Cellular and gene therapy — a rapidly growing field that involves modifying gene expression or cell properties for therapeutic purposes — has the potential to revolutionize healthcare by shifting the focus from managing chronic diseases to alleviating disease burdens and potential cures.
But these drugs come with a substantial cost and complex delivery network.
Currently, there are 24 active FDA approved therapies — ranging from $450,000 to $4.25 million per dose — and nearly another 300 novel cell and gene therapies in development. Vizient experts anticipate that 20 of these therapies will be approved by 2026, bringing category spend to approximately $25 billion.
Here are four challenges that are top of mind for healthcare systems:
- Access: Many of these cell and gene therapy agents mandate healthcare systems to be qualified treatment centers — a requirement imposed by manufacturers — which adds additional challenges and burden to administrative functions, contracting, educational training and workforce staffing. Hospital day bed capacity also can be a challenge, making it difficult to reserve a bed for possible admission of cell and gene therapy patients.
- Cost: The most expensive gene therapy has a $4.25 million price tag, begging the question: How will hospitals and patients pay for this in the long term? This, along with discussions regarding the availability of patient assistance programs, are necessary given the high cost of cell and gene therapy agents.
- Reimbursement: Payers have yet to determine their policies on this new class of drugs, requiring healthcare systems to work closely with their finance and payer management teams to create single case agreements and ensure payment for the drug and supportive care services are in place. Reimbursement is currently handled on a case-by-case basis with a timeline for reimbursement anywhere from six to nine months, creating a substantial bottleneck in an organization's cash flow.
- Operations: There's a lack of clear guidance on the safe preparation and handling of these therapies, with package inserts only recommending universal biohazard precautions. Most approved therapies, including CAR-T agents, need to be stored at ultra-low or cryogenic temperatures and require a high coordination of care. Organizations need to assess each product individually and seek guidance from manufacturers to develop their own policies and procedures for safe handling.
So, how can pharmacists and healthcare leaders prepare? Here are four strategies to consider.
- Have discussions now on your organization's readiness for these therapies including your operations and financial spend. Educate C-suite leaders of the potential impact of cell and gene therapy to generate support and resource allocation.
- Review and address the requirements and authorization processes to become a qualified treatment center. Consider how to best manage the unique storage and handling logistics required of these drugs at your healthcare system.
- Establish a multidisciplinary team at your institution that includes all operational aspects of delivering cell and gene therapy, such as finance, pharmacy, managed care, laboratory, nursing, physician and supply chain teams.
- Leverage relationships with managed care teams and other payers to clarify considerations for single-case or outcomes-based agreements to ensure reimbursement.
Learn more about cell and gene therapy in the Vizient 2024 Summer edition of the Pharmacy Market Outlook.