FDA Commissioner Says Agency Needs to Fill Data Gaps as It Looks to Increase Access to Targeted Therapies

Gottlieb spoke at the NCCN Policy Summit on the role of data analytics in drug trials

In an effort to make it easier for physicians to access information on increasingly targeted treatments, the FDA will soon make clinical endpoints of drug treatments available online, said FDA Commissioner Scott Gottlieb, MD. Those endpoints, he said, which will include drugs evaluated outside of the traditional randomized trial format, will allow providers to more easily judge what treatments may work for their patients, according to a FierceHealthcare report.

The move is part of the FDA's effort to rethink clinical trial design in the midst of a growing market for targeted drug therapies. But improving access to more data will be crucial to making that transition truly effective, Gottleib said at the National Comprehensive Cancer Network's Policy Summit. 

Data collected by providers and payers—such as which treatments cancer patients have already tried—can fill crucial knowledge gaps when evaluating the efficacy of alternative therapies. Those data can also aid in building value-based contracts for emerging treatments, especially in oncology, and could allow providers to flag patients who may be a good fit for these therapies more quickly, he said. 

Payers, in particular, "sit on a mountain of data" that could be of significant benefit to the FDA as it pursues alternate pathways for clinical drug trials, the article said. The agency pays significant amounts of money to access data from different organizations, some of which will not necessarily accept the results of the FDA's analyses. "I'd ask the broader community, don't just criticize," Gottlieb said. "Put your data where your arguments are. Look for new ways to collaborate around these post-market efforts." 

The agency is making significant investments in its own data infrastructures as well to facilitate the process of sharing clinical endpoints, Gottlieb said. The FDA requested $23 million as part of its 2019 budget to build a real-time analytics framework that can make it easier to study varied trial results. 

Some of these therapies have been tested for patient survival rate, but not all have, Gottlieb said. However, a drug for a patient with terminal cancer that eases his or her symptoms without the downsides of traditional chemotherapy still has significant clinical benefit, he said, thus the need to rethink the goalposts for testing, Fierce reported. 

"We’re trying to balance competing needs: the need for high degrees of statistical certainty, and the need for access to care," Gottlieb said.

The FDA also wants to clarify its regulations so as not to impede the growth of value-based contracting. Payers may not base coverage decisions on the factors the agency uses to approve medications and may instead look for information such as the impact on hospitalization rates. Gottlieb said the FDA wants to make it easier for payers, providers, and drug-makers to have those conversations to increase patients' access to alternative therapies. 

Gottlieb said two major roadblocks could also hinder the growth of value-based models: 

  1. The Department of Health and Human Services Office of Inspector General could block contracts based around outcomes measures, as they could be seen as drug companies inducing payers to cover medications. Gottlieb said the OIG has mostly tackled this issue on a case-by-case basis, and hasn't necessarily issued guidelines to follow.
  2. The Centers for Medicare and Medicaid Services could recalculate Medicare drug prices based on the effectiveness of medications, which could drastically slash what they're willing to pay for drugs that have a very limited effectiveness. 

He said these steps are also part of the agency's ongoing efforts to ease drug pricing concerns by boosting competition in markets. 

The FDA can do its part to increase competition by allowing more products to come to the market, but payers must play a role in creating a more competitive pricing structure, he said.

Source: FierceHealthcare; June 25, 2018.