Answers About Patient Assistance with Rare Medication

A few months ago, Patientworthy editor Kathy Devanny asked me some important questions about the assistance I receive from Patient Access Managers as a person living with the rare disease Cystinosis, who also takes orphan drug therapy to treat the disease progression. After I completed some research, and here are my answers:


  1. What is a Patient Access Manager

Most companies that have developed medications for rare genetic diseases have a patient services team. The team’s primary role is to help connect patients and families with available resources and access to their specialty medications.

A Patient Access Manager is a member of the patient services team. They are field based employees who travel often, meeting with patients (outside of their homes) and meeting with health providers.

Patient Access Mangers, or PAMs, are experts at navigating the complexities of the healthcare system. They act as advocates for patients and their families.

PAMs also provide reimbursement support services to health care providers that prescribe treatments for rare diseases. A PAM is there to help patients avoid gaps in coverage and avoid interruptions in therapy.

  1. Is there a standard form to have the patient sign to allow the PAM access to private health information? Could a loved one be designated to do this for a patient

Yes, an enrollment form (which is often the specialty drug prescription) must be signed. The patient does give consent and most legal consent follows HIPAA laws.

A loved one could do this, but the patient (if over 18) must call their insurance company and the pharmacy which distributes the drug to notify them verbally and often in writing of their permission for a parent/loved one to become involved.

  1. What are examples of the types of problems PAMs have helped with

PAMs help with the following:

  • Coverage for specialty medications
  • Health insurance for approved rare disease therapies
  • Navigation of health insurance landscape: open enrollment and when a patient or family loses previous insuranc

PAMs also help the patient’s physician with preauthorization approval. PAMs are reimbursement experts and a resource for doctors attempting to get specialty drug therapy access to their patients.

  1. How many patients approximately can one PAM handle

A PAMs bandwidth will vary based on different factors such as the type of medication (oral, injectable, infused) and the specific needs of the particular rare disease patient community that the PAM is supporting. Certain medications and disease states will require early and often support and communication with a PAM; and some will not require as much. The amount of cases a PAM can support will also depend on their experience. It will also depend on the different types of coverage related challenges that come with gaining access to the specific specialty medication that they support.

  1. Where do PAMs go to get their training in insurance, co-pays, and patient rights (what are the backgrounds of many PAMs)?

A typical PAM will have experience working with health care professionals as well as problem solving skills. Many PAMs are typically nurses, social workers and case managers by trade and many used to be pharmaceutical sales reps.

  1. What was one case considered their greatest success

Getting specialty drug therapies to the correct city when a patient travels is listed as a great success among PAMs. Many times families (and single adults) experience great emotional and mental stress over the loss of insurance because this results in a break in their child’s therapy routine or their own therapy routine. PAMs have often been the key to getting rare medication financially covered so a patient doesn’t have to go days or months without life-saving medication.


Rebekah Palmer

Rebekah Palmer

Rebekah Palmer is the author of two books published by Aneko Press: A Letter to my Friend and A Letter to Myself. She also has poetry included in the anthology Strength: Lives Touched By Cystinosis. She is a rare disease news curator for her blog Cystinosis Society. Please visit her author profile on Goodreads and her author page on Facebook called Jairus Daughter to ask any questions or to reach out.

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