Rare & Orphan Diseases

Is your orphan drug approved by regulators but still missing the necessary evidence to satisfy payers? With the average orphan drug today costing $137,782 per person/per yeara, what strategies can ensure reimbursement?

ICON experts recently convened a roundtable to discuss reimbursement and market access issues in rare and orphan disease, specifically how best to identify, obtain and communicate evidence. In Part 1: Identifying Evidence, they share lessons learned from years of orphan drug reimbursement experience:

  • Recognising differences in payer priorities
  • Early planning to collect evidence, addressing payer requirements
  • Considering observational trial designs and outcome measures
  • The value in partnering with patient advocacy groups

See how your orphan drug can benefit from the strategic expertise of ICON Commercialisation and Outcomes experts. They deliver multi-disciplinary expertise and strategic insights that integrate health economics, epidemiology, patient-reported outcomes, payer engagement, pricing and market access, to provide compelling evidence for payers.

Part 3: Communicating Evidence

Complete the form to download the whitepaper and learn the importance of setting early expectations and strategies for successful evidence communications

Transcript thumbnailPart 1: Identifying Evidence for Rare and Orphan Diseases Reimbursement Download Roundtable Transcript

Transcript thumbnailPart 2: Obtaining Evidence Get insight on how to look beyond a traditional clinical trial for gathering appropriate evidence Download Roundtable Transcript

Visit www.iconplc.com/rarediseases for more information on ICON’s expertise in Rare and Orphan Disease

aPicavet et al. Shining a light in the black box of orphan drug pricing. Orphanet J Rare Dis. 2014; 9:62