Compassionate Use

Compassionate Use of Unapproved Therapies

The National MPS Society has been contacted by several of our members concerning compassionate use of unapproved therapies for MPS conditions. In response, we would like to share the following thoughts.

The National MPS Society is pleased to have FDA approved therapies for three MPS diseases and a favorable FDA panel recommendation for a fourth that we hope will be approved in early 2014. These therapies, approved during the past ten years, are the result of the combined efforts of dedicated researchers, investigators, pharmaceutical companies, and most importantly, MPS families. We are also pleased that there are currently several other clinical trials in various stages that may result in FDA approved treatments for those and other MPS and related diseases in the future.

To receive FDA approval, a therapy must meet complex regulatory hurdles demonstrating both safety and efficacy that necessitates the formulation of precise clinical endpoints and inclusion/exclusion criteria for participants. We are aware of many heartbreaking situations in which individual MPS patients cannot participate in the clinical trials due to the inclusion/exclusion criteria. Once a therapy has begun the clinical trial process but has not yet received regulatory approval, the FDA regulations allow for compassionate use outside of the clinical trial under certain conditions. We again are aware of heartbreaking situations in which individual MPS patients cannot access unapproved therapies under the compassionate use provisions.

We as a Society sympathize with our families who cannot participate in clinical trials for their disease because of the inclusion/exclusion criteria, who cannot access compassionate use therapies for their disease because of the conditions, and who are still waiting for a clinical trial to start for their specific MPS disease. We have specific efforts underway to address these situations, including stopping the off shoring of clinical trials, easing the regulatory burden required for approval of rare diseases, and developing treatments for the remaining diseases. The Society will NOT rest until there are treatments and cures for ALL MPS and related diseases.

In response to our members who have contacted us about a specific request for compassionate use access to Shire’s intrathecal enzyme replacement therapy that has been publicized through social media, we contacted Shire and received the following response that we would like to share with you.