CASE STUDY

Begin with the End in Mind:
PhaseBio will Power their Rare Disease Clinical
Programs with Rave EDC — Beginning with initial
Phase I study
PhaseBio Pharmaceuticals is a clinical-stage biopharmaceutical company
committed to developing improved biotherapeutics for the treatment of orphan
diseases, with an initial focus on cardiopulmonary indications.

THE CHALLENGE
Orphan drug development presents unique challenges for any life sciencescompany. Defined as a rare disease or condition affecting less than 200,000
persons in the United States1, this presents an immediate enrollment challenge
due to the limited number of affected patients, compounded with at times
incomplete understanding of the disease and very limited prior data available todesign effective trials. Despite the smaller pools of affected patients, these drugs
still require safety and efficacy regulatory approval.
As an emerging and innovative biotech company, PhaseBio has evolved in recentyears with advanced pipeline growth opportunities, developing therapies with afocus on orphan-cardiopulmonary diseases. With growing clinical programs, theneed for a robust eClinical platform became a priority for them; promoting betterdata management, and greater efficiency through all phases of their clinicalstudies. PhaseBio identified the need for a robust data management platform
solution and outsourcing relationships that could scale up to a large, global
program.

THE SOLUTION
Along with a new, fully outsourced CRO relationship, Medidata Rave EDC willfurther enable PhaseBio to address current and future needs as they advance andimprove their therapies — providing new and improved treatments for patients
suffering from these rare indications.
Debunking the misconception that Rave EDC is just for large pharma companies,
PhaseBio found Medidata’s Rave EDC platform to be the single solution for theirfirst Phase I study, yet flexible enough to scale alongside their program growth.
“We have a very good understanding of our entire program beginning with Phase I
all the way through to Phase III, and know ahead of time that we plan to start as asingle site in Phase I, and rapidly expand as a global program,” said John Lee, chief
medical officer at PhaseBio.

1. Orphan Drug Act Pub L 97-414, as amended 1984
Copyright 2021 Medidata Solutions, Inc., a Dassault Systèmes company

1“We have a very goodunderstanding of ourentire program beginning with Phase I all theway through to Phase III,and know ahead of timethat we plan to start as asingle site in Phase I, andrapidly expand as a global program.”
John Lee,
Chief Medical Officer,
PhaseBio

THE PLATFORM OFCHOICE FOR CLINICALRESEARCH
The Medidata Clinical Cloud isthe cutting-edge platform thattransforms the clinical trial
experience for patients, sponsors, CROs, and research sites.
Designed with a unified data
platform, the Rave Clinical Cloudcreates a single source of truth
for all study-related data. Simply
put, enter data once and let theplatform master and populate it
throughout the end-to end suiteof Rave applications. Optimize
operational execution, decrease
the data entry and maintenanceburden, and reduce the numberof clinical systems across yourstudy teams. Throw away your listof passwords, you are now on atruly unified platform.

CASE STUDY

Rave EDC’s flexible architecture will support the data management trial demands as
PhaseBio advances from one site to multisite studies across the globe.
Driving factors that attracted PhaseBio to Rave EDC include the unified platform, auser-friendly solution, and forward looking capabilities. Having all clinical data on
one platform reduces the stress of merging data sets that would otherwise require
manual effort of the internal team. “Medidata Rave has the capability to scale withus, thanks to their robust data management platform, which means that we would beable to monitor multiple studies in parallel and potentially merge data sets as neededfrom these studies in order to support a streamlined development plan leading toapproval when we arrive at that stage, “ Lee noted.
In addition, PhaseBio has the distinct honor of becoming Medidata’s 1,000thcustomer. Medidata is the first and only eClinical company to achieve this milestone.

Medidata, a Dassault Systèmes company, is leading the digital transformation of life sciences.
Discovermore at www.medidata.com and follow us @medidata. Contact us at info@medidata.com | +1 866 515 6044
Copyright 2021 Medidata Solutions, Inc., a Dassault Systèmes company
Copyright 2021 Medidata Solutions, Inc., a Dassault Systèmes company

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