + genetic testing, to inform medical affairs strategy.

Find HCPs + Patients

Prepare the market so that when the therapy for the ultra-rare disease becomes available, patients can receive it.

The Approach

TOOLS

Remote Engagement

Patient Finding

Find + Educate HCPs

Reach out to and conduct meetings with KOLs and community physicians to increase knowledge about the ultra-rare disease and testing options.

Connect With Genetic Testing Companies

Liaise with genetic testing companies to add a specific gene test to panels. Ask that the indicator gene be identified in whole genome/exome panels and shared with HCPs.

Present at Conferences

Through Congress presentations, spread the word about disease symptoms and the condition’s genetic roots amongst the community, champions, families, potential patients, and HCPs.

The Results

OUTCOME

Rare Patients Identified

Business Objectives Exceeded

Client Expectations Surpassed 700%

The client initially said that finding just 1 patient would make the effort of genetic testing lab outreach pay off. The test has been added to panels of 25+ genetic testing labs, bringing in 8 new patients solely through expanded testing.

Key Medical Affairs Insights

Provided valuable medical insights which were shared globally with client medical affairs.

22 New Patients Found

The program was responsible for finding 22 out of an estimated 100 US patients.

1000 HCPs Educated

Through ongoing emails and follow-ups, the Amplity nurse reached hundreds of KOLs and community providers.