□ Organize a team of clinical and operational experts, led by a champion who anticipates use of the therapy within the system, to act as a primary resource to support formulary management and clinical implementation. □ Inform the appropriate decision-making bodies (e.g., P&T, medical board) about the anticipated therapy to start a transparent flow of information. Petitioning leadership to allocate resources to the formulary review process before possible FDA approval may be needed to ensure minimal time between approval and a formulary addition decision. □ Determine the effects of providing therapy on patients, providers, and the health system, with consideration of potential effects on standards of care (e.g., frequency of patient visits), implications of SMA inclusion in the Recommended Uniform Screening Panel, any significant unknowns of efficacy or safety (e.g., durability of effect, benefit for children and adults with type 2, 3, or 4 SMA), and other factors. Addressing these issues early may expedite the formulary review process. □ Address any misconceptions about gene therapies and safety of their use. For example, a high biosafety-level setting for administration of AVXS-101 is unnecessary and would add excessive burden. □ Identify efficiencies in administration. Addressing misconceptions can greatly reduce barriers to efficient delivery. Additionally, early establishment of a gene therapy delivery team and communication with leadership will help identify the ideal medication-use system. Performing a gap analysis can then allow staff to address additional potential barriers. □ Commit organizational leadership to a formulary decision as soon as feasible. In most cases, this will require prior FDA approval. Ensure decision-making bodies are well informed of any FDA review updates or decisions. □ Consider additional unknowns requiring a flexible formulary review process. These include regulatory approval, specifics of label, cost, and product availability timeline. By engaging in the review process early and developing novel ways to address these unique issues, a system will be better prepared for AVXS-101 and future gene therapies.
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