This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.

The outcome of this research will be used to inform various other workstreams as NNPDF works to assist families.

The core research objectives are to understand the following from Niemann-Pick patients and their families in the US

This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.

The outcome of this research will be used to inform various other workstreams as NNPDF works to assist families.

The core research objectives are to understand the following from Niemann-Pick patients and their families in the US;

• Level of health insurance literacy through assessment of health insurance terminology, and self-reported insights regarding information seeking, document literacy and cognitive skills

• If they have healthcare insurance, and if not, why not

• Types (including name) of insurance by which Niemann-Pick families are covered

• Information regarding Medicaid Waivers

• Attributes of insurance plans including, but not limited to;

  • Deductible amounts for individual and family
  • Out of pocket maximums for individual and family
  • Insurance premiums
  • Associated Health Savings Accounts
  • Associated Cost Sharing requirements
  • Services, drugs and assistive devices covered

• What the perceived out-of-pocket costs are for Niemann-Pick families (annually)

• When out-of-pocket maximums are perceived to be met for Niemann-Pick families

• Patient and HCP experience in terms of challenges with accessing desired care and coverage

  • Where are families experiencing challenges with accessing desired care and coverage? (drugs, assistive devices, services) Is it different for the different phenotypes of the disease?
  • What is the challenge? (financial, time, stress, others)
  • What is the life impact of that challenge? (work and educational impact, lack or delay of treatment, others)

Inclusion Criteria:

• Participant must be a person with Niemann-Pick disease who is 18 years or older or The parent/legal guardian of a person with Niemann-Pick disease. Please note: Parents whose child has passed on are able to participate in the RSVP. Parents whose child has passed on in the last two years are also able to participate in the interview. Please note that only one family member will be eligible to complete the RSVP and interview for one family.
• Confirmed diagnosis of Niemann-Pick disease, confirmed by membership in the NNPDF or by provision of a proof of disease form
• Able to read, write and communicate in English
• Able to grant informed consent
• Willing to complete a survey and RSVP, and to participate in a 30-minute telephone interview
• Ability to view or receive a document from the interviewer before or during the interview (web browser, ability to receive a text, fax or document by mail)

Exclusion Criteria:

- Inability to meet any of the above 6 criteria

Within the United States, patients or the parent/caregiver of patients who have been diagnosed with any of the 7 known subtypes of Niemann-Pick disease

The data for each question in the study will be analyzed and reported by Engage Health using descriptive statistics as appropriate for the nature of the data collected-frequency distributions, cross tabulations and measures of central tendency and dispersion.

September 2020