President Donald Trump signed two executive orders Wednesday to improve transparency and fairness in U.S. government agencies and to ensure that they are held accountable. The "Improved Agency Guidance Documents" order requires federal agencies to put guidance documents on easily searchable websites and to seek public input on the most important guidance they issue. It also enables Americans to ask agencies to withdraw guidance they believe is wrong, according to the White House. The second order prohibits agencies from enforcing rules that haven't been publicly disclosed in advance. It also instructs agencies to offer opinion letters when requested, so individuals and businesses that want to comply with the law can learn how to do so.
The Institute for Clinical and Economic Review (ICER) plans to assess the comparative clinical effectiveness and value of New York-based Intercept Pharmaceuticals Inc.'s Ocaliva (obeticholic acid) as a treatment for nonalcoholic steatohepatitis (NASH). Intercept submitted a new drug application with the FDA last month for the breakthrough therapy; an approval decision is expected in the first half of 2020. ICER's evidence report on NASH will be reviewed in May. Comments on the Ocaliva assessment should be submitted by Oct. 25.(See BioWorld, April 12, 2019.)
The FDA will provide more than $4.1 million over the next four years to fund two new research grants for natural history studies in rare diseases. Elizabeth Grubbs, at the University of Texas M.D. Anderson Cancer Center, will receive about $1.7 million for a prospective study in medullary thyroid carcinoma. The study will leverage a multi-institutional registry to characterize disease variables and patient perspectives that inform decisions on initiating chemotherapy and adhering to a chemo regimen. The knowledge gained through the study can be used in the design of clinical trials of emerging therapies for the cancer, the FDA said. Jonathan Soslow, at the Vanderbilt University Medical Center, will be awarded about $2.4 million for a prospective study in cardiac disease in Duchenne muscular dystrophy (DMD). The study will combine genetic differences with imaging and blood biomarkers to identify surrogate biomarkers that predict the risk of cardiac dysfunction in DMD and related diseases. The findings could improve the efficiency of future clinical trials in the diseases by decreasing their size and cost.