Since the start of the COVID-19 pandemic, Parent Project Muscular Dystrophy (PPMD) has surveyed Duchenne and Becker families to better understand how the patient experience with care, clinical trials, and access to approved therapies has been affected.
Survey aims include:
- Understanding the impact on care, trials, and access during the pandemic
- Informing a range of stakeholders in our community on impact from the patient and caregiver perspective
- Guiding PPMD on which resources to develop for the Duchenne community
- Helping to plan our community for future pandemics (Plan B)
The first survey was released in April 2020, results of that survey are reported here.
This report includes results from our second survey, with data collected from December 2020 through February 2021.
Key findings from the report:
- Most families reported attending in-person care visits during the pandemic (either only in-person visits (22%) or a mix of in-person and telemedicine (46%).
- Just over half (53%) of those surveyed reported not missing any medical testing, however 47% reported some or all medical testing missed due to the pandemic which is a cause for concern due to the need for consistent, regular monitoring of people with Duchenne.
- Overall, families reported positive experiences with telemedicine though some areas of care (physical therapy, gastrointestinal care, respiratory therapy) were not viewed as ideal for telemedicine by those reporting in this survey.
IMPACT ON QUALITY OF LIFE
- Unsurprisingly, findings showed that the pandemic has continued to have a negative impact on overall mental health and quality of life (QOL). Strategies to mitigate overall negative impacts should be explored.
- Respondents reported that delayed medical testing and monitoring with standard of care caused high levels of anxiety for families.
- External stressors related to work, money, school, were further exacerbated by the pandemic for families already dealing with the complexities of managing life.
- 22% of respondents reported that they felt in their view social isolation and/ or inactivity during the pandemic sped up their child’s disease progression.
- The number of people reporting financial impact due to the pandemic in our previous survey (77%) decreased in this survey, however 42% still reported some impact related to their job or income overall which is not insignificant.
CLINICAL TRIALS AND ACCESS TO FDA APPROVED MEDICATIONS
- There were no major issues reported with access to Duchenne FDA approved therapies.
- For those participating in clinical trials, 31% reported missing safety and efficacy assessments during the pandemic. The impact of potential loss of clinical trial data on the overall trial remains to be seen and will need to be evaluated.
- 10% reported that the pandemic had interrupted enrolling in a clinical trial.
We want to thank all the families who participated in the survey; the data will be used to inform future programing and strategy.