BIO’s Chief Scientific Officer Dr. Cartier Esham testified before the House Energy & Commerce Health Subcommittee yesterday on the need for more investment and dialogue to increase development of biopharmaceuticals for neurodegenerative diseases.
An estimated 50 million Americans are impacted by neurodegenerative disorders annually,Subcommittee Chair Anna G. Eshoo (D-CA) said.
“Lack of investment, difficult drug approval processes, and limited understanding of these extremely heterogenous diseases” are obstacles, she said. “Neurologic drugs take, on average, 57% longer to reach approval than drugs for other disease areas.”
“In 2021, we counted 653 clinical development programs for medicines to treat neurological diseases, 43% of which are for neurodegenerative medicines,” said Dr. Esham, citing BIO’s clinical development pipeline tracker. “By comparison, there are 2,798 oncology clinical development programs.”
Investment in neurological drugs has been growing, to “$1.7 billion in 2020, a four-fold increase from 2012,” but still less than the $7 billion going to oncology.
However: “The success rate for neurological programs transitioning from Phase 2 to Phase 3 is 26.8%, which ranks 11th out of the 16 disease categories we analyzed,” Dr. Esham continued, citing a BIO report published in February.
Industry is working with FDA to address a key issue in neurological clinical trials: definition of endpoints, Dr. Esham added.
BIO’s recommendations include:
- “Policies supporting efficient and effective regulatory environments.”
- “Expanded utilization of biomarkers” in endpoints, to determine what works.
- “Continued funding of basic research.”
The big picture: “The growing Alzheimer’s disease epidemic is expected to affect more than 13.8 million people in the U.S. by 2050 and cost well over $1 trillion annually,” said Dr. Esham, citing BIO’s report on innovation addressing Alzheimer’s.
The bottom line: While there are significant scientific and development challenges—as well as less-than-ideal investment—BIO members continue to innovate and stand ready to work with Congress to advance a modern, patient-centric, and inclusive clinical development paradigm.
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