More flexible regulations on the path for approving potential Alzheimer's disease treatments represent a huge opportunity, according to the US FDA, CROs and research advocates.
Last week, the FDA released draft guidance on the development of drugs to treat early stages of Alzheimer’s that revealed a shift in the agency’s thinking.
For patients with early Alzheimer’s disease, a potential treatment’s “effect on a cognitive measure alone may be sufficient to support approval under the accelerated approval mechanism, with a requirement that the sponsor demonstrate (in Phase IV) that the effect persists over time, or that an effect on global functioning emerges,” FDA spokeswoman Lisa Kubaska told Outsourcing-Pharma.com.
Previously the agency required a potential treatment to show an effect on cognitive and “global” functioning prior to approval.
“It’s exciting because it’s an acknowledgment that the field has moved from diagnosing in the end stage to diagnosing earlier before dementia starts,” Maria Carrillo, vice president of medical and scientific relations at the Alzheimer’s Association, told Outsourcing-Pharma.com.
Laurie Ryan, director of the National Institute for Aging’s Alzheimer’s Disease Clinical Trials Program, expressed similar excitement, noting that the FDA is making it clearer for industry on what path to take moving forward.
“The FDA has worked closely with investigators,” Ryan said, adding that although the three trials targeting amyloid are the only ones currently moving to later phases, there are many more targets researchers are looking at and other risk factors.
Even though there is currently “no consensus as to what particular biomarkers would be appropriate to support clinical findings in trials in early” Alzheimer’s, the FDA encourages “sponsors to analyze the results of these biomarkers independently with the understanding that these findings will be interpreted in the context of the state of the scientific evidence at the time of a future new drug application or biologics license application submission," according to the guidance,
“The science is definitely catching up,” Ryan said.
Huge opportunity for CROs
The draft guidance was also welcomed by Daniel Burch, PPD VP and global head of therapeutic central nervous system diseases, who told Outsourcing-pharma.com it will attract drug industry investment and create business for contractors.
“The commercial opportunity is huge and the unmet need is huge,” especially as the US government’s interest picks up, “this is the holy grail,” Burch said.
And the influx of spending on Alzheimer’s won’t just be restricted to larger pharmaceutical companies. Even smaller biotech companies are looking at beta amyloid and neuro-protective treatments from different angles, Burch added.
He compared the search for new Alzheimer’s treatments to a “vast sea of darkness with more lamp posts coming up,” and said that he’s “cautiously optimistic” that there will be new treatments to help improve the functioning of Alzheimer’s patients in the coming years. Because there’s such intense interest and increasing investment, treatments for Alzheimer’s could move in a similar direction to treatments for HIV, Burch noted.