Many pharmaceutical and biotech companies have chosen Xcenda as their strategic partner to support the complex pre- and post-commercialization challenges faced by orphan and ultra-orphan products. We apply our expertise with payers, providers, and patients to help manufacturers overcome the unique commercial challenges resulting from poor awareness and confusion when conditions have limited treatment options.

Quantify Value and Prove Unmet Need

With more than 20 years in the field of health economics and outcomes research, payers respect Xcenda’s analyses, including burden-of-illness studies, to quantify the value of an unmet need and raise payers’ awareness for the needs within their own patient populations. Using scientifically credible data to educate payers helps manufacturers avoid delays and unnecessary access hurdles. And with more of the cost of treatment shifting to the patient, it’s more important than ever for manufacturers to study the reimbursement landscape and provide a strong suite of patient and provider support services.

Take a Global Perspective

Several countries are starting to define more structured health technology assessment processes for orphan drugs. As a result, Xcenda recommends manufacturers prepare health economics and outcomes research evidence, including cost-effectiveness and budget impact analyses. When developing a global market access strategy for orphan and ultra-orphan products, manufacturers should consider price and volume, and look closely at ways to target different payers that may be important in the context of compassionate use.

Tactical Recommendations

While every product is unique, consider the following tactics to successfully commercialize an orphan product in today's challenging reimbursement environment.


12 to 24 Months Prior to Launch


  • Field reimbursement managers
  • Provider education including billing guides and reimbursement toolkits
  • Training
    • Internal teams (sales force, national account managers, government affairs, brand teams)
    • Providers
  • Global Health economics and outcomes research
    • Heath Technology Assessment (HTA) Strategy
    • Academy of Managed Care Pharmacy Format dossier
    • AMNOG Dossier
    • Economic modeling (budget impact models, cost-effectiveness models)