An uphill battle
Pediatric dermatologists share struggles and successes obtaining treatments for their patients
Feature
By Allison Evans, Assistant Managing Editor, December 1, 2021
In a perfect world, a dermatologist would diagnose their patient’s condition and prescribe a therapy, and the patient would receive treatment. Unfortunately, in reality, dermatologists diagnose a condition, prescribe a treatment, and then engage in hours, days, or months of back-and-forth with insurance companies, pharmacies, and patients. The result is often a less-effective therapy, wasted time and energy for everyone involved, and increased burden on the health care system.
In this month’s DermWorld, pediatric dermatologists share stories of their struggles and successes getting needed treatments to their young patients, and insurance policy experts provide a framework in which to understand how federal rules may apply, particularly with patients on Medicaid. While geographic location and patient demographics impact their experiences, they all have faced obstacles getting the right treatments to their patients in a timely manner — or even at all.
Denials: Private vs. Medicaid
Wisconsin Medicaid tends to have a rather formulaic process, said Stephen Humphrey, MD, FAAD, member of the Society for Pediatric Dermatology and assistant professor at the Medical College in Wisconsin. “Most of the time it’s pretty reasonable. Commercial insurance can be trickier because there are so many different plans, and we never know if a patient has a high-deductible plan unless they outright tell you. They may have a treatment that’s covered that you think shouldn’t be a problem. Then you may find that the topical was covered, but it was still $400 dollars because the patient hadn’t met their deductible.”
It is a different experience for pediatric dermatologist Heather Brandling-Bennett, MD, FAAD, member of the Society for Pediatric Dermatology and associate professor in the Department of Pediatrics at the University of Washington School of Medicine. “I work at a children’s hospital where about 50% or more of our patients are on Medicaid. Washington state Medicaid [Apple Health] has strict rules about what they will cover in terms of topicals and systemics.” She also finds dealing with private payers is becoming more challenging as it is unclear what they may or may not cover.
There are some medications for indications that are categorically denied, Dr. Humphrey said. “It’s frustrating because you’ve done your work as a physician to show that a treatment could be effective, and you’ve provided literature indicating that it’s a safe treatment and the benefits probably far outweigh the risks. But at the end of the day, insurance companies sometimes have the final say, which is disheartening.”
“I send appeal letters all the time for treatments I have prescribed, and think are appropriate for the patient, but insurance has initially denied,” said Julie Dhossche, MD, assistant professor of Pediatric Dermatology and associate program director of dermatology residency at Oregon Health & Sciences University. “Often it is just a matter of sending a letter explaining why the therapy is appropriate for this particular patient and they’ll approve it; other times it takes a peer-to-peer review. But, even with jumping through those multiple hoops, treatments get denied all the time,” she said.
“If a patient’s doctor picks up the phone and has a civil conversation with the utilization review people, in about 75% of the cases, they reach an accommodation,” said Sara Rosenbaum, JD, the Harold and Jane Hirsh Professor of Health Law and Policy at the Milken Institute School of Public Health at George Washington University. The vast bulk of reversals happen at the peer-to-peer stage as opposed to the next level of appeal, which could be within the insurance company or at the state level, she added.
“However, in the case of Medicaid, because of the special Early, Periodic, Screening, Diagnostic, and Treatment (EPSDT) benefit, everything’s different,” Rosenbaum said (see sidebar).
Medicaid’s pediatric protections
The current protections for the pediatric population are twofold: There is the Medicaid Drug Rebate Program, which requires an open formulary for all beneficiaries, and the Early, Periodic, Screening, Diagnostic, and Treatment (EPSDT) benefit, essentially reinforcing that kids are eligible for all necessary treatment, whether the treatment is generally covered by Medicaid or not, said Edwin Park, JD, a research professor at the Georgetown University Center for Children and Families.
Understanding the Medicaid Drug Rebate Program (MDRP)
The MDRP was created in 1990 by the Omnibus Reconciliation Act in response to rising drug prices and projected increased Medicaid spending. A manufacturer who wants its drug covered under Medicaid must enter into a rebate agreement with the secretary of the U.S. Department of Health and Human Services stating that it will rebate a specified amount related to the drug to the states, who in turn share the rebates with the federal government, Park explained.
“In exchange, Medicaid programs cover nearly all of the manufacturer’s FDA-approved drugs in an open formulary, including those under the accelerated-approval pathway,” said Park. “However, states can impose utilization management tools like prior authorization, preferred drug lists, step therapy, and others.” This approach is different from private insurers and Medicare Part D, which can use closed or more restrictive formularies, he said. The Medicaid rebate amount is set in statute and ensures that the program gets the lowest price (with some exceptions).
“A February 2021 study from the Congressional Budget Office (CBO) compared brand-name drug prices, net of rebates and discounts, across select federal programs and agencies including Medicaid, Medicare Part D, and the Department of Veterans Affairs (VA). It found that Medicaid gets the lowest net prices, far below that of Medicare Part D plans and well below even the prices negotiated by the VA,” said Park. The CBO study also found that Medicaid had the lowest net price for specialty drugs. “Medicaid did far better for non-specialty drugs than any other federal program or purchaser examined by the CBO,” Park said.
Even though the CBO study has shown that the Medicaid Drug Rebate Program results in lower prices, Medicaid policy continues to be debated, with some arguing that the program should shift to a more restrictive or closed formulary like the VA and private insurers, to save money, particularly on new blockbuster and high-cost drugs, Park noted. “The CBO study, however, makes clear that the Medicaid Drug Rebate Program can already obtain very low net prices without eliminating the open formulary protection, which could lead to state Medicaid programs unduly restricting beneficiaries’ access to needed prescription drugs.” There is renewed policy interest in the MDRP as states and the federal government explore policies related to drug costs.
Federally required pediatric coverage
Medicaid has very strong protections for children, said Park. State Medicaid programs must provide Medicaid enrollees under age 21 with comprehensive and preventive health care services through the EPSDT benefit. Services for children are covered under the EPSDT benefit if the services are deemed medically necessary for the individual beneficiary, according to the National Academy for State Health Policy (NASHP). Federal law requires states to cover “necessary health care, diagnostic services, treatment, and other measures…to correct or ameliorate defects along with physical and mental illnesses and other conditions discovered by the screening services, whether or not such services are covered under the state plan.”
The federal statute does not define “medical necessity” but rather describes a broad standard for coverage without providing a prescriptive formula for ascertaining necessity, the NASHP website explains. States can establish their own parameters for medical necessity decisions as long as those parameters are not more restrictive than the federal statute.
“Under these general principles, the presumption is always toward coverage,” Park said. The type of Medicaid and/or CHIP program a state employs (see “How does CHIP fit in?”) will have an impact on the level and flexibility of treatment coverage, he explained. “It’s on a state-by-state basis to determine how they would consider coverage of particular conditions and the drugs that go along with them.”
“Managed care plans and Medicaid have their own internal appeals process,” Park said. “There is always what’s called a ‘right to a fair hearing’ in which there has to be an option for an external appeal. So, there are due process protections for individuals.”
How does CHIP fit in?
Enacted in 1997, the Children’s Health Insurance Program (CHIP) was designed to layer onto Medicaid and expand coverage to uninsured children. Unlike Medicaid, which has open-ended funding, CHIP is a block grant program with time-limited funding and requires congressional extension of funding (currently funded through 2027).
“States can have a CHIP-funded Medicaid expansion (M-CHIP), a separate CHIP program, or a combination approach, which is what most states use,” Park said. “For states that have separate CHIP programs, they can have more restrictive formularies than Medicaid. States can require EPSDT-like protections, although drug manufacturers are not subject to the Medicaid Drug Rebate Program.”
In states where CHIP is administered as separate program, plans could be governed by a standard of medical necessity that is distinct from Medicaid EPSDT and that might look more like normal private insurance rules, which are more restrictive, said Sara Rosenbaum, JD, the Harold and Jane Hirsh Professor of Health Law and Policy at the Milken Institute School of Public Health.
Vitiligo
“Personally, vitiligo is a condition that has been very frustrating to treat,” Dr. Brandling-Bennett said. “We’ve had a lot of trouble getting Medicaid to cover anything for vitiligo, including phototherapy, which our state dermatology society is working to address. The response from the insurance company is that vitiligo is considered a cosmetic condition, which is really troubling, particularly when you consider this from an equity perspective,” she said.
“Labeling these conditions as cosmetic is not really looking at the whole picture of a patient and what it means to have those conditions. Having vitiligo as a white person is very different from having vitiligo as a dark-skinned patient. It’s just not equitable,” she continued.
“Insurance will cover topical steroids, which can only be used for a very brief period of time,” Dr. Brandling-Bennett said. “State-funded insurance, for example, won’t cover topical calcineurin inhibitors, which have become first-line treatment for facial vitiligo, particularly for eyelid involvement.”
When you have children with health needs who are on Medicaid, the clinician should never take no for an answer, Rosenbaum said. “It may take some negotiation. It may take some modification of a treatment plan, but in the case of children, the concept of ‘cosmetic’ really doesn’t exist.”
In situations where children on Medicaid are denied treatments for conditions like vitiligo, physicians should ask for a meeting with the medical director of the state’s Medicaid agency, Rosenbaum advised.
Despite many appeals on behalf of her vitiligo patients, Dr. Brandling-Bennett has met with many hard and fast denials. She is currently working on a research project in which her findings may be able to be used to persuade Washington state Medicaid to reconsider some of its coverage policies that have been detrimental to her young patients and their families. (Read about how Dr. Dhossche successfully changed Oregon’s Medicaid policy in the “Acne” section.)
Alopecia areata
Severe alopecia areata is a condition that pediatric dermatologists struggle to get insurance to cover treatments. “Not just the smaller patches of hair loss, which we can normally treat with topical or intralesional steroids, but children with widespread hair loss. Trying to get JAK inhibitors approved for hair loss is extremely difficult. They’re not FDA approved for this indication, which is a big sticking point,” said Dr. Humphrey.
Although the lack of FDA-approved indication is applied selectively as grounds for treatment denial, said Andrea Zaenglein, MD, FAAD, professor of dermatology and pediatric dermatology at Penn State College of Medicine, “they sometimes have no problem recommending certain drugs that have no FDA indication.”
“Methotrexate, which is an inexpensive medication sometimes used to treat alopecia areata off-label, is not challenging to get covered,” Dr. Humphrey added.
Insurance companies do not recognize the psychological impact of having severe alopecia areata, said Dr. Zaenglein. “Tofacitinib, a JAK inhibitor with promise for managing alopecia areata, is currently in clinical trials for adults. It can be quite effective for kids with alopecia areata, and it’s approved for rheumatoid arthritis down to age two. So, we do have safety data on it now, but you have to fight tooth and nail to get it covered.”
“Getting expensive medications for off-label use, even if there are good articles or good data for the pediatric indication, can be difficult,” Dr. Humphrey added.
In her career so far, Dr. Brandling-Bennett has only managed to get one private insurer to pay for a systemic JAK inhibitor. “As a pediatric dermatologist, I’m a believer in methotrexate. I use a lot of inexpensive medicines and don’t jump on biologics right away. But I’m treating a 10-year-old girl who won’t take her hoodie off. She’s withdrawn and depressed because she has no hair — and that’s far from a ‘cosmetic’ issue.”
“Most of my patients with alopecia areata have psychological manifestations from it,” Dr. Zaenglein said. “They have to see counselors. They’re on medications. To say that it doesn’t have an impact is really just ignoring the reality of the disease.”
Acne
“When I started as a dermatology resident in Oregon, I found out that the state insurance did not cover acne,” said Dr. Dhossche. “I had a patient who was suicidal and whose severe, scarring, and painful acne was a reported factor in his feelings.”
The patient’s isotretinoin treatment was denied despite several appeals, which prompted Dr. Dhossche to look into how these decisions were being made. “In Oregon there is a committee that meets every few months to review diagnoses and treatment evidence, and then decides whether a diagnosis will be covered or not, and what kind of surgical or medical treatment would be covered. I emailed the committee (the email address was on their website) and asked them if I could meet with them and provide expert evidence to advocate for a change in the coverage of severe acne.”
The committee agreed to hear from Dr. Dhossche and asked her to provide a document with her argument and references. She worked with one of the committee members via email and formulated a proposal, which she presented at the end of her testimony as the exact policy change she thought should be made. Dr. Dhossche was successful — the Oregon Medicaid program decided to cover the use of isotretinoin for severe acne.
In addition to her success with isotretinoin, Dr. Dhossche has also persuaded Medicaid to cover moderate-to-severe hidradenitis suppurativa, although there is a step treatment requirement with topicals and oral antibiotics before escalating to treatments like acitretin, infliximab, and adalimumab. Soon, laser treatment of port wine stains will be covered if it is on a patient’s face. She is currently preparing testimony to advocate for coverage of vitiligo.
Occasionally, some insurance companies will approve isotretinoin, but they may put a time limit for use, Dr. Humphrey said. “I’ve had a couple of patients who have completed five months of treatment but were below goal dose because I needed to decrease the dose for side effects or for other reasons. They’re not at goal dose, they still have acne, and insurance requires a two-month break before I can get it approved again.”
In addition to flat-out denials, step therapy requirements prevent patients who need a particular drug from accessing it in a timely manner. “I had a patient come in with scarring acne that needed isotretinoin on their initial visit with me,” Dr. Humphrey said. “I received a denial saying that I had to try topical or oral antibiotics first. I wrote a letter that explained that I could prescribe doxycycline and topicals now, which would not only waste time, and increase health care expenditures, but in three months I would still be prescribing that patient isotretinoin, because it is the most appropriate treatment for them. The insurance company ended up approving it.”
Other insurance hurdles
While going to battle — and winning — for a patient can be motivating, these battles don’t always end in victory. “I ended up writing a prescription for adalimumab for a seven-year-old patient who had a needle phobia but failed methotrexate and other psoriasis treatments,” Dr. Brandling-Bennett said. “Originally, I wrote for ustekinumab, which is FDA approved for age six and above. Insurance said I had to try etanercept or adalimumab. Adalimumab is not even FDA-approved for pediatric psoriasis in the United States. It’s approved for other pediatric indications, and it’s approved in Europe from age four and up, but it’s not actually approved for pediatric psoriasis in the United States.”
Dr. Zaenglein has been actively fighting for a teenaged patient with alopecia areata with Tricare. This battle has been drawn out for more than a year. “There’s no peer-to-peer process. Tricare just denies treatments with no mechanism for physicians to appeal.”
Like the obstacles Dr. Brandling-Bennett faces with coverage of topical calcineurin inhibitors for alopecia areata, Dr. Zaenglein also finds them to be extremely difficult to get approved for children under two with atopic dermatitis. “Sometimes I’ll have a six-month-old with very severe atopic dermatitis that needs a steroid-sparing agent. It may take a month to go through the appeal process. Meanwhile, this may lead to overusing topical steroids, which poses a big risk to that child.”
“Even though there’s adequate safety data and long-term safety studies for topical calcineurin inhibitors, we still have to appeal for each patient,” Dr. Zaenglein added. “The amount of energy you have to put into getting it approved is disproportionate to the risk to the patient.”
Fighting for patients
“If you can get somebody on the phone and appeal to them directly, it’s hard to say ‘no’ once you show how these kids are affected. But some still do, and you have to keep fighting,” Dr. Zaenglein said.
Don’t assume that the people involved in these coverage decisions know the information that you do, as an expert in dermatologic conditions, Dr. Dhossche said. “I would encourage physicians to look into their state’s Medicaid set up and learn who the people are who are involved in making coverage decisions. It may also be helpful to look into advocacy efforts that are being done by your state medical or dermatology society that you could get involved in.”
Appealing coverage denials can take hours or months of time, which can be prohibitive. “I hunker down and get in fightin’ mode,” Dr. Zaenglein said. “You may have to appeal three or four times, and some insurance companies have restrictions on how often you can appeal within a particular time period.”
My favorite thing is when I get a win, Dr. Zaenglein said. “I need to go to battle. You’ve got to do it for your patients. I wish it was easier, but it’s not.”
