Our Experienced Neuromuscular Teams Are Ready
PPD has the medical, regulatory and operational knowledge to help safely speed your treatments to patients with neuromuscular diseases. Our neuromuscular team has the global expertise required to help you overcome patient, site and regulatory challenges, gained from experience serving both emerging biotechs and larger pharma.
“My DMD patients are so optimistic about potential therapies, and with the acceleration of decentralized trial technology and the further adoption of home health, so am I”
Early Engagement Drives Success in Neuromuscular Trials
Trials for neuromuscular disorders such as Duchenne muscular dystrophy (DMD) and amyotrophic lateral sclerosis (ALS) face significant complexities: geographically dispersed patients, few sites with the requisite experience, diagnostic heterogeneity, and widely varying in-country care and health authority regulations.
With strong neuromuscular and rare disease experience, PPD’s operational, medical and regulatory experts are ready from the earliest stages of development to guide your study design to ensure you meet the stringent ethics requirements, while achieving enrollment targets, assessing the right endpoints and managing costs.
Increased focus on the cost of rare disease treatments
New rare disease products are improving outcomes for patients, but health system budgets haven’t caught up with the high prices of these therapies. Payors are managing the cost by restricting eligibility and reimbursement only to patients who are likely to have a significant benefit over standard of care. PPD has the expertise to help you address questions like these with confidence:
- What are the characteristics and disease outcomes of target patient populations?
- What value assessments are likely to drive the greatest impact for regulators and payors?
- How can we plan for and implement the right study design?
- How can we maximize access for patients?
Together in partnership, we will navigate the complex neuromuscular development journey with a shared goal in mind: to bring independence and freedom to patients with neuromuscular disorders.
Speak with one of our specialists to learn how we can help accelerate your therapy and free patients from neuromuscular disorders.