1、Identifying clients needs and challenges in rare disease clinical developmentClinical researchppdIntroduction 3 Demographics 5 Outsourcing with a Contract Research Organization(CRO)12Patient Resources&Drug Development 18Clinical Trial Details 25 Contents2 Rare diseases survey 1INTRODUCTIONRARE DISEA

2、SES SURVEY3 Rare diseases survey by PPD,part of Thermo Fisher ScientificThis survey was conducted in April 2023 among 100 respondents who work in some capacity within the rare disease sector and who currently outsource(or plan to outsource within the next 2 years)some of their research needs to a co

3、ntract research organization(CRO).The goal of the study was to identify clients needs and the challenges they are facing in terms of rare disease clinicaldevelopment.4 Rare diseases survey study was designed to be global but with a strong bias toward North America-based respondents,who comprise 70%o

4、f the audience;20%of the audience are from Europe,while the remaining 10%are in the Asia Pacific region.The bulk of the audience(72%)works at a biopharmaceutical company,while 18%are employed by a small molecule pharmaceutical company;the remaining 10%are at R&D laboratories(6%)or academic instituti

5、ons/universities(4%).91%of the audience noted that they have at least 10 years of experience within the life science sector,and 87%are in a director-level position or higher,representing an approach that supports high relevance and strong data integrity.By design,only respondents whose companies or

6、organizations are working within rare disease,and whose companies or organizations are either currently partnering with a CRO or plan to do so within the next 24 months,were able to complete the survey.RESPONDENT EXPERIENCEOf respondents have 10 years life sciences experiences91%RESPONDENT SENIORITY