Passing a complicated health bill in the rush of a lame-duck session is always problematic. Passing a 996-page bill that was negotiated behind closed doors and includes provisions that were never voted on before represents the kind of legislative sausage that the world’s greatest deliberative body should reject.
Uncertain Funding
While the bill includes some positive measures, the most important ones – funding for the National Institutes of Health (NIH) and the Opioid bill that previously passed – are not guaranteed in this legislation. Unlike the earlier version of the 21st Century Cures Act that was passed in 2015, the funding is not mandated in the new version. The funding would need to be approved by Congress every year, which would be a challenge given the opposition to this funding that has already been expressed by the Heritage Foundation.
Excessive Spending on Ineffective Cancer Drugs:
A new study, published in a journal of the American Medical Association last week, shows the danger of that kind of change. The study found that most newly approved cancer drugs do not help patients live longer or have a better quality of life. In fact, patients taking the most expensive cancer drug in the study ($170,000 per patient) did not live longer than patients taking placebo and felt significantly worse. And yet all 17 ineffective new cancer drugs are still being sold, contributing to the skyrocketing cost of health insurance and undermining the financial viability of Medicare. An editorial in the same journal points out how this harms patients and our healthcare system.
Harm to Stem Cell Therapies Research:
The Accelerated Approval for Regenerative Advanced Therapies section (Sec. 3033) makes it much easier for experimental stem cell treatments to be approved by the FDA without adequate testing to ensure their safety. Stem cell treatments are promising, but they are still highly unpredictable. Patients have become blind as a result of bones growing in their eyes, and disabled as a result of mucus growing in their spines, for example. Patients will be harmed and the development of effective stem cell research will be delayed if these treatments are sold to desperate patients before they are adequately studied.
Weaker Drug and Device Approval Thresholds:
Several other provisions in the bill would also drastically lower standards for the approval of prescription drugs and devices. For example, it would allow FDA to rely on companies’ summaries of their study results, instead of requiring the FDA to review the data itself, as is current law when companies want to sell drugs for new indications (treatments). It also encourages the FDA to make approval decisions based on “real world evidence” that is not necessarily scientifically sound. The FDA currently reviews and scrutinizes scientific data provided by companies, which is necessary to make sure the benefits outweigh the risks for any approved indication. Providing summaries would also reduce information about the possible risks to particular demographic groups, such as women or patients over 65. The bill also allows off-label promotion of medical products under certain circumstances. This reduces or even eliminates the incentive for companies to conduct scientific research to prove that their products are safe and effective for new indications.
Lower Antibiotic Standards:
Another section of the so-called “Cures” bill would allow antibiotics to be approved based on minimal evidence of safety and effectiveness through a “limited population” approval pathway. The bill would not require that the antibiotics be studied on the target population that the new drugs would be approved for. In other words, it is possible that the antibiotics would not meet the urgent need that they are intended for. Unfortunately, these antibiotics could then be widely advertised and used by patients who are not likely to benefit, and could be seriously harmed by them. In the long run, that would contribute to antibiotic resistance.
Weakening of Health Regulations:
The medical IT section of the bill would deregulate electronic medical records and decision support software. A study by the National Center of Health Research (NCHR) found that these types of health IT devices can cause life-threatening problems when they miscalculate incorrect drug dosages for chemotherapy drugs and other treatments. The Breakthrough Devices section (Sec. 3051) encourages shorter and smaller clinical trials for medical devices. These smaller studies make it impossible to include sufficient numbers of women, men, seniors, and racial and ethnic minorities. Moreover, a recent study of high-risk medical devices found that the median number of participants is currently only 65 patients, which is already too few to adequately evaluate safety and effectiveness for both men and women, let alone for elderly men and women compared to young adults, or for minority populations.