|The 21st Century Cures Act is an|
ambitious initiative that deserves
by Cindy Fazzi
Editor, MasterControl Insider
The 21st Century Cures Act is a massive piece of legislation (996 pages) with an equally tremendous ambition. The life science industry heralded it as a victory, while critics expressed some concerns. Everyone agrees, however, that there’s a need to support medical innovation and advance regulatory compliance. The reform-minded law unites all sides to that extent.
The legislation is estimated to cost $6.3 billion upon implementation (1). Signed into law by President Obama on Dec. 13, it generated buzz primarily for Vice President Joe Biden’s Cancer Moonshot initiative, which will receive $1.8 billion in funding under the act, and for providing $1 billion over two years in the fight against opioid abuse.
Accelerate FDA Process
The law also shone a spotlight on the U.S. Food and Drug Administration (FDA) because it gives the agency new authority to speed up the path to approval of drugs and medical devices. Support for innovation includes measures to help the government hire and retain scientists and to develop better ways for managing electronic health records.
Dr. Robert Califf, FDA commissioner, praised the law as a big step forward. “Great progress has been made toward our shared goal of advancing regulatory science so we can continue to speed the discovery, development, and delivery of medical products,” he said (2).
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Matt Lowe, MasterControl’s executive vice president, likewise lauded the effort to streamline the regulatory process (3). “The most significant thing I see is allowing safety data from existing drugs seeking new indications to be considered in the approval process,” said Lowe, a mechanical engineer with over a dozen years of medical device experience in product development, product management, and regulatory compliance.
In the same vein, the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Advanced Medical Technology Association (AdvaMed) expressed support for the initiative (4). Scott Whitaker, AdvaMed president and CEO said, “When fully implemented, this legislation will help accelerate the discovery, development, and delivery of medical advancements to ensure more timely access to new treatments and cures for patients in need.”
The law’s “pro-patient, science-based reforms” will enhance the competitive market for biopharmaceuticals and drive greater efficiency in drug development, according to Stephen J. Ubl, PhRMA president and CEO.
While the FDA and the life science industry see the 21st Century Cures Act as the key to speeding up the regulatory process, critics consider it a double-edged sword. They question whether the very measures that will speed up the approval processes might jeopardize public health over the long haul.
Public Citizen said the law weakens the FDA standards for product approval and lowers the bar for the type of evidence needed to meet post-approval requirements. In particular, the nonprofit advocacy group criticized the provisions that allow summary level review and “real world evidence” (5).
Summary level review (6) allows “a summary of clinical data that demonstrates the safety and effectiveness of a drug with respect to a qualified indication” to be used to support the approval of a supplemental application. Real world evidence (7) refers to data regarding usage of a drug from sources other than randomized clinical trials.
Critics are concerned that these types of data are lower quality and will erode the FDA’s standard for evaluating evidence of a medical product’s effectiveness.
The FDA’s head noted the common misperception that randomized clinical trials and real world evidence are incompatible. “In fact, the use of randomization within the context of clinical practice will constitute a major advance in evidence generation and we are actively encouraging proposals with this combination of randomized trials conducted in real world practice,” said Califf (8).
Life Science Burden
Life science companies know too well the burden of voluminous, repetitive evidence required during the FDA approval process.
“Why replicate safety data for a drug already in widespread use just because a company is seeking a new indication? This is costly and a waste of time and delays a new therapy from reaching the market in a timely manner,” said Lowe. “When the drug was first approved for its initial indication(s), the trials were done for safety, so why conduct them again?”
Onward and Upward
Like most reform-minded initiatives, the new law will elicit strong reactions. And like life itself, the truth about its effectiveness and shortcomings lies somewhere along the continuum of pros and cons.
What’s undeniable is that the 21st Century Cures Act is a big and ambitious undertaking with bipartisan support. Only time will tell if this is the antidote to our public health needs, but it’s a huge step in the right direction, and it deserves our support and the efforts of all stakeholders to make it work.
Cindy Fazzi is the editor of MasterControl Insider, a monthly publication for MasterControl users. She writes about the life science industry and other regulated environments. Her two decades of experience as a news reporter, writer, and editor includes working for the Associated Press in Ohio and New York. She has a master’s degree in journalism from Ohio State University.
(1) The 21st Century Cures Act, http://docs.house.gov/billsthisweek/20161128/CPRT-114-HPRT-RU00-SAHR34.pdf
Estimated cost, https://www.cbo.gov/sites/default/files/114th-congress-2015-2016/costestimate/hr34.pdf
(2) “21st Century Cures Act: Making Progress on Shared Goals for Patients,” by FDA Commissioner Robert M. Califf, Dec. 13, 2016, http://blogs.fda.gov/fdavoice/index.php/category/reg-sci/
(3) Matthew M. Lowe, executive vice president at MasterControl, is the author of “Convergence of Compliance and Technology: How Technology Has Changed Regulatory Compliance in the Past Decade.”
(4) AdvaMed represents 80 percent of medical technology firms in the U.S. PhRMA represents the country’s leading pharmaceutical and biotechnology companies and supports 3.4 million jobs in the U.S.
(5) From Public Citizen’s website: http://www.citizen.org/documents/concerns-21st-century-cures-november28.pdf
(6) and (7) From the 21st Century Cures Act, http://docs.house.gov/billsthisweek/20161128/CPRT-114-HPRT-RU00-SAHR34.pdf
(8) Ibid note 2.