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MASAC Recommendation # 127


The following recommendation was approved by the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation on November 17, 2001, and adopted by the NHF Board of Directors on November 18, 2001.

The National Hemophilia Foundation has recognized the importance of exploring the use of gene transfer to cure bleeding disorders. NHF recognizes that a combination of animal and human testing will provide significant information to modify and optimize delivery systems that will lead to long-term cures of bleeding disorders. This clinical research is an iterative process, meaning it will change as new data are obtained and integrated into new ideas and new solutions to overcome existing hurdles. We have encouraged animal testing when research questions on safety and efficacy of new vectors can be evaluated. At a certain point, however, human clinical research is required to answer questions about how the treatment works in patients and to look for human-specific complications.

As clinical research data are obtained, if unexpected findings are observed, we would ask the study sponsors, the FDA, and the National Institutes of Health Recombinant DNA Advisory Committee (RAC) to consider the risks to the patient and to others. If those risks can be quantified and mitigated, trials should proceed with appropriate risk/benefit analysis and risk mitigation. This case-by-case evaluation will permit incremental improvements in safety and efficacy of gene transfer to be developed for the improved treatment of hemophilia and other monogenic disorders.

This material is provided for your general information only. NHF does not give medical advice or engage in the practice of medicine. NHF under no circumstances recommends particular treatment for specific individuals and in all cases recommends that you consult your physician or local treatment center before pursuing any course of treatment.

CHAPTERS: Please feel free to distribute to your members.
PHYSICIANS: Please distribute this information to all providers in your area who treat patients with bleeding disorders.