While living with any disease brings challenges, living with a rare disease can add additional layers to those challenges.
Finding an effective treatment can feel like a major victory, especially if you have spent months or years searching for answers. Unfortunately, that feeling of victory can quickly turn into frustration when your health insurance provider refuses to cover a treatment recommended by your doctor – a treatment that has given you hope for your future.
A health insurance denial for a disease with limited treatment options can leave you with serious health consequences. Or, overwhelming financial burdens should you opt to pay for the treatment yourself.
Insurance denials for rare disease therapies are all too common; treatments are often expensive, considered experimental, or fall outside standard insurance coverage guidelines. If you are facing not only a rare disease but an insurance denial for a treatment your doctor believes will help, a denial is not necessarily the final word.
Understanding why your insurer has denied your rare disease treatments and how to challenge such a decision can help you get the care or treatment you need. Regardless of the reason for your health insurance denial, you have the option to appeal the decision and take it as far as an independent review.
When you have a strong legal advocate by your side, the likelihood of success increases exponentially. Attorney Scott Glovsky is the advocate you want and need in your corner. Scott has spent his career helping people get justice when insurance companies renege on their obligations.
Was your rare disease treatment denied by insurance? It may seem like a difficult time, but you do not have to go through this alone. Contact us online or call 626-243-5598 to get the answers you need to move forward and take the first step in potentially getting the treatment you need.
What Is a “Rare Disease”?
In the United States, a rare disease is considered a medical condition that affects a relatively small number of people. The legal definition of a rare disease stems from the Orphan Drug Act of 1983, which defined a rare disease as one that affects fewer than 200,000 people nationwide. Although each rare disease affects a relatively small number of people, when considered together, rare diseases are surprisingly common.
Rare diseases are referred to as orphan diseases because historically there was little financial incentive for pharmaceutical companies to develop treatments for conditions that affected relatively few patients. The Orphan Drug Act encouraged research and development through incentives such as tax credits, grants, and periods of market exclusivity. Between 2017 and 2021, just under half of all FDA drug approvals were for “orphan” drugs, used to treat rare diseases or conditions.
The National Institute of Health estimates there are between 7,000 and 10,000 distinct rare diseases, affecting 25-30 million Americans (about one in ten people). Across the globe, rare diseases affect between 300 and 400 million people; half of all those living with a rare disease are children. At least 80 percent of rare diseases have a genetic component, making genomic sequencing a critical tool for diagnosis. Unfortunately, fewer than 5 percent of rare diseases have an FDA-approved treatment, which leaves patients with limited or no disease-specific therapies.
What Are Some of the Primary Challenges Experienced by Patients with a Diagnosed Rare Disease?
If you or a loved one suffers from a rare disease, you are likely acutely aware of the obstacles associated with the disease. Patients with rare diseases can spend years consulting one specialist after another in search of answers – one study estimated that the average diagnostic journey for a person with a rare disease is about six years and seven specialists. This is due to the complexity and overlap of symptoms with more common medical conditions, such as lupus or MS.
Navigating the healthcare system can be overwhelming and isolating for those with rare medical conditions and can lead to delayed diagnoses or misdiagnoses. Since only a small percentage of rare diseases have FDA-approved therapies, patients are left with no therapeutic options, “management” rather than a cure, or treatment with unapproved “off-label” medications.
Care for a rare disease can require traveling long distances to specialized centers, resulting in high medical costs, and non-covered therapies – severe economic barriers for many families. Since the patient population for rare diseases is small, it can be challenging to find doctors, nurses, and local healthcare providers who are familiar with the disease. Since rare diseases are often chronic, progressive, and disabling, mental health can be impacted in addition to physical symptoms.
Patients can struggle to maintain relationships or function in school and work settings, leading to loneliness, isolation, and depression. Additionally, while many patients diagnosed with a serious disease may be eligible to participate in a clinical trial, far fewer trials are available for those with a rare disease.
What Are Some Examples of Rare Diseases?
There are many rare diseases, with many more being discovered. Neurological and neuromuscular rare diseases include Huntington’s Disease, ALS (Lou Gehrig’s Disease), and Rett Syndrome. Rare genetic and metabolic diseases include Cystic Fibrosis, Ehlers-Danlos Syndrome, Duchenne Muscular Dystrophy, Gaucher Disease, and Pompe Disease. Rare autoimmune and blood disorders include aplastic anemia and Hemophagocytic Lymphohistiocytosis (HLH).
Some medical conditions considered “ultra-rare” include Hutchinson-Gilford Progeria Syndrome and Auto Brewery Syndrome. Drugs for rare diseases can include biologic drugs, gene therapy drugs, IVIG, drugs prescribed off-label, and super expensive drugs like Radicava for ALS or Spinraza and Zolgensma for Spinal Muscular Atrophy.
Why Are Rare Disease Treatments Often Denied by Insurance?
An insurer can deny a rare disease treatment for a number of reasons. The insurer may deem the treatment “experimental” or “investigational,” and while in some cases this may be true, in others these terms may translate into “too expensive.” Each insurer has its own definition of experimental or investigational treatments, and newly approved treatments – like emerging gene therapies – are likely to encounter significant challenges.
A treatment for a rare disease could be denied under the umbrella of “lack of medical necessity.” As with experimental and investigational designations, each insurer has its own standards for medical necessity, which often lead to disputes between treating physicians and insurers. In some cases, a specialist’s recommendation for a specific treatment for a rare disease may carry more weight with the insurer than a general practitioner’s.
Treatments for rare diseases that are “off-label” can also be denied by insurers despite the fact that off-label drug use is exceedingly common. In fact, according to NCBI, off-label drug use (prescribing a medication for a different condition, age group, or dosage than officially approved) accounts for between 20 and 25 percent of all prescriptions. In specialized fields like pediatrics and oncology, these numbers can exceed 50 percent.
While pharmaceutical companies are prohibited from marketing drugs for unapproved uses, it is both legal and routine for doctors to do so, based on emerging research and clinical judgment. Where rare diseases are concerned, off-label drug use may actually be the accepted standard of care, while still being denied by insurers.
Many insurers require step therapy before approving a treatment for a rare disease. Step therapy involves “fail first” protocols, meaning the patient must have tried other (usually less expensive) treatments, and those treatments must have failed. Unfortunately, in rare diseases, there may be few, if any, alternative treatments that allow the patient to proceed with step therapy.
Finally, some insurers may have restrictions on rare disease specialists or can dispute a claim because it is out-of-network – even if there are no in-network specialists in your area. What many of the reasons given by insurers for denying a claim come down to is simple – cost. Insurers will look for a legitimate reason other than cost to deny a treatment for a rare disease, simply because they do not want to pay the price. If you are on the receiving end of a treatment denial, this can feel entirely unfair – after all, you have held up your end of the bargain by paying your monthly premiums.
What Are Some Common Rare Disease Treatments That Often Face Insurance Challenges?
Rare disease treatments frequently face insurance hurdles due to high costs, experimental or investigational classifications, and limited long-term clinical data. A few of the specific rare disease treatments that consistently face denial or overly restrictive prior authorization include:
- Gene and cell therapies, including one-time, potentially curative treatments like Luxturna for inherited retinal dystrophy or Zolgensma for Spinal Muscular Atrophy. Insurers routinely push back on these upfront, single-dose costs by classifying them as experimental or by demanding long-term data that often does not exist.
- Specialized metabolic and medical foods are often prescribed for patients with rare conditions like Phenylketonuria (PKU), which requires specialized medical formulas and low-protein foods to prevent neurological decline. Insurers often deny coverage, claiming these are “dietary choices” rather than required medical treatments.
- Off-label prescriptions are commonly prescribed for rare diseases because there are no approved drugs specifically approved for these rare conditions. Insurers may then deny the claims, citing that the medication is not explicitly indicated for the disease.
- Ultra-orphan drugs are drugs designed for very small patient populations; because the patient pool is so small, clinical trial data are limited, which makes it almost impossible to meet traditional insurer thresholds for medical necessity.
- Enzyme replacement therapies, specialized medical equipment, home care services, and immunotherapy drugs are also frequently denied by insurers for those with rare diseases.
What Should You Do After Receiving a Denial for a Rare Disease Treatment?
The first thing you should do after receiving a denial letter for your rare disease treatment is to determine the exact reason the insurer is denying the treatment. Look for a specific denial code or criteria such as “experimental,” “not medically necessary,” “no prior authorization,” or “missing documentation.” Some issues can be resolved fairly easily, and the claim can then be resubmitted. Perhaps the code is wrong, or there is missing or incorrect information. Review the deadline, as you typically only have 60-180 days to file an appeal.
In some cases, you may need to request your claim file by calling your insurer to request the exact clinical policy guidelines that were used to determine the claim denial. If the denial cannot be “fixed,” you may need to file an appeal. This first appeal, often known as an internal appeal, requires resubmission of your claim to your insurer, perhaps including a letter from your doctor that clearly explains why you need this specific treatment.
Your doctor may explain why standard treatments are ineffective or unsafe in this letter or include peer-reviewed journal articles or clinical trial data. He or she may highlight the lack of alternative treatments for your rare disease. You may also need supporting documentation, like medical records and treatment guidelines.
If waiting on an internal appeal decision could seriously jeopardize your health, you can request an expedited appeal, which requires your insurer to issue a decision within 72 hours. If your insurer reverses its initial decision, then it will pay for your treatment. If the internal appeal is unsuccessful, your next step is an external appeal through a neutral third party.
If your claim is escalated to an independent external review, then it will be evaluated by medical experts completely independent of your insurance company. If the external review results in the decision by your insurer being overturned, the insurer is legally bound to abide by this decision. (We recommend speaking with an experienced health insurance denial attorney before filing an external appeal because this lawyer can help you understand your options, including potentially taking legal action as opposed to submitting an external appeal.) Navigating appeals can be difficult and can benefit significantly from having a strong legal advocate by your side, like attorney Scott Glovsky.
You may also benefit from speaking to the California Department of Managed Health Care, which ensures health plan members have access to equitable, high-quality, timely, and affordable health care within a stable delivery system. Attorney Glovsky can help you determine your path forward, whatever that path may be.
When Does an Insurance Denial Constitute Bad Faith?
There is a difference between an insurance denial and bad faith by the insurance company. Bad faith occurs when the insurer wrongfully denies a valid claim and acts unreasonably or arbitrarily without legal or justifiable cause. An honest mistake or a valid dispute over policy language is a legitimate denial, while bad faith occurs when an insurer places profits over policyholders’ rights. Key indicators of insurance bad faith include:
- Failing to conduct a proper investigation by ignoring key evidence or disregarding the opinions of treating physicians
- Failing to follow up on information that supports the claim
- Providing vague explanations or no explanations at all for a claim denial
- Misrepresenting the terms of the policy
- Engaging in unreasonable delays
- Retroactively altering policy terms
It can be challenging to differentiate between a bad-faith denial and a legitimate denial. A simple disagreement over the value of a claim or a miscommunication does not automatically constitute bad faith. Proving bad faith can benefit significantly from an experienced bad-faith denial attorney from the Law Offices of Scott Glovsky.
How Can an Insurance Denial Attorney from the Law Offices of Scott Glovsky Help?
Attorney Scott Glovsky can help evaluate the reason for your denial, assist you in developing solid medical evidence, help manage your appeals and reviews, and pursue litigation on your behalf when necessary. Rare disease patients already face significant medical challenges, and insurance denials can create additional barriers to potentially life-changing treatment. Many insurance denials can be overturned through strong medical evidence, expert support, and the appeals process. Attorney Scott Glovsky and his team can provide all of this and much more while helping you preserve your appeal rights and pursue every available option. Contact the Law Offices of Scott Glovsky online or call 626-243-5598 today.