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Pediatric IBD Issues are Universal, with Transition Being the Next Step
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In reading our cover story this month, I found myself agreeing with our experts, knowing that the hurdles they face in pediatric inflammatory bowel disease often carry over to our adult IBD population as well.
This cover story discusses the pitfalls of insurance coverage for biologics, the combination therapy vs. monotherapy debate and the state of predictive risk models.
Medication Access
To start, the issues of medication access discussed by our pediatric gastroenterology experts exist across the board in IBD and these hurdles are only likely to be larger in the future as new drugs become more expensive while the anti-TNF biosimilars theoretically become less so. More decision-making may be ceded to the third-party payers, unfortunately.
While I can appreciate the need for insurance companies and manufacturers to make a profit, it is our duty to ensure our patients get the treatment that we deem most appropriate. The perfect example, cited in the cover story, is an insurance company requiring a pediatric patient with Crohn’s disease to fail 5-ASA agents before approving a biologic treatment.
What this insurance company deemed a first-line treatment is not actually indicated for Crohn’s disease in the first place and should not be a requirement before receiving a proven effective treatment. Part of the blame for this lies with ourselves, because for more than 20 years, gastroenterologists have tried to prescribe mesalamine for Crohn’s despite a lack of evidence. In a way, our attempt to prescribe “safe” but ineffective therapies has come home to roost.
And for this, we must “repent” and be willing to take the time to appeal the insurance companies’ denials when they jeopardize our patients’ care. We must advocate for them. You are more likely to get a positive response from an insurance company when you cite studies and/or practice guidelines in your appeals letter that best support your preferred treatment methods. Citing the literature increases the chance that you can get a denied therapy approved. I find it helpful to save the letter in a separate file folder because you may be able to re-use the arguments and points made when another patient faces a denial of coverage. At Mayo Clinic, we centralize the appeals letters so each of us can rely on the arguments of our peers and we are not reinventing the wheel.
This discussion of insurance appeals also calls to us and our societies to ensure that our practice guidelines are up to date and reflect the current standards of treatment. While this is difficult to do and sometimes only happens every 5 years or so, we must be diligent.
Our diligence will be rewarded when we are able to cite society practice guidelines in our appeal letters. It is part of the ammunition we need to get medication approvals and the best care for our patients, both pediatric and adult.
Combination vs. Monotherapy
Another complex topic that is both applicable to the pediatric and adult IBD communities is the debate between combination therapy with a biologic and immunomodulator vs. biologic monotherapy. You will see in the cover story the concern among many pediatric gastroenterologists that thiopurines are potentially detrimental to children. Yet, in adults, we advocate combination therapy in many cases.
Many have suggested that aggressive therapeutic drug monitoring (TDM) may allow one to get the full benefit of a biologic without the need for thiopurines, and that may be proven eventually, but our evidence base has not evolved to that level yet. Much of the anti-drug antibody formation occurs early on, during the induction process, before one would have had time to even check for antibodies. We do not yet have the necessary information to state unequivocally that “optimized monotherapy with TDM” is equivalent to combination therapy in terms of efficacy. I believe there is still a role for combination therapy in high-risk adults.
Predictive Models
In the cover story, our experts also comment on predictive models and their role in assessing future outcomes of pediatric IBD.
These are important and what we need to see are these models being validated in other cohorts. Oftentimes, these models are created based on a small- or medium-sized cohort and may be validated in a similar cohort from the same center that is approximately the same size.
Younger age at diagnosis in general is a risk factor for more severe disease. Certainly, patients with small bowel and ileo-colonic involvement are at higher risk for requiring surgery than a patient with just colonic disease.
But, to really, truly validate risk models, you need to validate them in large groups of patients. That is the only way they will be taken up as a clinical tool.
Transition to Adult Care
With further risk stratification, perhaps the transition from pediatric gastroenterologist to an adult practice can be assisted even further.
I ask that pediatric gastroenterologists initiate the transition process and ensure patients aging out of their care truly understand their disease and the treatments they received. I recently heard Robbyn E. Sockolow, MD, from NewYork-Presbyterian Hospital/Weill Cornell Medical Center, give a great talk on the pediatric-to-adult transition. She advised spending at least some of the visit speaking with the patient alone to test their knowledge. Hopefully this one-on-one time would show the patient the need for independence from parents when it comes to personal health care. When patients come to me as adults, they should understand their diagnosis and current and previous treatment options. Each patient should be able to independently name their medications and dosages.
As always, please let us know your opinions on these matters and join our conversations on Twitter with @HealioGastro and myself @EdwardLoftus2.
Disclosure: Loftus reports consulting with Abb-Vie, Janssen, Takeda, UCB, Amgen, Pfizer, Salix, Eli Lilly, Mesoblast, and research support from AbbVie, Janssen, Takeda, UCB, Amgen, Pfizer, Genentech, Gilead, Receptos, Celgene, MedImmune, Seres, Robarts Clinical Trials.