The following is a statement provided by MASAC,聽the National Hemophilia Foundation’s Medical and Scientific Advisory Council.
Patients on home therapy receive regular shipments of clotting factor concentrate (CFC) or non-factor replacement therapy (e.g., emicizumab-kxwh) from their pharmacy providers, often on a monthly basis or as home supplies are depleted. Having an adequate supply of these treatment products at home is critical to achieve safe patient care and continuity of treatment.
Moreover, a national emergency or natural disasters (e.g., hurricanes, tornadoes, floods, earthquakes) may occur, resulting in patients being cut off from delivery for several days. During these periods of time, the need to continue regular care regimens, including prophylactic treatment remains important. Lack of an adequate supply of treatment products to cover such situations might place patients at-risk for severe complications, including death.
Therefore, MASAC makes the following recommendations:
- During a period in which a national emergency has been declared under the National Emergencies Act, or at such other times (e.g., declaration of a natural disaster, pandemic or other public health crisis) that it may reasonably be anticipated that there could be an interruption in supply, distribution, transportation or home delivery of clotting factor concentrates or non-factor replacement therapy (e.g., Emicizumab-kxwh) patients with bleeding disorder treated on regular prophylaxis should be able to obtain a prescription refill for their prophylaxis regiment when their home quantity reaches at a minimum an estimated two week鈥檚 supply.
- When refills are provided under these recommendations, the doses remaining at home should not be deducted from the doses to be dispensed for the next delivery.
- These recommendations are supplemental to those continued in MASAC Document #242 – MASAC Recommendations Regarding Doses of Clotting Factor Concentrate In The Home. The recommendations of MASAC Document 242 should apply during other periods of time.
- Whether under these supplemental recommendations or the recommendations of MASAC Document 242, health plans, home care delivery companies, pharmacy benefit managers and pharmacies should allow exceptions to allow for early refill requests based on documented work, school or travel needs where home delivery on a timely basis would not otherwise be practical.