Monthly Archives: December 2016

New Law Could Speed FDA Approvals: Will Patients Still Be Safe?

This past week, the US House of Representatives passed legislation that has become known as the CURES ACT. This is thought to be the largest and most powerful healthcare related law since the passage of the Affordable Care Act during Obama’s first term. The Cures act provides for a large increase in funds for boosting biomedical research and also takes aim at speeding up drug and medical device approvals through the Federal Drug Administration (FDA). As expected, other items are also placed inside this bill such as funding for Opioid abuse as well as a program to better treat mental illness—making it very likely that President Obama will sign the law once it is approved by the Senate.

Many medical researchers have worried about what may happen to important and essential research funding for the National Institutes of Health (NIH) under a Trump administration and this law provides for more that 4 billion dollars for the NIH over the next decade.

How Might This Law Affect the FDA Process?

Currently drug and device makers must present data obtained from rigorous randomized controlled clinical trials (RCTs). RCTs provide the best type of evidence for the safety and efficacy of a drug or device and can Under the new law, companies would be able to use “surrogate endpoints” rather than hard outcomes to obtain approvals—this would make it far less expensive and time consuming for pharmaceutical and device companies to move new products through the FDA at a much quicker pace. Company controlled registry data would be accepted as evidence and RTCs would not always be required.

What Could This Mean for HealthCare Consumers?

Proponents of the legislation argue that this will be a huge step forward in public health. By providing such a large amount of funding for biomedical research as well as funding for work on opioid addiction and mental health treatments, that are likely to provide better treatment options for millions of Americans. In addition, simplifying the FDA approval process is likely to spur innovation—companies will be able to devote more time to research and development and patients will be able to reap the benefits of new therapies much faster. The FDA process is currently weighed down with lots of rules and regulations—providing a more clear path to approval may encourage smaller innovators and entrepreneurs to produce new products.

Opponents to the law argue that removing the requirement for robust data with hard endpoints to support the safety and efficacy of new drugs and devices may place patients at risk. Consumer and public safety groups worry that the law may open the door to the approval of unsafe drug and device approvals. Using observational data and patient feedback rather than RTCs may allow unsafe drugs and devices to enter the market; Companies may be incentivized to rush drugs and devices to the market with only anecdotal evidence in support of their efficacy or safety. Some experts argue that the law will de-legitimize the FDA and lead to patient harm.

The CURES Act—A Physician Perspective

As a practicing cardiologist, I rely on robust data to help me decide how best to treat my patients every single day. I trained at Duke University where we have the largest and most prolific center for the conduct of cardiovascular clinical trials in the world—the Duke Clinical Research Institute (DCRI). I was taught to only use a drug or device that had been rigorously evaluated by RCTs and that all other data was insufficient to determine safety and efficacy. Observational studies can provide correlation but not establish a true “cause and effect” relationship. However, it is often frustrating when we hear about a potentially practice-changing drug or device that is available in Europe but NOT available in the US due to a protracted FDA process. The FDA is currently not adequately funded to handle the amount of regulatory and administrative work they must accomplish in order to efficiently evaluate drugs and devices in a way that still protects patients from harm. The Cures act does provide more funding to the FDA but it is far short of what the agency really needs. I do think that they agency can still ensure patient safety by shifting the way they look a drugs and devices—more needs to be done in the area of pharmacovigilence or post market drug and device monitoring. Possible novel mechanisms for this monitoring could be “social listening” where artificial intelligence (AI) programs are used to sift through social media posts by patients in order to identify possible adverse drug or device events that may warrant investigation. Currently, the agency relies on self-reporting of these events—leading to under-reporting and duplication of reports—making the data relatively unreliable.

We must remember that the pharmaceutical and device industries are ‘for profit” and that they pursue FDA approvals in order to sell their products—as with most businesses, when they can cut expenses (such as in research and development costs) they will do so. This law will certainly allow for more innovation at a lower cost but I worry that this law will do little to change the cost of drugs (think Mylan and EpiPen). Unless there is some provision for more Pharmaceutical regulation I suspect that this law will have little effect on drug costs—it will help the bottom line of industry executives.

The provision of the Cure act that ensures funding of the NIH is incredibly important to researchers in health and science at academic institutions throughout the US. Much of the most important research that is conducted by medical schools and universities will be funded by the NIH and directed at finding cures for cancer and heart disease (just to name a few) through cutting edge genetic medicine. This funding is essential to advance science and develop new and exciting treatments for chronic disease—without it, many projects will never come to fruition and many bright minds may leave institutions that are focused on research in science and medicine.

Ultimately, I am pleased with the Cures act. I think that it is important legislation that will help bring treatments to patients faster—saving lives and improving quality of life. We must, however, continue to balance promoting innovation with patient safety. We must also ensure that, as we move forward, that attention is given to monitoring the practices of the drug and medical device industries—we cannot allow price gauging and we must ensure that all patients have fair and affordable access to the latest and most effective therapies.