The U.S. Food and Drug Administration frequently finds itself criticized for taking too long to approve drugs and devices, increasing the cost (and subsequent fundraising pressure) for drug developers and medical device makers to get their products to market. Now it’s facing a new type of criticism over accelerated approval programs in place for more than a decade that risk jeopardizing patient safety.
The article, published in the Journal of the American Medical Association, and cited by Pharmalot highlights three drugs approved by the regulator as examples: Boehringer Ingelheim’s blood thinner drug Pradaxa to reduce stroke risk in people with atrial fibrillation, AstraZeneca’s (NYSE: AZN) thyroid cancer drug Caprelsa, and Novartis’ (NYSE: NVS) multiple sclerosis pill Gilenya. Of the three, Pradaxa comes in for the most criticism.
“within less than a year of approval, a survey showed Pradaxa accounted for more serious adverse drug events reported to the FDA during the second quarter of 2011 than any other regularly monitored drug. Risk of hemorrhage was prominent in older patients – median age of 80 years – a subgroup for whom declining kidney function or other factors may have increased bleeding risk”. As a result, the European Medicines Agency called for closer monitoring of kidney function. according to the authors, Thomas Moore, a senior scientist at the Institute for Safe Medicine Practice, and Curt Furburg, a health sciences professor at the Wake Forest School of Medicine.
Although European regulators asked for label changes, but said the drug’s benefits are still worth the risk for appropriate patients, according to Fierce Pharma. The authors express concern that with the U.S. Senate’s push for an expansion of expedited review, with the new category of “breakthrough drugs” this will become a more contentious issue. Risk and innovation are permanently joined at the hip, just as the authors’ article title, “Safety Risks of Innovation” suggests.
A letter from the FDA sparked by the article and obtained from the FDA by Pharmalot acknowledges as much when it grants accelerated approval for certain drugs.
“The expedited review programs cited by the authors… have been used successfully for many years to help expedite approval of promising new drugs for serious or life-threatening conditions. Priority review and accelerated approval have been in place for nearly two decades, and fast track has been in place for almost 15 years. Importantly, these programs do not change the statutory standard for approval but rather allow some flexibility in meeting that standard. ‘Breakthrough drugs’ represents a new category.
“Making regulatory decisions about drugs always involves uncertainty and risk. FDA works directly with the affected patient populations and treating physicians when considering just how much uncertainty and risk are reasonable to accept. We know, for example, that patients and physicians are willing to accept more risk with a treatment that is effective for serious diseases, particularly if existing treatment options are limited, and we factor this risk tolerance into our decision making.”
Particularly when there’s an unmet need, I think it’s ethical to accelerate drug approval, as long as physicians, pharmacists and the patients are fully informed of all of the treatment’s risks, it should be their collective decision. As with Pradaxa, safety reports will spur the regulator to conduct a safety evaluation.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
It’s commendable for scientists and physicians to share their concerns about the inherent safety risks involved with fast track approval, particularly as the Affordable Care Act increases liability for providers for patient outcomes. It will be interesting to keep an eye on how payers evaluate the drugs in the fast track programs and how flexible they are willing to be in giving plan members access to these drugs.
