June 4, 2018
President Trump on Wednesday signed into law the Right-to-Try Act establishing a pathway for terminally ill patients to use non-FDA-approved drugs, calling the legislation “great for the people” and a “fundamental freedom,” but it was a castoff remark he made about lower drug prices that had everyone talking. Referring to the legislation, Trump thanked HHS Secretary Alex Azar and FDA Commissioner Scott Gottlieb at the signing ceremony and also credited Gottlieb for his efforts to shorten the development and approval process. “You have a lot of things in the wings that frankly if you moved them up, a lot of people would have a great shot,” he said, adding that “hundreds of thousands of lives” would be saved by the new legislation. The law allows terminally ill patients who have exhausted all other options to try experimental treatments that have not yet received FDA approval. Eligible treatments must have completed a Phase I trial, be in the process of active development, and be the subject of an NDA, BLA or IND. The new law has its critics among industry and patient advocacy groups with reservations about its potential impact on patient safety. The Association of Clinical Research Organizations came out strongly against the bill’s passage, calling the legislation “deeply flawed.” ACRO argued the bill does not go far enough to protect patients and “compromises the clinical trial process [and] undermines the FDA’s authority to assess safety and effectiveness.” After several iterations, the version of the bill that finally passed is “devoid of all patient protections” and “does not set a standard for informed consent, has a much broader definition of eligibility and contains vastly weaker reporting requirements,” according to the National Organization for Rare Disorders. “Thus, not only will this legislation be ineffective, as all Right to Try laws are, it will also present a danger to the many patients we represent.” In a statement following the signing ceremony, Gottlieb said the FDA recognizes “the important balance between making sure patients have the assurances Congress intends, while enabling timely access to promising treatments in these devastating circumstances.” He said the agency will “implement this new law consistent with these longstanding values.” During the ceremony, Trump also made a side remark that unnamed major drugmakers would announce “voluntary massive drops in prices” in “two weeks” adding that “for the first time ever in this country, there will be a major drop in the cost of prescription drugs.” At the White House daily press briefing Wednesday, Press Secretary Sarah Sanders said the White House could not provide further details, “but we do expect some specific policy pieces to come out on that soon.” Industry groups appeared puzzled as to what the president was referencing, although the Association for Accessible Medicines responded to the remark in a tweet, saying it looks forward to continue working to reduce prices and out-of-pocket costs. PhRMA declined to comment and BIO did not respond to a comment request.
Age, Kidney Health May Be Keeping Women from Heart Failure Trials, Researcher Finds
Age and relatively poor kidney health may be keeping women from enrolling in heart failure clinical trials, a Swedish researcher says. Just 21 percent of patients enrolled in the recent PARADIGM-HF were women and researchers at Umeå University are blaming the trials’ target doses of angiotensin-converting-enzyme (ACE) inhibitors and angiotensin receptor blockers (ARB), which the researchers say “disproportionately excluded women.” “In the community-based heart failure population, just 26 percent of women with heart failure and a reduced ejection fraction could reach the target dose compared to 43 percent of the men,” lead researcher Helena Norberg wrote for her colleagues in a study published in the journal of the European Society of Cardiology on Monday. That, in turn, may be due to the facts that women in the subject population tended to be significantly older (81 years old for women versus 75 years old for men) and have worse kidney function than the men, Norberg said. “Future trials in heart failure should use achievement of maximum tolerated doses, rather than fixed target doses, as entry criteria to ensure that women are more represented,” Norberg wrote.
Researchers Struggle to Make Informed Consent Forms Understandable to Patients, Study Finds
Researchers are still struggling to come up with informed consent paperwork that is accessible to their patients, a team of Mexican researchers has found. The team examined informed consent forms for industry-sponsored multinational clinical trials for arthritis over a 17-year period and then tested patients’ perceptions about the process they were enrolling in. Researchers concluded that all of the informed consent forms were anywhere from “somewhat difficult to read” or “average” in difficulty. The mean “readability” score for all the forms was 57, “without significant changes from 1999 to 2016,” lead author Hector de la Mora-Molina wrote for the team. There still remains a gap between patients’ claimed understanding of informed consent and their knowledge of basic ideas. “The disparity between the readability” of informed consent forms and patients’ health literacy “continues even after decades of attempts of regulatory agencies and numerous published suggestions,” the researchers concluded. The study was published in Seminars in Arthritis & Rheumatism.