Cytotherapy Clinical Trials in Genetic Disorders of the Blood and Options for Reimbursement

Abstract Cell and gene therapies hold great promise to improve the health of patients with severe or fatal conditions. However, there are challenges to finance and reimburse these regenerative medicines. In this Chapter, the various cell and gene regenerative therapies in clinical phases of development for the treatment of bleeding disorders and hemoglobinopathies will be discussed. The US healthcare system is facing challenges about the affordability and absorbing the treatment prices of these novel therapies and raised concerns from the public, US administration and payers. The different stakeholders recognize the fact that novel innovative payments arrangements for gene and cell therapies are needed. Value-based pricing combined with annuity-based payments is an attractive solution for payers and manufacturers to participate in this shared-risk financing model where continued reimbursement is linked to the long-term efficacy and safety of these potentially transformative and long-lasting therapies.