Tips for navigating PNH treatment access and affordability obstacles

Last updated Aug. 6, 2025, by Brad Dell

Paroxysmal nocturnal hemoglobinuria (PNH) can involve many challenges, such as fatigue and trouble swallowing.

But in addition to dealing with the physical aspects of PNH, there are the financial burdens of receiving specialized treatments and the stress of dealing with potential health insurance issues.

Fortunately, there are many resources and strategies — from learning to understand insurance policies to communicating with pharmacists and health providers — to help you navigate roadblocks to getting and affording treatments.

Read the fine print

Familiarizing yourself with the specifics of your health insurance plan can help you better advocate for your needs when talking to customer service, anticipate potential coverage hurdles for a new PNH treatment, and prepare for potential unexpected costs.

Read your full health insurance policy carefully, paying close attention to how your insurer defines “medical necessity” for rare diseases and high-cost medications.

Medical necessity refers to the services, treatments, and supplies that are essential to your healthcare. Based on this determination, an insurer decides whether or not a treatment will be covered.

You may also want to check for clauses related to “off-label prescriptions” in case your doctor prescribes a treatment that fits that description. These are medications that can be used to treat PNH but are not specifically approved for the condition by the U.S. Food and Drug Administration.

Many insurers employ policy specialists. If you are finding any of the policy details confusing, you can contact customer service to request a meeting. They may be able to define terminology and walk you through how those definitions apply to managing PNH.

Understand your insurance’s appeals process

Many prior-authorization requests for treatment are denied by health insurance companies. And while relatively few of those decisions are appealed, many appeals end up being successful.

Appealing insurance denials involves paperwork and waiting, but it may be worth the effort.

The frustration of coverage denial is multiplied when dealing with time-sensitive health matters from PNH. Understanding how to navigate appeals before denials occur might speed up your side of the appeals process. You can ask your insurer for a step-by-step guide, a list of needed documentation, and any requirements for formatting the document.

The insurer might also have a specific department, person, or board responsible for rare disease-related claims. Getting their direct contact information may help you more quickly clarify problems and speed up processes.

Speak to a manager

An insurance customer service agent may have limited information about your case file, so they won’t always have a full picture of the problems you are dealing with. They may also be more restricted than managers in what they can do to address your situation.

If you’re not making progress when speaking with your insurer’s customer service about an issue, you should say that you want to speak to a manager. This may help escalate your case’s prioritization.

Check your state’s Department of Insurance

Your state’s Department of Insurance (DOI) is responsible for ensuring that your insurance operates fairly and complies with state laws. If you feel you’ve been treated unfairly or unlawfully, contact them for guidance.

Before you even have an issue, consider visiting your DOI’s website for information on state laws to research their services. Some DOIs have rare disease advocacy programs, pre-authorization review services, or expedited complaint processes for life-threatening conditions.

Apply for financial assistance

Many PNH treatment manufacturers offer financial assistance to reduce your out-of-pocket costs. Information about applying for these patient assistance programs (PAP) typically can be found on the manufacturer or treatment information website.

You can also contact manufacturers’ patient support lines to ask if they have bridge programs. Bridge programs help you continue to access treatments when insurance coverage lapses or changes.

Some organizations and patient advocacy groups have programs that can help you cover costs related to living with PNH.

• The National Organization for Rare Disorders provides patient assistance programs that assist in obtaining medication and affording insurance premiums and copays.
• The PAN Foundation provides up to $9,400 a year in grants to cover insurance premiums.
• The Aplastic Anemia and MDS International Foundation’s Patient and Family HelpLine can point you to resources that help with financial assistance.

If you apply for a PAP or nonprofit assistance, it could be helpful to contact patient support to request a detailed walkthrough of the application process, warnings about common pitfalls, and whether specific documentation and formatting can lead to faster approval.

Build a relationship with your pharmacists

Pharmacists often have coupons that can reduce the cost of your treatment and may be knowledgeable about financial assistance programs. Building relationships with pharmacists can also improve your communication with them when dealing with urgent or complex matters related to treatment access.

Many pharmacists can also discuss with you the potential financial pros and cons of using white bagging (pharmacist delivers prescriptions to provider) versus brown bagging (pharmacist delivers prescriptions to patient), which can vary from case to case.

If you are using a specialty pharmacy for any PNH treatments, you may have access to dedicated liaisons or case managers. Relationships with these support staff can be invaluable when navigating insurance changes and managing costs.

Discuss financial challenges with your healthcare team

If affordability is a barrier to you following a care plan you’ve agreed upon with your PNH doctor, it becomes a team problem. Consider informing your healthcare team about financial hardships that affect treatments.

Your team may have experience in dealing with treatment affordability and insurance hurdles. Many hospital social workers have the training and resources to help you address treatment access obstacles.

While you are figuring out an affordability plan, your doctor may have more affordable treatment options.

If you are receiving treatment in a hospital, you can ask whether the hospital has financial assistance or payment plans.

Help providers with paperwork

If treatment coverage is denied and you appeal, your doctor may have to write a letter of medical necessity detailing why you need the treatment.

You can try to speed up the appeals process and make it easier on your doctor by doing some of the legwork for them. Send your doctor a packet with all relevant data, such as denial letters, clinical trial data supporting your treatment, personal impact statements, and relevant sections of your policy.

Navigating insurance issues and managing steep treatment costs can feel overwhelming. However, by arming yourself with knowledge and drawing on available support, you can do your best to try to overcome these obstacles and get access to the PNH care you need.

PNH News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.