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Surveys & Market Research

Newborn Screening Research Study

NNPDF, APRMF, NPCanada and Firefly Fund Seek Your Participation in a first-of-its-kind Newborn Screening Research Study

NPC community foundations in the US and Canada are partnering with AllStripes to create a research database that will power multiple NPC research studies and we need YOUR help to get the first important sibling study underway. This first research project with AllStripes, referred to as the sibling study, will focus on the question of whether early diagnosis and intervention for NPC result in better health outcomes and quality of life for patients. Results from this study may be used to seek a nomination for NPC to be added to newborn screening lists across the country, including the federal Recommended Uniform Screening Panel (RUSP).

We need NPC caregivers in the US, Canada and the UK that are willing to sign-on, sign-up, register, and consent to research for this important study. It will take you less than ten minutes and all data shared with researchers is de-identified, or anonymized. No identifying information is shared.

The sign-up process is EASY and fast. One parent can sign-up multiple children under his/her account. Go to: allstripes.com/NPC.

Important Notes:

  • Presently, the research can only include patients living in the US, Canada, and the UK.
  • Caregivers must consent to research, complete the HIPAA waiver and list at least one facility where the patient has received care.
  • Patients own their data and parents will have access to their child/children’s records in a secure portal.
  • There is NO cost to participate.

Questions? Contact Joslyn Crowe, NNPDF Executive Director: jcrowe@nnpdf.org


ASMD Market Research Opportunity

NNPDF has been asked to share the following ASMD market research interview opportunity. Evidera is seeking to conduct phone interviews with adults with ASMD to better understand the ASMD journey.

Description of study:
This ASMD adult patient study involves developing health state vignettes that describe living with ASMD. Evidera hopes to speak with adults living with ASMD (consultants) to ensure these health state vignettes accurately reflect the patient experience.

Time commitment for each consultant:
The telephone interview would last approximately 60 – 90 minutes. In some cases, Evidera may request to speak with consultants a second time after revising the health states based on their feedback. Evidera typically contract consultants for up to 3 hours to ensure their time is covered.

Payment for consultants:
All consultants would be reimbursed at a rate of $100/hour (up to three hours) for their time and consultation.

Next steps:
If you are interested in learning more about this market research opportunity for adults living with ASMD, please email Laurie Turner, Family Services Manager at lturner@nnpdf.org.

06/23/2021


ASMD Accelerate Study

Wylder Nation Foundation has recently partnered with PicnicHealth to launch the ASMD Accelerate study as a way for families to easily contribute to advancing research for ASMD.

The study is currently enrolling both patients with Infantile Neurovisceral ASMD (NPA) and Chronic Neurovisceral ASMD (NPA/B).

There are two “caregiver” scenarios for enrollment:

  1. Those who are currently on the medical journey and caring for a child with NPA or NPA/B For these families, participation will not only help researchers better understand the disease but will also provide them with digital access to all of their medical records in one central location and give them the ability to share records with whomever they feel necessary such as other doctors or specialists.

  2. Those whose child has unfortunately passed away from NPA or NPA/B For these family’s participation is equally important to helping researchers better understand the disease and can serve as a way to carry on their child’s legacy and hopefully improve outcomes for future children diagnosed with the disease.

Enrolling in the study is easy and can be done via the study landing page. Once enrolled all you will need to do is provide a list of the doctors and/or care facilities your child has visited, and Picnic Health will do all the work of obtaining the medical records, structuring them, and uploading them to the family’s personal timeline.

Click here for additional information and enrollment.


ASMD Accelerate Study Webinar

04/22/2021

03/25/2021 jjb

Closed Surveys

Niemann-Pick Type C1 Disease Interview Research Study

Are you currently caring for someone with Niemann-Pick Type C1 Disease (NPC1)? Interested in participating in an online (via Zoom) or telephone interview?

  • Worldwide Clinical Trials is looking for approximately 10-15 individuals who are currently caring for someone with NPC1 in the US, and who are at least 18 years of age.
  • If you qualify for the study, you will be asked to participate in one Zoom or telephone interview.
  • Each interview will last no more than 60 minutes. During the interview, you will be asked questions about symptoms and impacts the person you are caring for may have experienced as a result of their condition. You will also be asked to complete a new questionnaire and provide feedback on the questionnaire during the interview.
  • The interview will be conducted by a member of the project team from Worldwide Clinical Trials, a research organization, with experience in this type of research.
  • Each interview will be scheduled at a time that is convenient for you including during the day, during the evening or on weekends.
  • After you complete the interview, you will receive a $20 in the form of a pre-paid debit card.
  • The study has received IRB approval.

Please email study coordinator Jessica Riley, at Jessica.Riley@cchmc.org to learn more about the study.

Version 1.0 29SEP2020


NPC Therapy Accelerator Survey

Families or caregivers of persons with Niemann-pick Type C disease are invited to take a new survey that is designed to collect information about your priorities and preferences for treating Niemann-Pick Type C.

It is organized by the NPC Therapy Accelerator and in the survey, you will read about hypothetical treatments for Niemann-Pick Type C. Some parts of the potential treatments described in this survey are based on real and investigational treatments and some are hypothetical. The information collected here is important to help the U.S. Food and Drug Administration (FDA) understand your thoughts and feelings about potential treatments for NPC.

This survey is intended for parents and current and past caregivers of those with Niemann-Pick Type C. Those who have multiple children with Niemann-Pick Type C should complete one survey for each child and only one survey should be submitted for each child (e.g., two parents should not complete the survey separately on behalf of the same child). For parents/caregivers of older children or adults with Niemann-Pick Type C who are involved in their treatment decisions, you may consult with your child when responding.

The survey will remain open until Monday, December 7th. Your participation in this survey is optional and if you start the survey, you can stop at any time. The survey takes about 30 minutes.

11/06/2020 jjb


Niemann-Pick Type C Survey Opportunity

Pillar Patient Advocates is seeking 5 caregivers and 2 adult patients with a diagnosis of Niemann-Pick disease Type C to participate in a 30-minute market research interview. During the interview, you will share your experience related to NPC while also responding to an online survey. In appreciation, $75 will be paid to participants.

If you are interested in learning more about the study and to see if you qualify, please complete the registration available through this link: research.net/r/FYPHHRD. For transparency, Pillar Patient Advocates is working with BioVid Corporation to ensure your information is anonymous.


NNPDF Health Insurance Survey:

Understanding Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick disease Through the Eyes of Patients and Families

NNPDF is conducting a survey of the U.S, Niemann-Pick disease community to further understand the patient and healthcare provider experience in Niemann-Pick as it relates to health insurance knowledge, coverage, as well as the challenges experienced by patients and their families pertaining to access of medications, services and devices.

Who can participate? Niemann-Pick patients 18 years of age and older or their parents. This is not the full list of eligibility criteria. Find out if you qualify and get more information on this study by visiting Understanding Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick disease Through the Eyes of Patients and Families.

A $100 honorarium is available to participants.

Why is this important? This important survey will identify health insurance usage, access, and barriers across the country. The survey will also allow us to better understand where the community may need more tools or assistance in understanding the complexities of health insurance. The data gathered from this national survey will provide a credible quantitative platform for NNPDF when speaking with legislators, key opinion leaders, and media about access to medications, pricing, economic impact, or legislative reform. This is the most comprehensive survey of the Niemann-Pick health insurance landscape to be conducted in the U.S.

Learn more here.

Question? Contact Joslyn Crowe, Executive Director.

07/01/2020 jjb


NNPDF, APRMF, NPCanada and Firefly Fund Seek Your Participation in a first-of-its-kind Newborn Screening Research Study – Please sign up to participate HERE by Tuesday September 15th, 2020.

NPC community foundations in the US and Canada are partnering with RDMD to create a research database that will power multiple NPC research studies and we need YOUR help to get the first important sibling study underway. This first research project with RDMD, referred to as the sibling study, will focus on the question of whether early diagnosis and intervention for NPC result in better health outcomes and quality of life for patients. Results from this study may be used to seek a nomination for NPC to be added to newborn screening lists across the country, including the federal Recommended Uniform Screening Panel (RUSP).

We need NPC caregivers in the US and Canada that are willing to sign-on, sign-up, register and consent to research for this important study. It will take you less than ten minutes and all data shared with researchers is de-identified, or anonymized. No identifying information is shared.

Act Now: Signing up to participate by Tuesday September 15, 2020, is the first important step. The sign-up process is EASY and fast. One parent can sign-up multiple children under his/her account. Go to: rdmd.com/NPC

Important Notes:

  • Presently, the research can only include patients living in the US and Canada.
  • Caregivers must consent to research, complete the HIPAA waiver and list at least one facility where the patient has received care.
  • Patients own their data and parents will have access to their child/children’s records in a secure portal.
  • There is NO cost to participate.

Questions? Contact Joslyn Crowe, NNPDF Executive Director

08/26/2020 jjb


ASMD Survey Opportunity

RTI Health Solutions is recruiting certain types of patients with Niemann-Pick ASMD, type A/B or type B. Click here to learn more about this survey, or to find out if you are eligible contact Andrew Fox at RTI Health Solutions at 919-541-1274 or afox@rti.org.

06/24/2020 jjb


ASMD Survey Opportunity

Help Needed for Niemann Pick Disease Type A & B (NPD A & B) / ASMD Research

Interested in sharing your experience as a patient or caregiver for a family member with NPD A or B?

  • The interview will be conducted on the phone at a time convenient for you and will take ~60 minutes
  • During the interview, we would like to discuss your story as a patient or caregiver: your experience with doctors, your diagnosis, your day-to-day life, and your hopes for future therapies
  • You will be compensated for your time, and your participation is confidential and anonymous.
  • Note: this study is only for patients and caregivers not currently participating in a clinical trial.

Ready to participate?

To learn more about how you can participate in this research, contact Eli Leavitt: eleavitt@trinitylifesciences.com or 1-781-577-6392.

Your responses will directly help a healthcare company make more informed decisions relating to the treatment of NPD A & B.

05/26/2020 jjb


NPC Survey Opportunity

APRMF, NNPDF, and all the partner organizations are pleased to invite you to take part in a critical survey NPC PFDD Benefit/Risk Survey for Niemann-Pick Type C (NPC) caregivers on priorities for treating the disease, including your views on benefits and risks of future treatments. This survey is an addendum to the pre-meeting survey many members of the community completed prior to the NPC Patient-Focused Drug Development (PFDD) meeting in March of this year. We hope you will take the opportunity to complete this survey by July 29, 2019.

As many of you know, questions around what risks families living with NPC would be willing to take in order support therapy development were a significant discussion topic during the March meeting with the FDA. This survey is designed to answer some of these questions and to inform regulators working on these products now and into the future. Data from this survey, along with data from the pre-meeting survey and content from the PFDD meeting, will inform a Voice of the Patient report on NPC. The report also will help the FDA and researchers better understand the specific priorities of the NPC community, with the ultimate goal of more and better therapies for the disease becoming available. For more information on the NPC PFDD initiative, visit this website.

We hope that you will take approximately 30 minutes to complete this survey at (survey closed). This is an important step as we work together to beat NPC! If you have any questions, please don’t hesitate to reach out.

07/26/2019 jjb


ASMD Survey Opportunity

Name of Survey/Market Research Opportunity: Qualitative study to better understand caregiver’s burden of ASMD (Niemann-Pick disease type B or A/B)

Start Date and Deadline for Participation: May 1 – August 30, 2019

Criteria (i.e. age, Niemann-Pick disease type, geographic location, other): Parents or caregivers of patients diagnosed with chronic forms of ASMD (NPD B or A/B); age 18 or older; residing in the US or UK; ASMD patients can be of any age, but are not in olipudase clinical trials

Number of participants required: 20 parents/caregivers (10 in the US and 10 in the UK)

Location of Survey/Market Research Opportunity: Phone interviews

Who is conducting the survey if not the sponsor? Sanofi Genzyme

Summary of opportunity: Limited data available on ASMD regarding humanistic and socio-economic burden of ASMD has on caregivers and families. Exploring the experiences of caregivers will fill this gap.

Community Contact/How to Participate: Milenka Jean-Baptiste | Email: ASMDcaregiverstudy.SM@ppdi.com
Phone: (USA) 800-805-7314 | (UK) 8000291830
Interested potential participants should contact Evidera staff to complete the screening process to determine their eligibility to enroll in the study at afox@rti.org

Anticipated high level summary date: September 2019

07/24/2019 jjb


NPC Survey Opportunity

Name of Survey/Market Research Opportunity: Qualitative Analysis of Niemann-Pick Type C 5-Domain NPCCSS

Start Date and Deadline for Participation: Currently open until recruitment is met. Expected September 2019

Criteria: Caregivers of children (6 months or older) or adults with Niemann-Pick Type C; or adults diagnosed with NPC. All participants completing the survey (adult patient or caregiver) must be age 18 or older. U.S. participants only.

Number of participants required: Approximately 36 participants for a web survey and approximately 18 participants for a telephone interview.

Location of Survey/Market Research Opportunity: Online survey and telephone interview with a written pre-interview timeline activity to be completed at participant’s convenience.

Who is conducting the survey? Clinical Outcomes Solutions

Sponsored By: Orphazyme

Summary of opportunity: The purpose of this research is to better understand, from patients and caregivers of patients with NPC, what their path to diagnosis was like, what major health-related events have happened since diagnosis, what their current experience of NPC symptoms is, which symptoms are most important to them, and how these symptoms impact their lives.

What does the study involve? The study has two parts. First, participants will complete a 45-minute web survey. Then, some participants will be invited to further take part in a follow-up 60-minute telephone interview. Phone interview participants will also be asked to complete a pre-interview timeline activity that maps out their experiences before and after diagnosis.

Eligible participants will be given a $45 e-voucher in the form of a Visa/Amazon gift card for participating in the web survey. Participants selected to participate in the telephone interview will be given an additional $150 Visa/Amazon gift card upon completion of the interview.

This research is confidential. Any contact information you provide will only be used for the purposes of compensation and scheduling the phone interview if you take part.

How to Participate: To receive a link to the web survey, or to find out more about this study, please contact Bridget Iwamuro by email at NPCSurvey@clinoutsolutions.com or by phone at +1 (312) 874-1868.

This research study will be available to a limited number of participants. Please respond as soon as possible if you are interested in participating. Thank you!


NPC Survey Opportunity

Name of Survey/Market Research Opportunity: Oral Medication Administration Survey

Start Date and Deadline for Participation: April 15 – 19, 2019

Criteria (i.e. age, Niemann-Pick disease type, geographic location, other): Any patient living with Niemann-Pick type C or their primary caregiver.

Number of participants required: N/A

Location of Survey/Market Research Opportunity: Online

Sponsored/Funded By: Orphazyme

Summary of opportunity: Opportunity for people living with NPC or their caregivers to share their preferences for soft foods/liquids used to administer oral medications.

Community Contact: Regan Sherman res@orphazyme.com

How to Participate: surveymonkey.com/r/orphazymesurvey

Study invitation for Niemann-Pick disease patients/caregivers:

Orphazyme is working to create a new therapeutic approach to treat Niemann-Pick disease Type C. The name of the active substance Orphazyme is investigating is called arimoclomol citrate, which is administered orally via hard capsules three times a day (morning, noon and evening). As with many active substances, the taste of arimoclomol citrate can be perceived as bitter. Capsules may be administered in two ways:

  • Swallowed whole with a suitable amount of liquid
  • Capsules may be opened and the powder dispersed into a liquid or soft food

Regulatory guidelines require that Orphazyme demonstrates the appropriate compatibility of arimoclomol in various liquids and soft foods. Therefore, Orphazyme would like your opinion on which liquids and soft foods you may prefer for the administration of oral medicinal products, to ensure that information provided to regulatory authorities is representative of what NPC patients and families are most likely to use. Please take a few moments to share your opinion via an online survey.

The time frame for this study is limited so please respond today if you are interested in participating. Thank you!


ASMD Survey Opportunity

Name of Survey/Market Research Opportunity: Stated-Preference Survey to Measure the Preferences of Patients and Parents for Control of Outcomes and Symptoms of Acid Sphingomyelinase Deficiency (ASMD, also known as Niemann-Pick disease type B)

Start Date and Deadline for Participation: March 20-June 30, 2019

Criteria (i.e. age, Niemann-Pick disease type, geographic location, other):

For adult patients:

  • 18 years or older
  • Self-reported physician-diagnosis of chronic forms of ASMD (NPD type B or type A/B)
  • Not part of clinical trial for ASMD
  • Read and understand English
  • Resident of the US or the UK
  • Provide informed consent

For parents of patients:

  • 18 years or older
  • Have a child of any age with self-reported physician-diagnosis of chronic forms of ASMD (NPD type B or type A/B)
  • Child is not part of a clinical trial for ASMD
  • Read and understand English
  • Resident of the US or the UK
  • Provide informed consent

Number of participants required: Minimum 20. No maximum.

Location of Survey/Market Research Opportunity: Online

Who is conducting the survey if not the sponsor? RTI Health Solutions

Summary of opportunity: To better understand and quantify the burden of different ASMD-related symptoms and patient’s preferences for treatment outcomes/symptom control.

Community Contact/How to Participate: Andrew Fox afox@rti.org

Anticipated high level summary date: October 2019

3/19/2019 jjb


NPC Survey Opportunity

Start Date: 1/8/2019        End Date: 2/5/2019

Description of desired participants for study
Parents or caregivers of children diagnosed and currently living with Niemann-Pick disease Type C (NPD-C or NPC) in the United States.

Study invitation for Niemann-Pick disease patients/caregivers
BioVid (www.biovid.com), an independent medical marketing research firm, is seeking the opinions of parents or caregivers of children living with Niemann-Pick disease Type C (NPD-C or NPC) regarding current treatments and expectations of future treatments for NPC.

This study is a 15-minute internet survey. All qualified respondents will be paid $50 for their time and efforts. This survey is strictly confidential. Your name and address are only used to process payment.

Interested parents or caregivers can click on the below link to answer a few brief questions to determine qualification. If qualified, the survey will begin immediately after the brief qualifying questions and should take no longer than 15-minutes to complete.

Should you have any questions or concerns, please feel free to contact Ginny Rillera directly via email at grillera@biovid.com or by phone at (215) 458-9319.

This survey will be available for a limited number of participants. To ensure participation, please click on the link above before the end of the month. Thank you!

1/15/2019 jjb


ASMD Study Opportunity

Description of desired participants for study
Caregivers of patients, diagnosed with Acid Sphingomyelinase Deficiency (ASMD) / Niemann-Pick Type B or Intermediate Type A/B (respondents must be age 18+).

Study invitation for Niemann-Pick disease patients/caregivers
Fulcrum Research Group, an independent market research firm, is conducting a study to better understand the experiences of patients with Niemann-Pick disease / Acid Sphingomyelinase Deficiency (ASMD). Specifically, they are looking to speak with caregivers of patients diagnosed with Niemann-Pick disease Type B or Intermediate Type A/B.

The research will be confidential, and insights will be used for research purposes only.

The study has two parts: a brief online survey and a telephone interview. If you qualify to participate, you will be asked to complete a 15-minute survey as Part I. For Part II, you will be interviewed over the phone for 60 minutes about your experience with Niemann-Pick disease / ASMD (note: you will need access to a computer with internet during this time).

If you qualify for and participate in this research, you will receive a token of appreciation for your time and effort.

To find out more details about the study and to see if you qualify, please contact Kim Slusher via email at kim@pinpointpatientrecruiting.com or by phone at 980-677-1404.

The time frame for this research study is limited so please respond soon if you are interested in participating. Thank you!

2/1/2019 jjb


Supporting one another. Supporting our community.