As part of our information gathering efforts, we will frequently post surveys for all or various groups of our membership to complete. You must be registered with the NFDF to take part in surveys and to gain access to the results. Please take all the surveys, current and past, that apply to you as promptly as possible to facilitate the best use of the information we receive. Once you have taken a survey, you may review the anonymous results from all participants who took each survey. See the appropraite survey results link under survey link.
Please select the link(s) below to participate in the survey(s) and to view the corresponding survey results when available.
Community response to FDA question about Fabrazyme market exclusivity withdraw
This survey is now closed to enable our response to the FDA on Monday, 13 July.
The consensus of the NFDF Board of Directors, Medical Advisory Board, and members was to recommend the FDA NOT withdraw the Genzyme Corporation's market exclusivity for Fabrazyme for these reasons: 1) all established protocols were being adhered to when the virus in the Allston plant occurred, 2) the shortage is a temporary shortage being well managed by treating physicians and viewed as unfortunate but with understanding by patients, 3) the NFDF community does not want any action taken for this situation that may jeopardize other companies willingness to seek treatments for orphan diseases.
This said, we believe manufacturers have a lawful and moral obligation to provide adequate drug to patients upon receipt of market exclusivity and other incentives provided by the Orphan Drug Act. Drug shortages in cases with unjustifiable circumstances should be subject to exclusivity withdraw.
Thank you for your participation! The more we learn from you, the better our ability to make improvements and to meet the needs of the Fabry disease community.
