Anticipate delays because of unknowns of the therapy (e.g., REMS requirements, care setting for administration) before product launch, to avoid inefficiencies during the formulary review process; the time to address unforeseen obstacles is amplified by each step of formulary review. This is especially important for therapies where age or weight cut-offs are likely to be imposed on the approved labeling.
Provide education to address concerns or misconceptions about gene therapy (such as doubts about efficacy and safety that linger from early trials), to ensure safe and efficient delivery of care.
Engage in collaboration and knowledge-sharing with local and regional providers early to assist in developing care-delivery mechanisms.
Ensure that stakeholders thoroughly understand the processes involved in adding complex, high-cost therapies to their health system formularies to maintain trust and support shared decision-making among colleagues.
Continue and improve upon a patient-focused system of advanced care.
Promote engagement between senior leadership and clinical specialists requesting formulary additions, to ensure that provision of these therapies aligns with the system’s overall mission. This interaction should facilitate better connection with patient care and, consequently, with patients themselves.
Ensure that decision-making bodies communicate the scope of their authority before any formulary evaluation. Providing clear expectations of what it means to add or deny an item to the formulary system can help avoid miscommunication throughout the process and assist in the resolution of conflicts.
Recognize that conditions requiring the therapy are frequently devastating to patients and their families. Providers involved with patient care should act as their champions while understanding the consequences of limited health care resources when a system provides high-cost therapies. The primary provider should appreciate the methodical approach that is required to responsibly add therapies to the formulary.
Establish a multidisciplinary committee that includes patient representatives, ethicists, finance experts, senior leadership, and chairs/leaders from multiple clinical disciplines to accept accountability for advising the system on major formulary decisions. This committee works together with the P&T committee to ensure shared decision-making and stakeholder engagement throughout the evaluation process.
Strive to avoid or reduce the time between potential FDA approval and payer coverage determinations (i.e., when reimbursement is possible). Unfortunately, delays are inevitable and often outside of the provider’s control. To assist both providers and patients during this period, senior leadership should consistently engage staff and the community to provide updates and direction.
