AARP highlights rising specialty prices as FDA looks to tackle development costs, speed

The price of drugs — particularly specialty drugs — is the perennial issue plaguing both patients and the companies that make them, as the cost of making a drug, estimated at around $2.6 billion with an average time of 10-15 years, will be factored into the cost of a treatment. Recently, though, patient advocates and regulators have begun acknowledging a need for something to be done about this trend.

The AARP Public Policy Institute’s latest report took a look at longitudinal trends in drug pricing, examining changes in costs of specialty medications between 2006 and 2015. Looking at the 101 most commonly used specialty drugs, the institute tracked the percentage by which prices increased over the course of nine years, with 2015 showing the largest annual increase in this period, increasing 9.6%. The average 2015 cost for a single specialty medication used on a chronic basis was $52,486, according to the report — slightly less than the median U.S. household income of $55,775 and more than twice the median $25,150 income of Medicare beneficiaries.

“If recent trends in specialty drug prices and related price increases continue, it will almost undoubtedly become more difficult for patients to access and afford necessary specialty medications,” the report says. “The pricing patterns (price levels and price increases) for specialty drugs will lead to serious problems with respect to access and affordability issues in the future.”

The Food and Drug Administration is emerging this year as also concerned with the trend AARP points to. A key plank of the platform the agency has built out is its Drug Competition Action Plan, which has focused on generics — whose prices AARP notes declined by 19.4% in 2015 as the price of branded drugs used commonly by older patients increased 15.5%. But FDA commissioner Dr. Scott Gottlieb indicated in a recent speech that efforts to bring down drug prices aren’t solely concerned with generics.

At the Regulatory Affairs Professionals Society’s 2017 Regulatory Conference this week, Gottlieb said that drug development is getting costlier and taking longer. This is taking place as Hofstra University notes that between 2005 and 2015, the clinical trial success rate was at 9.6%, compared with 16.4% between 1996 and 1999, and 21.5% between 1983 and 1994.

“We’re on an unsustainable path, where the cost of drug development is growing enormously, as well as the costs of the new medicines,” Gottlieb said. “We need to do something now, to make the entire process less costly and more efficient. Otherwise, we won’t continue to realize the practical benefits of advances in science, in the form of new and better medicines. … Even when we can afford to develop breakthroughs, more people will have a hard time paying for them if we can’t reduce the cost of drug development, and find better ways to capture those savings. The high cost of development also reduces competition.”

Among some of the other issues Gottlieb identified was the frontloading of development costs in the early stages, with late-stage development inflation rising slower than early stages. As a result, Gottlieb said, fewer new ideas get advanced, and drugs that succeed are priced to reflect the high cost of development.

Gottlieb said that the FDA’s Center for Drug Evaluation and Research was working to modernize the Office of New Drugs, and that there would be a Strategic Policy Roadmap detailing the steps being taken. He focused on the agency’s efforts to modernize how the FDA collects clinical information, and the growth of trial designs that eschew the three-stage design for other ways of testing a drug’s efficacy for a combined-phase, or “seamless” trial.

Seamless trials use a long, continuous trial to save time, reduce costs and minimize the number of patients necessary, and Gottlieb noted their use and usefulness among candidates for cancer therapy.

“This approach is well suited to the kinds of drugs that are being developed now, where drugs intervene on common elements found across multiple kinds of disease states,” he said. “At FDA, we’ve identified more than 40 active commercial investigational new drug applications for large first-in-human oncology trials alone that use these seamless strategies.”

In addition to efforts to better invest in computing tools to evaluate clinical trial data, Gottlieb said the FDA also would be working on writing t least 10 disease-specific guidance documents in the coming year.

“We need to do these things to make sure we’re providing an efficient path for the translation of cutting-edge science into practical treatments that are going to benefit patients,” Gottlieb said. “We need to do these things because the rising cost of drug development is unsustainable.”

The Pharmaceutical Research and Manufacturers of America responded to Gottlieb’s comments Tuesday, saying they would work with the agency to explore new trial approaches.

“While the pace of science has evolved considerably, the simple fact is from drug discovery through … FDA approval, developing a new medicine takes 10-15 years and costs an average of $2.6 billion,” PhRMA said. “America’s biopharmaceutical research companies are committed to working with the FDA to explore innovative approaches to drug development and clinical trials aimed at enhancing the efficiency of the development and review processes and accelerating patient access to safe and effective medicines. … These modern approaches to clinical trials should embrace appropriate methodologies that reflect the current state of science, including adaptive study designs, model-informed drug development as well as the use of real-world evidence for regulatory decision-making.”