\n<\/p>\n
The FDA\u2019s recent decision<\/a> to withhold approval of a new skin cancer treatment fell like a hammer on doctors who treat melanoma and patients who saw that the drug had prolonged the lives of a third of the participants in a clinical trial.<\/p>\n \u201cIt was devastating news,\u201d said Trisha Wise-Draper, a dermatologist at the University of Cincinnati who had patients enrolled in the trial.<\/p>\n \u201cThis is life or death for maybe 2,000 patients,\u201d added Eric Whitman, medical director of the Atlantic Health System\u2019s oncology service. A Wall Street Journal editorial<\/a> assailed the ruling, noting that it \u201cwill have a chilling effect on drug development.\u201d<\/p>\n Despite the benefit to some patients, oncologists and pharmaceutical industry analysts say there were legitimate concerns about the treatment, called RP1, that may have led the FDA to reject it in any event. The company, they noted, had ignored repeated FDA suggestions that it change the design of the trial used to seek approval for the medication.<\/p>\n The FDA\u2019s decision would have raised few eyebrows before the current administration took power. But Marty Makary, who took charge as commissioner 13 months ago, altered the agency\u2019s culture and damaged the trust it had built over decades while regulating 20% of U.S. consumer spending, said Steven Grossman, a regulatory consultant and former Health and Human Services official.<\/p>\n \u201cPeople have to speculate about the standards and processes by which the agency makes decisions,\u201d he said. \u201cAnd that uncertainty is bad for everybody \u2014 patients and sponsors and investors.\u201d<\/p>\n Under Makary \u2014 who resigned this week \u2014 senior officials have either suppressed<\/a> or pushed forward<\/a> some drug approvals<\/a> and policies<\/a> at the behest of President Donald Trump or HHS Secretary Robert F. Kennedy Jr., ignoring the advice of agency professionals. In defending his actions, Makary often eschewed the agency\u2019s traditionally measured language about its decisions.<\/p>\n In response to criticism for rejecting the melanoma treatment, for example, Makary accused its manufacturer, Replimune, of \u201ccorruption,\u201d saying it was \u201cengaging in corporate spin\u201d to make the FDA look bad.<\/p>\n \u201cI don\u2019t work for Replimune. I work for the American people,\u201d Makary said in a May 5 interview on CNBC. Kennedy backed him up during a congressional budget hearing in which Kennedy mistakenly claimed that patients in Replimune\u2019s clinical trial had also received chemotherapy.<\/p>\n Makary did not respond to requests for comment.<\/p>\n \u201cAll the norms have been thrown out the window, so we don\u2019t know what underlines an agency decision,\u201d said Paul Kim<\/a>, a former FDA staffer and Senate aide to Sen. Edward Kennedy who\u2019s now a pharmaceutical industry consultant in Boston. \u201cEven when there are legitimate scientific and regulatory reasons why a drug will not be approved, we\u2019re left guessing whether it\u2019s legitimate grounds or just a political play.\u201d<\/p>\n