An integrated development plan in rare & orphan medicinal products (OMPs): Significantly accelerating time to approval and strengthening asset value for all key industry stakeholders

Companies looking to develop medicines for rare and orphan diseases face a range of challenging issues:  

• Clinical and regulatory strategy must be developed and executed within a complex and changeable regulatory framework, competitive trial setting and politically sensitive environment.  

• Accelerated regulatory approval pathways means companies face early commercialization challenges that must be addressed in a compressed time frame. • Development programs can pose significant risks when knowledge about the disease state and rare patient population is limited, leading to many associated research and development (R&D) challenges. 

• Companies require exceptional relationships with geographically dispersed patients, their families and the healthcare professionals (HCPs) that comprise the disease community. Leveraging our extensive insight into these issues in the rare and orphan space, Syneos One has developed the ADAPT (Accelerate, Drive, Anticipate, Partner, be Transparent) diagnostic tool - an evidence-based approach* to benchmark an organization’s development of rare and orphan medicinal products against best-in-class industry practices. The ADAPT diagnostic tool enables companies to:  

• Identify opportunities to accelerate patient access to new therapies; 

• De-risk development and generate necessary levels of evidence; 

• Optimize synergies between multifunctional teams and regulatory and financial incentives; 

• Facilitate early patient engagement and long-term partnership with all key stakeholders; and 

• Provide support, advice and insights that build trust within in the rare disease community.

 * Based on the evaluation of a cohort of 218 assets with 436 orphan drug designations granted in 40 therapeutic classes that have been launched since 2016