The
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dards of safety and efficacy. Provided that such a Ameet Sarpatwari, J.D., Ph.D. back door remains closed, a more user-friendly Jonathan J. Darrow, S.J.D., J.D. system of expanded access as described by Lurie Aaron S. Kesselheim, M.D., J.D. et al. may help reduce pressure for more prob- Brigham and Women’s Hospital lematic measures currently being considered by Boston, MA publication of their article, the authors report no furCongress that would substantially lower the therSince potential conflict of interest. 3 drug-approval standards of the agency. If the 1. Guidance for industry: individual patient expanded access new FDA procedures enable patients with life- applications: Form FDA 3926. Silver Spring, MD: Food and Drug threatening conditions who lack adequate thera- Administration (http://www.fda.gov/downloads/Drugs/Guidance peutic options and who do not qualify for ongo- ComplianceRegulatoryInformation/Guidances/UCM432717.pdf). 2. Quicker access to experimental drugs. New York Times. Febing clinical trials to access potentially beneficial ruary 12, 2015:A28. experimental treatments through expanded- 3. U.S. House of Representatives Energy and Commerce access protocols, it would remove a main ethical Committee. 21st Century Cures Act: discussion draft (http:// energycommerce.house.gov/sites/republicans.energycommerce justification for the excessive acceleration of for- .house.gov/files/114/Analysis/Cures/20150127-Cures-Discussion mal FDA approval of drugs before they can be -Document.pdf). DOI: 10.1056/NEJMc1501823 adequately tested.
PML in a Patient without Severe Lymphocytopenia Receiving Dimethyl Fumarate To the Editor: Fumaric acid esters, which are prescribed for the treatment of psoriasis and multiple sclerosis, are considered to have a favorable risk profile. However, treatment-related progressive multifocal leukoencephalopathy (PML) has been described in association with long-lasting, severe lymphocytopenia (
n e w e ng l a n d j o u r na l
of
m e dic i n e
dards of safety and efficacy. Provided that such a Ameet Sarpatwari, J.D., Ph.D. back door remains closed, a more user-friendly Jonathan J. Darrow, S.J.D., J.D. system of expanded access as described by Lurie Aaron S. Kesselheim, M.D., J.D. et al. may help reduce pressure for more prob- Brigham and Women’s Hospital lematic measures currently being considered by Boston, MA publication of their article, the authors report no furCongress that would substantially lower the therSince potential conflict of interest. 3 drug-approval standards of the agency. If the 1. Guidance for industry: individual patient expanded access new FDA procedures enable patients with life- applications: Form FDA 3926. Silver Spring, MD: Food and Drug threatening conditions who lack adequate thera- Administration (http://www.fda.gov/downloads/Drugs/Guidance peutic options and who do not qualify for ongo- ComplianceRegulatoryInformation/Guidances/UCM432717.pdf). 2. Quicker access to experimental drugs. New York Times. Febing clinical trials to access potentially beneficial ruary 12, 2015:A28. experimental treatments through expanded- 3. U.S. House of Representatives Energy and Commerce access protocols, it would remove a main ethical Committee. 21st Century Cures Act: discussion draft (http:// energycommerce.house.gov/sites/republicans.energycommerce justification for the excessive acceleration of for- .house.gov/files/114/Analysis/Cures/20150127-Cures-Discussion mal FDA approval of drugs before they can be -Document.pdf). DOI: 10.1056/NEJMc1501823 adequately tested.
PML in a Patient without Severe Lymphocytopenia Receiving Dimethyl Fumarate To the Editor: Fumaric acid esters, which are prescribed for the treatment of psoriasis and multiple sclerosis, are considered to have a favorable risk profile. However, treatment-related progressive multifocal leukoencephalopathy (PML) has been described in association with long-lasting, severe lymphocytopenia (
