MANAGEMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD)- RESULTS FROM A GLOBAL SURVEY OF HEALTHCARE PROVIDERS FROM NINE COUNTRIES
Author(s)
Narayanan S, Olesen D
PTC Therapeutics, Inc., South Plainfield, NJ, USA
Presentation Documents
OBJECTIVES: Assess the physician perception and dynamics of management of patients with DMD. METHODS: A quantitative survey was implemented in May 2017 in the U.S, Europe5(Germany/France/Italy/Spain/UK), Turkey, Colombia, Brazil among specialists treating a minimum threshold of DMD patients; physicians must have been in practice between 2-35yrs and spent >=25% of time in direct patient care. Fifty-minute survey (in local languages) captured physicians/site characteristics, perceptions of early intervention and disease management, as well as the dynamics of patient diagnostics, genetic testing, specific DMD treatment attributes and stakeholder interactions. Descriptive statistics were computed. RESULTS: Preliminary analysis included 170 physicians (pediatric neurologist: 51.8%; neuromuscular specialist: 28.2%; adult neurologist: 12.4%; US: 24.7%, Europe5: 45.3%, Turkey: 7.6%, Colombia: 8.8%, Brazil: 13.5%) completing the survey. Mean number of DMD patients in practice: 43.0, mean number of patients personally managed within previous12mo: 26.9; 80.6% & 80.0% physicians were affiliated with muscle centers and hospitals, respectively. Physicians used diverse pathways to treat DMD patients (not mutually-exclusive): Physical therapy/rehabilitation (92.9%), pulmonary/respiratory care (92.4%), orthopedic/orthotics (86.5%), prescription medications (84.1%), symptom management/supportive care (82.9%), cardiac care (81.8%), scoliosis management (80.0%), nutritionals/vitamins (76.5%), psychological (74.1%), palliative care (48.2%). Key physician perceptions of DMD management (rated >=5 on a likert scale of 1(strongly-disagree)-7(strongly-agree)): preserving functionality at all stages of disease is key (91.2%), delaying loss of ambulation is a meaningful outcome (90.6%), pressing need for earlier detection of DMD (87.7%) and earlier start of treatment (86.5%), early diagnosis/treatment could meaningfully delay irreversible muscle damage (84.5%); 84.1% discussed genetic-test results with patients; 68.8%, 57.7% and42.9% reported using timed function tests, 6-minute walk test and north star ambulatory assessment to measure disease progression, respectively. CONCLUSIONS: This cohort of physicians highlighted the diversity in DMD disease management. Majority of physicians highlighted the importance of early DMD diagnosis/treatment, preserving functionality and delay loss of ambulation.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PSY108
Topic
Health Service Delivery & Process of Care
Topic Subcategory
Prescribing Behavior, Treatment Patterns and Guidelines
Disease
Neurological Disorders, Rare and Orphan Diseases