ABSTRACT
Gene and cell therapies have raised a lot of excitement in terms of its curative potential with just a single treatment in many patient populations. Advancements in gene and cell therapies have raised both excitement and anxiety in the global medical community. Lack of institutional reimbursement can cause delays in gene therapy uptake particularly for patients who do not qualify for treatment on an outpatient basis. Pricing of gene therapies can form a real dilemma for pharmaceutical companies. Gene therapies provide long-term benefits in many patients, but the treatment cost is immediate. The discrepancy between time of payment and the realization and confirmation of benefit over time create large payer challenges, particularly for orphan indications, where the price tag is inherently high. Value-based payment arrangements may offer some solutions for the specific gene therapy pricing challenges. Initial examples in Italy and Spain are likely just the beginning of new deal opportunities that can bridge patient needs and payer concerns.
