Over the past few months, the Food and Drug Administration has approved a number of new drugs, including products for obesity and HIV. Consequently, FDA Commissioner Margaret Hamburg recently told scientific advisers the “agency is considering letting makers of such treatments conduct faster clinical trials with a smaller group of patients than now required.
A “special medical use” label could be used, allowing doctors to administer the drugs to patients in most dire need, she said.
“This is an issue of having the right science and data to assess risks and benefits but also a broader societal discussion about risks and benefits that individuals and communities are willing to take on and under what circumstances,” Hamburg said in a recording of a meeting released by FDA, as reported by Businessweek. Moreover, the agency “needs to better account for the needs of patients with deadly or debilitating diseases who may be willing to accept the additional perils of unproven drugs,” Hamburg said.
For example, the two recently approved obesity drugs—Arena Pharmaceuticals Inc. (ARNA)’s Belviq weight-loss pill and Vivus Inc. (VVUS)’s Qsymia—were both rejected by FDA in 2010 because of safety risks that took the companies another two years of research to justify. Under the new “special medical use” pathway Hamburg mentioned, the drugs may have been approved earlier.
The pathway “would allow products to come to market faster but would ensure they were used only in patients where there was an applicable risk-benefit situation would be good,” said Allan Coukell, deputy director of medical programs for the Pew Health Group in Washington, who has been involved in the FDA discussions. “It would be good for developers, for companies and it would be good for public health.”
The FDA first brought the idea up during debate earlier this year over reauthorization of user fee programs that fund new drug and medical-device reviews, said Karen Riley, an FDA spokeswoman. One more program that funds animal drug reviews must be reauthorized next year and would open a window to create the special medical use pathway.
Details are still being worked out, Coukell told Businessweek. How much evidence would be required and how does a company generate the data to expand a limited approval later are still unknown, he said.
The accelerated pathway might be used for drugs to treat infectious diseases, antibiotics to combat drug-resistant bacteria and weight-loss treatments, Hamburg said at the Oct. 3 meeting with advisers without identifying any specific medicines. Drugmakers may have a chance to prove the benefits outweigh the risks, removing the restrictive designation and allowing sales to a broader population, Hamburg said. President Obama’s scientific advisors also recently suggested this kind of approach.
The Pharmaceutical Research and Manufacturers of America (PhRMA), and other drugmakers, wouldn’t comment. When the report from the president’s advisers came out, the organization said it would work with the administration to explore ideas in the report.
Hamburg and FDA’s Future
Hamburg also recently appeared at a meeting hosted by the Center for Strategic and International Studies (CSIS), where she noted that US regulators are increasingly reaching out to partner with their foreign counterparts, transitioning from a domestic agency to one with a global focus. She offered a wide-ranging look at a number of topics she has seen during her four-year tenure including globalization, regulatory science, partnering with global regulatory agencies and protecting US consumers, reported RAPs.
There is speculation that Hamburg will likely remain as FDA Commissioner through 2014 if Obama is re-elected, reported Elsevier BusinessIntelligence. “The reasoning, staffers say, is that 2014 represents the year the Affordable Care Act really kicks into high gear with the eligibility expansions in Medicaid and the creation of state insurance exchanges. The administration wants to ensure all of the provisions are implemented and “survive.” Plus, the administration does not want to go through a high-profile confirmation process in the current partisan environment.”
More than anything else, Hamburg said she was particularly struck by the efforts FDA has made to ramp up its global capabilities in the last four years. “It’s only in the last few years that we’re really developed a whole new strategy where we no longer view ourselves as a domestic agency,” she said. “We believe that we have to be a global agency with a global mission with partnerships within government and industry and the scientific community that are global as well.”
“The major reality is that we live in a globalized world,” Hamburg said. “We have to recognize that our responsibilities extend far beyond our shores,” she added, citing the estimated 80% of drugs originating outside of the US ultimately consumed by US citizens. The globalization of product manufacturing is a powerful driver of FDA's responsibility, Hamburg continued, “And really requires changing our model for our regulatory and public health goals.”
Part of protecting the global supply chain involves working closely with global regulatory partners, and even relying on them to conduct some of the groundwork on behalf of FDA staff. Hamburg said FDA is currently working on developing new ways to share information, regulatory workflow and risk-based approaches with their foreign counterparts. “We need to be able to share the best science, the best experience and expertise to target the highest risk products and to use our resources in ways that will make the greatest difference identifying and addressing the critical vulnerabilities in a product's supply chain,” she said.
“If we can help to raise the level of capacity in these countries, we have less work to do because we can be confident that these products are being manufactured to standards that are roughly equivalent to ours.” She recognized, however, the risk of relying on foreign regulators and the enormous political consequences if the agency fails even once. “If we rely on an inspection done by another country and then there's a problem, everyone's going to say, 'How could you possibly have let that happen?' We do have to be realistic about that.”
Beyond just regulators, Hamburg said has been eager to partner with the life sciences industry to help bring new regulatory capacity to under-resourced foreign regulatory agencies.
“We simply need to have new models, and those models have to build on the notions of partnership, both with other regulatory authorities and with industry to raise standards and share information in new ways. I think that building regulatory capacity, either by helping other nations strengthen their capacity or doing so on a region basis, is absolutely critical.”
FDA is working on several projects with members of the development community, including the World Bank, which Hamburg said is bringing together regulators and industry to find new models to build regulatory capacity. Hamburg also said she hopes to elevate the role of regulatory on the international stage, and in particular at high-profile meetings such as the G8 and G20.
“Frankly, I believe that we need to integrate this thinking about regulatory capacity and the role of product safety into other investments that we as a nation and other nations are making—how we're spending development dollars and how we're using strategic diplomatic resources.”
The chief limiting factor at the moment is funding, said Hamburg. Though FDA is increasingly global, she conceded agency staff is worried about expanding FDA’s mandate without having the staff or funding resources to make sure all its bases are covered. She said that the “circumstances demand” increased funding.