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ALGSA: What Matters to You in ALGS Research

This survey will help the ALGSA guide discussions with our families, researchers and other key stakeholders at the 2019 Symposium and Scientific Meeting’s Research Roundtable. We encourage you to participate in this survey even if you are unable to attend the Research Roundtable. Your answers will help direct the future of ALGS research. Thank you for your participation.

If both you and your child have been diagnosed with Alagille Syndrome, please complete the survey two separate times so we may have an accurate record.

There will be a section at the end of the survey for optional comments, thoughts, questions, etc. not covered in the questionnaire.

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* 1. Are you attending the Research Roundtable at the 2019 ALGS Symposium in Cincinnati in June?

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* 2. What is the current age of the Alagille Syndrome patient? (Please complete multiple surveys if there is more than one family member with Alagille Syndrome).

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* 3. What is the sex of the Alagille patient?

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* 4. Where does the Alagille Syndrome patient live?

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* 5. What age did you or your child receive a diagnosis of Alagille Syndrome?

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* 6. Were you or your child misdiagnosed before your Alagille Syndrome diagnosis?

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* 7. If yes, what do you feel could have prevented the misdiagnosis?

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* 8. What was yours or your child’s misdiagnosis?

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* 9. Did you have genetic testing to confirm the diagnosis of Alagille Syndrome?

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* 10. If you did receive diagnosis from genetic testing, which gene is involved in your specific case?

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* 11. Do you feel genetic testing is important for rare disease? Why or why not?

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* 12. Does anyone else  in the family have a clinical or genetic diagnosis of Alagille Syndrome? If yes, please list all that apply.

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* 13. Is anyone else in the family suspected of having Alagille Syndrome but have not been clinically or genetically diagnosed? If yes, please list all that apply.

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* 14. Have you or you child been diagnosed with another disease/condition in addition to ALGS (ex. moyamoya, Tetralogy of Fallot, etc.)

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* 15. Have you ever or do you now participate in a clinical drug trial for Alagille Syndrome?

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* 16. If yes, was the treatment effective? (Please explain below)

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* 17. If yes, was the protocol easy to follow?

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* 18. What were the specific challenges to your family?

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* 19. Is there anything about this clinical trial you would have changed?

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* 20. If you could improve or influence future clinical drug trial experiences, what would you suggest? Please explain.

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* 21. Do you or your child take prescription medications for Alagille Syndrome symptoms?

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* 22. If yes, please list prescription medications here.

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* 23. Do you have medical insurance?

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* 24. Does the cost of these drugs affect your decision regarding usage?

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* 25. Have you ever received an insurance denial for any medication/vitamin/nutritional supplement taken for Alagille Syndrome or related symptoms?

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* 26. Do you feel the current medications taken are effectively treating you/your child’s ALGS symptoms?

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* 27. Do you feel the current medications/treatment is the only treatment currently available?

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* 28. If the side effects of a new drug were more severe than your current treatment but clinical evidence showed it would significantly improve your quality of life, would you take the new drug?

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* 29. Which three of these would most impact your decision to try a new drug?

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* 30. Without considering side effects, which one of these would be the MOST important to you in a future therapy?

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* 31. Have you or your child received a transplant as a result of Alagille Syndrome?

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* 32. If you or your child did receive a transplant, please list the type

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* 33. Have you received more than one transplant of the same organ?

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* 34. Was your physician, or your child’s, knowledgeable about ALGS?

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* 35. Did they take efforts to become educated about Alagille Syndrome?

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* 36. How can the knowledge gap be addressed to better educate the medical community about ALGS?

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* 37. Please list your research priorities in order: (excluding a cure)

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* 38. If you answered "Treatment for other symptom" in Q37, please list the symptom here.

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* 39. If you answered "Other" in Q37, please list the other priorities here.

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* 40. What is your current understanding of Alagille Syndrome research?

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* 41. Do you have a good understanding of the difference between biotechs/pharmaceuticals and other scientists/researchers?

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* 42. Which group do you feel is the most important and why?

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* 43. Do you feel you personally have a voice in Alagille Syndrome research?

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* 44. Ideally how could the ALGSA help get your voice heard?

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* 45. What is misunderstood about Alagille Syndrome research?

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* 46. What questions would you like to ask Alagille Syndrome researchers and scientists? Please be as specific as possible.

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* 47. With whom do you feel the responsibility lies to push forward the research on Alagille Syndrome?

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* 48. Do you feel you can impact scientific research on ALGS?

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* 49. Do you conduct your own personal research to learn more about Alagille Syndrome?

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* 50. Patient Centered Outcomes Research Institute (PCORI) works in Comparative Clinical Effectiveness research. Have you heard of this?

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* 51. PCORI works to ensure patient perspectives are considered during research. Would you like to participate in efforts that would ensure the patient voice is heard?

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* 52. What sources do you turn to for information about Alagille Syndrome?

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* 53. Do you struggle to find answers to your questions through the literature or sources you find?

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* 54. How does Alagille Syndrome negatively affect your daily life? (check all that apply)

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* 55. The Alagille Syndrome Alliance works hard to share the knowledge that ALGS is more than just a liver disease. Please describe the overall impact of ALGS on your daily physical life, your child's life, social impact, financial impact, etc.

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* 56. Would you be interested in working alongside the Alagille Syndrome Alliance to bring the patient voice to research and advocacy initiatives outside of the clinical trial setting?

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* 57. May we contact you if we have additional questions?

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* 58. Is there anything else you would like for us to know related to your ALGS diagnosis, living with the disease or ALGS research?

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