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Abstract Details

Willingness to Participate in Natural History Studies amongst Patients with Sporadic Inclusion Body Myositis (sIBM): A Comparison Between the United States and Australia
Neuromuscular and Clinical Neurophysiology (EMG)
P3 - Poster Session 3 (5:30 PM-6:30 PM)
12-003
To gauge patient attitudes toward natural history studies, particularly ones that involve invasive procedures, like muscle biopsies, in sporadic inclusion body myositis across two countries with distinct healthcare models.
Defining natural history of sporadic inclusion body myositis (sIBM) is vital for designing effective clinical trials. Given the recent identification of serological markers in patients with this disease, it is important to understand if natural history and disease behavior is variable based on serological status. We studied sIBM patients in the USA and Australia to assess their attitudes towards natural history studies.
An 8-item survey was developed on Google Forms and disseminated through The Myositis Association for the two regions studied. The items measured satisfaction with current sIBM therapeutics, perception towards natural history studies, and willingness to complete on-site visits and various procedures.
A total of 646 (539 USA, 107 AUS) sIBM patients responded to the survey. Approximately 91% of respondents (89% USA, 93% AUS) were willing to partake in a natural history study with biannual visits involving physical assessments, questionnaires, and serum collections for DNA analyses. The more involved procedures such as the muscle biopsy and DNA testing were, on average, accepted by 75% and 97% of respondents, respectively. Self-pay for study-related expenses (e.g., meals, parking) deterred 26% of all respondents from participating in the proposed study.
Our survey demonstrated an unmet need in sIBM independent of geographic variation. Additionally, the difference in responses received from American and Australian sIBM patients lead us to consider the impact of the combined universal and private insurance platform utilized in Australia on the higher satisfaction with treatment options scale. Regardless, the vast majority of all respondents were willing to participate in a complex natural history studies because they are unsatisfied with current options.
Authors/Disclosures
Veena Mathew
PRESENTER 		No disclosure on file
Ali A. Habib, MD (University of California, Irvine) Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech/Roche. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon/ Amgen. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Kyverna. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Jansen/Johnson & Johnson. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMDSerono/Merck. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunis Biomedical. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman-La Roche. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jansen/Joohnson & Johnson. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from Horizon/Amgen. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Regeneron. The institution of Dr. Habib has received research support from Arcellx. The institution of Dr. Habib has received research support from Novartis. The institution of Dr. Habib has received research support from EMDSerono/Merck. The institution of Dr. Habib has received research support from Cour Pharmaceuticals.
Namita Goyal, MD, FAAN (University of California, Irvine) Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abcuro. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Souffle. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL Behring. Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Amgen. The institution of Dr. Goyal has received research support from Fulcrum. The institution of Dr. Goyal has received research support from Healey Platform. The institution of Dr. Goyal has received research support from Medicinova. The institution of Dr. Goyal has received research support from Transposon. The institution of Dr. Goyal has received research support from Abcuro. The institution of Dr. Goyal has received research support from Calico. The institution of Dr. Goyal has received research support from Clene. The institution of Dr. Goyal has received research support from Janssen. The institution of Dr. Goyal has received research support from PepGen. The institution of Dr. Goyal has received research support from PTC. The institution of Dr. Goyal has received research support from Roche. The institution of Dr. Goyal has received research support from Sanofi. The institution of Dr. Goyal has received research support from Prilenia. The institution of Dr. Goyal has received research support from RAPA. The institution of Dr. Goyal has received research support from Avidity. Dr. Goyal has received publishing royalties from a publication relating to health care.
No disclosure on file
Merrilee Needham, MD Dr. Needham has received personal compensation in the range of $500-$4,999 for serving as a Consultant for teva. Dr. Needham has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for abcuro. Dr. Needham has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for sanofi aventis.
Tahseen Mozaffar, MD, FAAN (University of California Irvine) Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas Gene Therapy. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics. The institution of Dr. Mozaffar has received research support from NIH. The institution of Dr. Mozaffar has received research support from Muscular Dystrophy Association. The institution of Dr. Mozaffar has received research support from Sanofi. The institution of Dr. Mozaffar has received research support from Argenx. The institution of Dr. Mozaffar has received research support from Amicus Therapeutics. The institution of Dr. Mozaffar has received research support from Astellas Gene Therpay. The institution of Dr. Mozaffar has received research support from Cartesian. The institution of Dr. Mozaffar has received research support from Cabaletta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.