I must admit that I have fallen asleep at the wheel. As a grandparent of two granddaughters with rare diseases, I am often in charge of dispensing their medications; and as you might imagine these meds are critical to their care.
Recently, a colleague brought to my attention that the very drugs I dispense to those little girls may be imperiled by statutory changes proposed in Governor Cuomo’s 2014-15 Budget. So off I went to see if I could confirm the risk.
Buried in the budget bill, I found the following:
Subdivision 4 of section 365-a of the social services law is amended by adding a new paragraph (a-3) to read as follows: drugs that may not be dispensed without a prescription that are prescribed for any indication other than a medically accepted indication, as defined by federal law. The commissioner of health, a managed care provider operating pursuant to section three hundred sixty-four-j of this title, or both, may require prior authorization for any covered outpatient drug to determine whether such drug has been prescribed for a medically accepted indication as defined by federal law, and may deny prior authorization if, after giving the prescriber a reasonable opportunity to present a justification, it is determined that the drug has been prescribed for other than a medically accepted indication, as defined by federal law;
The issue here is commonly referred to off label prescribing:
Off-label use is declared when a drug was prescribed for a patient whose age was not listed on the package label, no pharmacokinetic (PK) data was listed in the package insert, and/or if the drug was used for a non-FDA approved indication.
Common sense might compel you to conclude that any and all prescribing of a drug for a purpose for which it has not been approved is the most reasonable course. However reason, as applied here would fail you; and that is especially so in pediatrics, as an FDA website points out:
Most drugs prescribed for children have not been tested in children. Before the Food and Drug Administration initiated a pediatric program, only about 20 percent of drugs approved by the FDA were labeled for pediatric use. By necessity, doctors have routinely given drugs to children “off label,” which means the drug has not been approved for use in children based on the demonstration of safety and efficacy in adequate, well-controlled clinical trials.
And the American Academy of Pediatrics makes the use of off label drugs crystal clear with the release of a report at its American Academy of Pediatrics (AAP) National Conference and Exhibition in New Orleans on October 21, 2012:
The rationale for the lack of clinical data on the efficacy of drugs on children and research to back it up is:
the historical lack of pediatric drug testing due to a combination of reasons. The primary reason is that pharmaceutical companies generally have viewed children as a market that would bring only small financial benefits. The drugs that have been adequately studied in children–vaccines, some antibiotics, and some cough and cold medicines–have a large market.
Most of us never give such immense problems much thought until a decision is imposed upon us as a result of the imposition of a thoughtless public policy. The reality here is that the very lives of all children in this state are imperiled if enormous powers are conferred upon Medicaid managed care organizations (MCO) to micromanage policies that are best left to physicians and parents of critically and chronically ill children. Over 32 million Americans are afflicted with rare diseases, half of them children where the disease is often fatal. The diagnostic process often takes up to 6-7 years and during that period parents and physicians need great latitude in treating the symptoms presented by any one of 6000-7000 disorders for which there are as few as 200 approved treatments. As the budget travels through the hearing process it would be imperative to examine the rationale that preceded this request for transferring such broad powers.
Edward J Fennell President NYS RARE Disease Alliance Coordinator, Albany Medical Center’s Rare Disease Forum 2226 East Schodack Rd. Schodack NY 12063 518-729-4262 h 518-285-9701 c Efennell43@gmail.com nysrda.com