Even in the best of circumstances, the quest to bring the latest medicine from the scientist's lab to the patient's bedside can take several years.
And while that's unlikely to change for most therapies, new legislation passed by Congress could significantly speed that process for the most important treatments.
Late in June, Congress reauthorized the Prescription Drug User Fee Act, a program by which the drug industry helps fund the drug review process at the Food and Drug Administration. Included in that bill was a new provision giving the agency power to construct streamlined approval plans for "breakthrough" therapies, which the law defines as those that would address serious, life-threatening conditions, and which early clinical data suggest would represent a substantial improvement over the current standards of care.
Anthony Dudzinski, chief operating officer at Eatontown-based American CryoStem, said the breakthrough designation could help a number of new treatments reach patients faster.
American CryoStem operates a stem cell and tissue bank primarily serving people who want to keep their own cells for future medical or cosmetic treatment. But the company is beginning work on therapies using a patient's cells to treat wounds and injuries such as diabetic ulcers, bedsores and joint disease.
"We think it is important for the FDA to take a look at this use of one's own cells and one's own cellular material as an alternative to some of the really severe treatments and standards of care that are out there, and consider allowing them to be accelerated," he said.
Dudzinski said using one's own cells can virtually eliminate the risk of rejection that can come with transplants or the implantation of medical devices. The technology is in use elsewhere in the world, he said, but without FDA approval, Americans would have to go overseas to receive such treatments.
American CryoStem isn't alone in its hope that the FDA will more quickly take advantage of new science.
Indeed, scientific advancement was at the heart of the push for the breakthrough designation, according to Jeff Allen, executive director of the Washington, D.C., advocacy group Friends of Cancer Research.
Allen said scientists increasingly are able to target therapies to specific patients. In doing so, they often see major patient improvement earlier in the testing process.
With that progress, however, came new questions about how best to conduct clinical trials for such breakthroughs. For instance, Allen said, in some cases, it may be better to conduct smaller trials, or different types of trials, to confirm a drug's efficacy in the most streamlined way possible. That may solve ethical dilemmas like whether to give a large amount of people a placebo as part of a clinical trial when there's a high likelihood the drug will prove effective in patients given the drug.
The FDA already grants speedier review to some drugs, including cancer and HIV/AIDS drugs. Allen said the new designation will take a more holistic approach, bringing the FDA and drug sponsor together earlier in the process to figure out the most efficient way to test and approve a drug.
"What the breakthrough designation is supposed to do is address more of the experimental process of when you see an early effect in Phase 1, how can you shorten the traditional Phase 1, Phase 2 Phase 3 testing in order to sufficiently designate effectiveness?" he said.
Aside from the benefits to patients, the designation could also mean investment dollars for companies like American CryoStem.
"There is kind of a growing trend, and there has been for some time, where the large investors and pharma companies are coming to the table at later and later stages of research," Dudzinski said. Companies that can fast-track their products using the breakthrough designation would have a much better shot attracting investors and partners, he said.
Debbie Hart, president of the biotechnology trade group BioNJ, said that's critically important.
Hart said many startups continue to face hurdles attracting capital. She said receiving the breakthrough designation would not only let investors see the light at the end of the financial tunnel, it would lend credence to a company's science.
"That's one more feather in a company's cap, to say they might actually be one of the successful ones," Hart said.
Still, while the breakthrough designation is seen as a potential regulatory breakthrough by many, its true effectiveness won't be known for many months.
The FDA has 18 months to issue draft guidelines for the program. Those guidelines will be subject to a two-year public comment period, after which the agency will have another year to finalize the rules.
Allen hopes what emerges from that process is an FDA prepared to take an "all hands on deck" approach when a drug company has a potential breakthrough on its hands.
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