This panel session will explore the ethics involved in determining the costs of health care for people with rare diseases, i.e., industry investment and return in orphan drug development and delivery; payer expense of total medical costs; and biopharms' responsibility to provide access, patient assistance programs, product development, quality control and improvement. The obvious and subtle financial impact of rare diseases on affected individuals, their families, and caregivers also will be discussed.



Objectives:

Present overview of elements that affect the total cost of delivering effective care for rare diseases and how decision makers are influenced in the process and, ultimately, determine price and access to resulting therapy while balancing investor expectations.

Show what's it is like to live with rare diseases and seek unprecedented or optional treatments for disorders typically ignored by the mainstream commercial sector.

Offer insight into how biotech/pharma can demonstrate that they are caring, responsible companies through communication, development, access, reimbursement and other decisions and actions.