Rare Disease Report

Chapter 5: Patient Finances

Managing rare diseases can be costly. A survey of patients by Shire Pharmaceuticals and Global Genes noted that more than one-half incur direct medical expenses not covered by insurance.1 Many rare diseases require a team of specialists and medical equipment, devices, and physical therapy that may or may not be covered by insurance.

Additionally, as prices for orphan drugs rise even higher, insurance companies are going to require more detailed descriptions and explanations to make sure an orphan drug is appropriate for a patient. They need to properly vet trial data and to confirm that they only give the drug to patients who are proven likely benefit from the treatment. A recent example includes patients with Duchenne muscular dystrophy being denied access to approved medications because their symptoms were not the same as those that presented in the clinical trial that got the drug approved.

Below are some suggestions for navigating the plethora of financial issues that a patient with a rare disease and their family or caregiver may face:

Insurance Issues
It is beyond the scope of this Rare Diseases Resource Guide to provide specific assistance in dealing with the multitude of insurance coverage scenarios for the estimated 7000 rare diseases. However, to get the most benefit from their insurance coverage, patients may find it helpful to keep records of the following:
  • All medical bills, including lab work, admissions, diagnostic tests, clinic visits, and treatments
  • Insurance claims
  • Reimbursements
  • Details of any contact with insurance agents (name, date, outcome of conversation)
  • Medical costs not covered or reimbursed
  • Prescriptions provided and filled
  • Travel expenses (meals, lodging, gas, mileage) to distant specialty clinics

What Can Patients Do When a Claim Is Denied or Coverage Refused?
  • Ask the doctor to write a letter explaining why a procedure, treatment, or test was necessary, and then resubmit the claim.
  • Request a review after a third resubmission.
  • Ask to speak to a supervisor or other person in a position to reverse a decision.

Pharmaceutical Company Assistance Programs
Many pharmaceutical companies that manufacture and develop orphan drugs have patient support programs to help those with specific rare diseases deal with the multitude of economic and regulatory issues they may encounter. These patients are encouraged to go to a manufacturer’s website to see whether an assistance program is available. In many cases, highly trained case managers, who are familiar with the unique set of challenges faced by patients with specific rare conditions, are available to assist patients.

Patient Advocacy Groups
Many advocacy groups for rare diseases have websites available to assist patients needing guidance with insurance and other issues unique to their condition. A list of well-established and reputable patient advocacy groups can be found in Appendix B.

Raising Money for Financial Support
If a family member is diagnosed with a rare life-altering disease, it is very common that one parent will be unable to work full time or at all, especially when the patient is a child. Families may need to seek financial support to deal with the multitude of expenses in dealing with a rare disease. Additionally, some aspects of care may not be covered by medical insurance.

Fundraising Activities: A variety of methods are available to help families raise funds for medical expenses. It is often best to look for help from organizations and institutions to see whether they can provide assistance.

Fundraisers are a great way to bring a community together and educate its members about a rare condition. When planning a fundraiser, it is important to contact your local government to obtain the necessary permits. Advocates should consult legal experts to ensure that the activity planned is permitted.

It is also important to clearly document for participants how the money will be raised, what the money will be used for, and whether the deductions are tax-deductible. Organizations that are not 501(c)(3) organizations (nonprofits) may have difficulty raising funds.

Crowdfunding: Physical fundraising options aren’t always the easiest to organize, so many families look to the internet to raise funds for medical expenses. As with physical fundraising options, crowdfunding takes a great deal of effort to create a Web presence that can lead to success. Sites such as GoFundMe (gofundme.com), GiveForward (giveforward.com), YouCaring (youcaring.com), and Fundly (fundly.com) are commonly used by families seeking funds for medical expenses.
Crowdfunding is also used by rare disease communities and families to raise funds for research. Consano (consano.org) gives people a means to donate directly to a research project that would have only been funded by granting agencies in the past. The Rare Genomics Institute (raregenomics.org) helps families struggling to determine what rare disease a family member has and uses crowdfunding to raise money to get genetic testing for patients.

Useful Websites for Financial Assistance Programs

The National Organization for Rare Disorders (NORD) Patient Assistance Programs: rarediseases.org/patients-and-families/patient-assistance
NORD administers patient assistance programs (PAPs) to help patients obtain lifesaving or life-sustaining medication that they otherwise could not afford. The organization offers financial assistance with insurance premiums, patient co-pays, diagnostic testing expenses, and consultations with disease specialists not covered by a patient’s insurance plan. NORD also works in partnership with pharmaceutical companies and patient organizations to provide travel and lodging assistance for participants in specific rare disease clinical trials.

NeedyMeds: needymeds.org
NeedyMeds is a 501(c)(3) organization whose mission is to help people who cannot afford medication or healthcare costs. Free or low-cost prescription medications are provided to low-income patients who are uninsured or underinsured and meet the necessary guidelines.

RxAssist: rxassist.org
RxAssist is a Web-based PAP that works with drug companies to provide free or low-cost drugs to uninsured patients. Each program has its own set of rules, but usually the patient must be: (1) a US citizen or legal resident, (2) have no prescription insurance coverage, and (3) have an income that is 200% of the federal poverty level.

The HealthWell Foundation: healthwellfoundation.org
The HealthWell Foundation is a 501(c)(3) organization that assists patients with insurance who cannot afford their co-pays, coinsurance, and premiums for important medical treatments.

Patient Advocate Foundation (PAF) Co-Pay Relief: copays.org
PAF is a 501(c)(3) organization that provides direct financial support to insured patients in need of assistance. The list of diseases for which PAF provides assistance is limited. Rare diseases covered include multiple myeloma, myelodysplastic syndrome, and renal cell carcinoma.

The Patient Access Network (PAN) Foundation: panfoundation.org
PAN helps underinsured persons access healthcare by providing assistance programs for numerous diseases, including several rare diseases (eg, acromegaly, Gaucher disease, homozygous familial hypercholesterolemia, methicillin-resistant staphylococcus aureus, retinal vein occlusion, short bowel syndrome, uveitis).

Patient Services Inc (PSI): patientservicesinc.org
PSI is a 501(c)(3) organization that provides financial assistance for some rare diseases, including Fabry disease, hereditary angioedema, mucopolysaccharidosis I, and Pompe disease.

  1. Rare Disease Impact Report: Insights from patients and the medical community. Shire and Global Genes. April 2013. Available at: globalgenes.org/wp-content/uploads/2013/04/ShireReport-1.pdf Accessed September 22, 2017
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