“Today discoveries supported by NIH often do not come to FDA’s door for six, eight, 10, or even 12 years. And the average cost to get a single drug from the laboratory through the approval process to the medicine cabinet is, according to some estimates, about $1 billion. Other estimates say it’s double that or even more.” –Lamar Alexander
On March 10, U.S. Sen. Lamar Alexander (R-Tenn.) held the first committee hearing on his bipartisan initiative with Ranking Member Patty Murray to “examine how we get drugs, devices and treatments from the discovery process through the regulatory process into our medicine cabinets and doctors’ offices.” Alexander, who chairs the Senate Health, Education, Labor and Pensions committee, looks to address the cost and processes of moving discoveries from bench to bedside.
“Today discoveries supported by NIH often do not come to FDA’s door for six, eight, 10, or even 12 years,” Alexander said. “And the average cost to get a single drug from the laboratory through the approval process to the medicine cabinet is, according to some estimates, about $1 billion. Other estimates say it’s double that or even more.”
Alexander noted that this objective – improving biomedical innovation, including at the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) – has much shared interest, citing President Obama’s precision medicine initiative and the House 21st Century Cures effort.
He added: “This work will affect every single American – from a very ill patient who has run out of treatment options and is counting on the most cutting-edge drug, to an active child with asthma who’s hoping to run faster and farther with the aid of a new drug.”
Other remarks from the senator were as follows:
"We’ve got three major objectives in this committee:
Fixing No Child Left Behind, Reauthorizing the Higher Education Act, And third ... one we’re all looking forward to without exception ... improving biomedical innovation, including the Food and Drug Administration (FDA) and the National Institutes of Health (NIH).
"This initiative builds on work the committee has done – legislation was passed in 1997 and as recently as 2012 to try to get at same goal of speeding drugs and devices while still ensuring they are safe. This is a subject that has a lot of interest.
"President Obama this year announced his new Precision Medicine Initiative, saying: '21st century businesses will rely on American science, technology, research and development. I want the country that eliminated polio and mapped the human genome to lead a new era of medicine – one that delivers the right treatment at the right time. In some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable.'
"In the House, Energy and Commerce Chairman Fred Upton and Representative Diana Degette have been working on parallel tracks on their 21st Century Cures initiative to accelerate the pace of cures in America.
"In late January, Sen. Burr and I released a report titled 'Innovation for Healthier Americans' that examined the process we have in place today for drug and device development. We have received over 80 comments already, and have shared those with the staff of all members on the committee.
"Our committee also has a staff bipartisan working group that has been meeting for around a month now, learning more about the key agencies involved in biomedical research and development.
"We have with us today Dr. Francis Collins, Director of the National Institutes of Health, which funds and enables much of the early stage research that leads to medical breakthroughs. And Dr. Margaret Hamburg, the head of the Food and Drug Administration, which regulates all the medical products we come in contact with.
"Dr. Collins wrote in 2013 that:'Drugs exist for only about 250 of the more than 4,400 conditions with defined molecular causes. And it takes far too long and far too much money to get a new drug into our medicine cabinets. This is an old problem that cries out for new and creative solutions.'
"Since Dr. Collins wrote that, the number of conditions with defined molecular causes has increased to more than 5,400, yet the number of new drugs approved has not kept pace with these discoveries.
"Dr. Hamburg has said that 'We are left relying on the 20th century approaches for the review, approval and oversight of the treatments and cures of the 21st century.' So today’s hearing is a perfect place for us to start—with the heads of these two critical agencies, both of whom have sounded the alarm on our existing process for drug and device development.
"I look forward to hearing from the witnesses more about their thoughts on these five issues that Senator Burr and I identified in our report:
It costs too much to bring medical products through the pipeline to patients. As science and technology advance, the discovery and development process takes too long for medical products to make their way to patients. FDA’s responsibilities have grown to include many activities unrelated to the core function of regulating medical products to advance the public health. The disparity in scientific knowledge at FDA and the fast pace of biomedical innovation are slowing, and in some cases, stifling innovation in American medicine. A working FDA is essential to continuing biomedical innovation in the United States and maintaining America’s global leadership in medical innovation.
"In the words of Andrew Eschenbach, the former Commissioner of the FDA and Director of the National Cancer Institute: 'We stand on the cusp of a revolution in healthcare. Advances in molecular medicine will allow us to develop powerful new treatments that can cure or even prevent diseases like Alzheimer's and cancer. Tomorrow's high-tech cures can also slash health-care costs and eliminate ineffective treatments.'
"I look forward to taking the first step toward addressing these important issues. If we do it right, our work here will help improve the lives of every single American."