“The 21st Century Cures Initiative is a welcome reminder that America’s policymakers can still come together to meet and solve great challenges.”
The Subcommittee on Health continued its efforts this week on the #Path2Cures with a hearing on Wednesday to examine “21st Century Cures: Modernizing Clinical Trials.” Subcommittee members examined how improvements to the clinical trial process can accelerate the discovery, development, and delivery cycle of new cures and treatments. Paul Howard of the Manhattan Institute explains the need for modernization in The Morning Consult writing, “we need to create a clinical trial process for the 21st Century, one that takes advantage of all of the technologies at our disposal to ensure that every patient has an opportunity to participate in clinical trials.” Howard notes that 21st Century Cures is, “bringing stakeholders together to transform how America discovers, develops, and delivers new medicines.”
July 9, 2014
We Need 21st Century Clinical Trials to Produce 21st Century Cures
By Paul Howard
America got a gut-wrenching view of the complexities of drug development earlier this year when Josh Hardy, a 7-year-old cancer patient undergoing a bone marrow transplant, developed a life-threatening adenovirus infection that his compromised immune system couldn’t fight off.
With no other effective options, Josh’s parents and physicians turned to a small biotech company called Chimerix, which was developing an experimental antiviral drug to treat a related virus.
At first, Chimerix turned down the family’s request for the drug. Chimerix had previously operated an “expanded access” program for patients like Josh, but had closed it two years ago to concentrate its scarce funds on completing clinical trials for another viral infection, CMV.
Chimerix eventually made its drug – CMX001 – available to Josh after facing a barrage of social media criticism, and after the FDA agreed to allow Chimerix to treat him in the context of a new small trial on patients with adenovirus. Fortunately, the drug cleared Josh’s infection in a matter of days, and today Josh’s family is optimistic for his full recovery. …
Josh’s moving story certainly underscores how difficult, expensive, and uncertain the current drug development paradigm is – and how current clinical trial protocols often don’t meet the needs of desperately ill patients. Granting expanded access to a handful of patients at a time, or even a few hundred, won’t help us attain what should be our overarching goal – slashing the time and cost required to bring important new medicines to patients.
Instead, we need to create a clinical trial process for the 21st Century, one that takes advantage of all of the technologies at our disposal to ensure that every patient has an opportunity to participate in clinical trials (if they want to) testing therapies tailored meet their specific disease state. …
What is needed now is a national effort to overhaul an outdated clinical trial paradigm that serves too few patients at too high a cost – in dollars and lost lives.
Fortunately, there is a long-term, bipartisan effort in the House of Representatives to truly accelerate patient access to important new therapies. The 21st Century Cures Initiative, led by Energy and Commerce Chairman Fred Upton (R, MI), and Rep. Diana DeGette (D, CO) is bringing stakeholders together to transform how America discovers, develops, and delivers new medicines. In a Congress too often defined by partisan rancor, the 21st Century Cures Initiative is a welcome reminder that America’s policymakers can still come together to meet and solve great challenges.
Josh’s struggle is repeated thousands of times every day. And in an era of Facebook and Twitter, every patient has a ready-made megaphone to demand better solutions when they come up against unnecessary roadblocks.
Washington, for a change, appears to be listening.
Read the complete article online here.