In a couple of weeks our hacking group will meet again. A lot has happened since we were covered in SIRENSONG, a blog exploring pharmaceutical relationship marketing, emarketing and innovation with a focus on rare disorders.

We have engaged, not only students, but professors from a number of universities and professionals from our mentoring firm Kitware Inc.  In addition we have an agreement with FindZebra, which is said to be the rare disease search engine that outperforms Google.

Medicare’s Value-Based Payment Program | VBP | ACP.

Caring for loved ones of any age who have rare and long term complex chronic conditions is tumultuous and when changes are introduced into the health care system, as they have through the Affordable Care Act, we should be aware of the substantive changes. The value based payment program is one of those changes.

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:

Off-label treatments were ordered for 96 percent of all pediatric patients, and 100 percent of patients ages 13-17, in the intensive care unit of an urban children’s hospital

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.

There was an article in the NY Times the other day that caught my attention. The article was about Dr Thomas Insel, the longest serving director of the National Institute of Mental Health. Apparently he is a man of vision and immense focus, and you can presume from his tenure, not without prodigious political skills.

It was a statement of one of his critics that drew my attention. “N.I.M.H. is betting the house on the long shot that neuroscience will come up with answers to help people with serious mental illness—It does little or no psychosocial or health services research that might relieve the current suffering of patients.”

 One could say the same for the US Policy on rare diseases and a recent article in Rare Disease Journal asserts that is the case: In the United States, patients with rare diseases have limited hope for treatments due to inadequate market incentives for the pharmaceutical industry to develop these orphan drugs. The available data suggest that the US health-related policies have focused on the diagnosis of these diseases by concentrating on research, improving access to credible laboratories, newborn screening and facilitating a coordinated research effort.

 For the rare disease community and those who profess to represent it, we need to address the issue of limited therapeutics currently available and the production timeline for those on the horizon.

The facts are crystal clear; of the approximately 6000 plus rare disease, therapeutics are available for about 200 of them. When Dr Collins, Director of NIH was asked about the challenge of developing new clinical interventions, he responded that traditional research methodology takes 20 to 30 years to produce interventions.

As a caregiver to two young grandchildren with rare diseases and an activist in the field, those realities are disturbing, yet they are the facts. Based on those facts, the tasks ahead are crystal clear: first and foremost do all that is possible to address the needs of patients and caregivers dealing with diseases without cures and do all that is possible to modernize research methodology.