A new bill looks to decrease the time for the development and release of new drugs and medical devices by modernizing clinical trials and removing barriers to collaboration and innovation, according to legislators.
Washington, DC-A new bill looks to decrease the time for the development and release of new drugs and medical devices by modernizing clinical trials and removing barriers to collaboration and innovation, according to legislators.
The key provisions of the 21st Century Cures Act-which was recently unanimously approved by the House Energy and Commerce Committee-are intended to speed the marketing approval process of drugs and medical devices.
For drugs, the bill would allow approval of certain drugs based on early-stage clinical trial results, allowing use of surrogate markers in clinical trials, and providing more efficient review of applications for additional indications. The bill would also mandate U.S. Food and Drug Administration (FDA) consideration of patient-experience data in the approval process.
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For medical devices, the bill would allow for priority review for breakthrough medical devices, a pathway for allowing certain modifications to cleared medical devices without supplemental 510(k) submissions, and expansion of the humanitarian device exemption.
“If we want to save more lives-and we do-and keep the leader in medical innovation and the jobs here, we’ve got to make sure that there’s not a major gap between the science of cures and the way that we actually regulate these therapies,” says Chairman Fred Upton (R-MI), co-author of the bill.
Optometry Times Editorial Advisory Board member William Townsend, OD, FAAO, says that changes to the FDA-approval process are needed.
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“Instead of an agency whose goal is to approve products if they’re safe and effective, the FDA has been put into a defensive position in that it doesn’t want anything to go through that isn’t safe,” Dr. Townsend says.
Dr. Townsend uses the example of diquafosol, which was submitted by Inspire, now Merck.
“FDA required 100 percent clearance of staining,” says Dr. Townsend. “Most adult patients walking into our office will show some dgree of staining. In the case of diquafisol, the FDA set the bar at an unreasonably high level. But the drug has been approved and used internationally with great success. The effort to promote safety can become so protective that we deny people some products that really should be approved. The good thing about this bill is that it still allows for follow-up by the FDA, which is important.”
According to the Committee on Energy and Commerce, the bill aims to accelerate discovery, development, and ultimately, delivery of life-saving drugs and medical devices by:
• Removing barriers to increased research collaboration.
The committee says that the bill breaks down existing barriers to sharing and analyzing health data generated in research and clinical settings.
• Incorporating the patient perspective into the drug development and regulatory review process.
The bill strengthens the FDA’s ability to take the experience of patients with particular diseases and conditions and the effect of their current therapies and use that information to modify and improve potential therapies.
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• Measuring success and identifying diseases earlier through personalized medicine.
The bill would advance personalized medicine and provide guidance for more collaborative development, understanding, and utilization of drug development tools such as biomarkers.
• Modernizing clinical trials.
According to the committee, the bill will allow for greater use of patient-generated registries to speed up the recruitment of participants. The bill will also allow researchers to screen patients in advance to determine if their genetic predisposition will make them better candidates for targeted therapies. It would clear the way to use new and creative adaptive trial designs and deploy the most modern statistical and data tools, while reducing existing, duplicative, or unnecessary paperwork requirements.
• Removing regulatory uncertainty for the development of new medical apps.
The committee says that regulatory uncertainty has slowed down the development of medical apps that generate real-time patient data, which it says hold tremendous promise for improving health care. The bill clarifies apps’ regulatory path moving forward and will speed up the creation and development of these tools.
• Providing new incentives for the development of drugs for rare diseases.
The bill creates new economic incentives for the development of therapies for serious and life-threatening conditions, including rare diseases, which the committee says will translate to more cures.
• Helping the entire biomedical ecosystem coordinate more efficiently to find faster cures.
The bill would create a new coordinating mechanism to remove the choke points that slow the connections among scientific discovery, drug and medical device development, and how these therapies are approved and made available to the patient. According to the committee, the bill improves the entire biomedical ecosystem by ensuring the innovation infrastructure works as quickly and efficiently as possible.
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• Investing in 21st century science and next-generation investigators.
The bill creates a dedicated and offset funding stream of $2 billion per year for five years that will allow congressional appropriators to invest additional resources without impacting current budget caps. The bill includes provisions to invest more resources in the next generation of scientists.
• The bill helps keep and create jobs in the U.S.
The bill will help fight off foreign competitors in order to keep jobs-and add more-in the United States.
Pharmaceutical Research and Manufacturers of America (PhRMA) recently released a statement supporting the bill.
“PhRMA applauds the House Energy and Commerce Committee and the bipartisan efforts of Chairman Upton and Ranking Member Pallone, Health Subcommittee Chairman Pitts, Health Subcommittee Ranking Member Green and Representative DeGette on the passage of 21st Century Cures legislation to improve the discovery and development of new medicines and treatments for patients,” says PhRMA president and CEO John Castellani. “The thoughtful, year-long process that brought countless stakeholders to the table ultimately results in important steps forward for patients.
“Exempting future Food and Drug Administration (FDA) user fees from sequestration is critical to ensuring the FDA is able to fulfill its public health mission by fostering timely patient access to safe and effective new medicines and advancing regulatory science. We are pleased to see this important provision included in the legislation,” he says.
The Pharmaceutical Care Management Association (PCMA) also issued a statement as well; however, the group expressing concerns about how the bill would affect Medicare.
“If the 21st Century Cures initiative can only be advanced by cutting billions from America's premier health program-Medicare-then its costs outweigh any potential benefits it may yield down the road,” the statement reads. “Leveraging payments to Part D plans to fund this legislation ironically could have the unintended consequence of making it harder for beneficiaries to access the very medicines that 21st Century Cures seeks to advance. We urge policymakers to explore different offsets that don't pose new risks to other parts of the healthcare delivery system.”
PCMA isn’t alone in its concerns. Ed Silverman, The Wall Street Journal’s column Pharmalot, recently wrote that, while supporters say the bill will give the FDA the tools to get treatments to patients faster, critics say the section of the bill devoted to drug development raises some red flags.
“Specifically, they point to language that would allow the FDA to approve additional uses for drugs without having to rely on randomized controlled trials,” writes Silverman. “These are considered to be the gold standard for determining whether a medicine offers a benefit and help gauge the extent to which there are risky side effects.”
Silverman writes that instead the bill relies on clinical experience, defined as a mix of observational studies, patient registries, and therapeutic use.
“None of these, however, are viewed as scientifically rigorous for establishing whether a drug may be effective. Instead, critics say the language in the bill is sufficiently, perhaps deliberately, vague,” Silverman writes.