New Study Quantifies Duchenne Caregivers' Assessment of Risk vs Reward

Caregivers give thoughts on attributes they are looking for in DMD treatment.

Caregivers give thoughts on attributes they are looking for in DMD treatment.

It is often suggested that patients of rare disease view the risk/ benefit ratio of a treatment differently than those with more common ailments. If a rare disease is fatal, odds are rare disease patients and caregivers’ will be more willing to take chances on a drug that they feel will improve their life or quality of life. While that may be true, it is often difficult for regulatory agencies to justify an increased risk for an uncertain benefit if there is no data about the patients/caregivers’ attitudes to justify that decision.

That is starting to change. The Duchenne muscular dystrophy (DMD) community, led by Parent Project Muscular Dystrophy (PPMD), recently published a survey that quantified caregivers’ thoughts about various attributes they are looking at in a treatment for DMD. The results are very interesting and should be considered a model for other patient advocacy groups follow.

The survey stemmed after lengthy discussions and meetings with caregivers, advocates, academic researchers, and pharmaceutical drug developers to determine what attributes are most important to the interested parties. The attributes they came up with are listed below.


Effect on muscle function

  • Stops the progression of weakness
  • Slows the progression of weakness
  • Done not change the progression of weakness

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