An Integrated Development Plan in Rare & Orphan Medicinal Products – Accelerating Time to Approval and Strengthening Asset Value

The global pandemic has taken a heavy toll on the rare and orphan disease community and on drug development. As the industry’s only fully integrated biopharma development organization, Syneos Health® has a unique view into these challenges. We understand that novel and integrated approaches linking Clinical, Regulatory, RWE and Commercialization, including early engagement of key stakeholders, are required to accelerate products to market and de-risk the development of critical patient therapies.

To help innovative companies determine and execute the best path forward, our asset development experts from Syneos One® have developed ADAPT (Accelerate timelines, Drive evidence generation, Anticipate risks, Partner with stakeholders, be Transparent) an integrated evidence-based diagnostic tool that optimizes clinical and regulatory strategy in order to accelerate products to market and build strong value propositions for market access and adoption.

Built from a robust evaluation of over 200 orphan assets launched since 2016 with 436 orphan drug designations granted in 40 therapeutic classes, creates a best-practice benchmark, specific to each customers’ opportunity, to maximize value for all stakeholders along the product development journey. This sophisticated and innovative product development approach – informed by our breadth of capabilities, depth of therapeutic expertise and integrated perspective – builds a comprehensive and informed strategy and roadmap to ensure asset success.

Maryna Kolochavina, PharmD, PMP, Executive Director, Syneos One, Rare Diseases

Maryna has over 15 years in life cycle management of a number of complex rare and orphan conditions, including pediatric rare and ultra-rare diseases. Maryna has strong experience in planning and execution of multiple studies/project phases (pre-clinical, I, IIA/IIB, IIA/IIIB, IV) and designs, such as disease and product observational registries with prospective and retrospective data collection, pregnancy registries, observational FUP and retro chart review studies; compassionate use programs (including Named Patient Program, Expanded Access Programs, ATU, Special Access, IND); safety surveillance studies (including active and passive surveillance studies, descriptive drug utilization studies, PASS, PAES); non-interventional studies (NIS) with ePRO, HEOR and EPI modules; rare ATMP and stem cells studies, device studies, computer adaptive testing and Omics studies (tissue research). Her educational background includes PharmD in clinical pharmacology from National Medical Academy, Gdansk, Poland, and translational work in rare skin diseases. Maryna is certified ACRP, BARQA, ASQ and PMI. She is an active patient advocate for rare and orphan patients in the Patient Voice program at Charité, Berlin, Germany.

James Featherstone, Global Executive Vice President, Head, Syneos One

Dr. James Featherstone, Global Executive Vice President and Head of Syneos One, brings extensive experience in creating and executing global growth strategies with over 25 years in the healthcare industry.  Prior to joining Syneos Health Jim was  Senior Vice President at Quintiles where he was responsible for the development of their strategy consulting business in Europe and global Commercial strategy for the organization including Commercial operations in emerging markets. Dr. Featherstone’s experience includes portfolio optimization / in & out licensing & M&A and commercialization strategy.