FDA’s approval process for drugs is “slow and cumbersome,” President Donald Trump said in his first address to a joint session of Congress on Feb. 28.
In a wide-ranging speech on national security, the economy, foreign policy, and health care, Trump zeroed in on FDA, citing a patient’s experience with drug development as evidence for slashing “restraints” at the agency: “Today is Rare Disease Day, and joining us in the gallery is a rare disease survivor, Megan Crowley,” Trump said Tuesday evening, addressing the Senate and House members, and Supreme Court justices. “Megan was diagnosed with Pompe Disease, a rare and serious illness, when she was 15 months old. She was not expected to live past five.
“On receiving this news, Megan’s dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan’s life. Today she is 20 years old—and a sophomore at Notre Dame. Megan’s story is about the unbounded power of a father’s love for a daughter.
“But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need. If we slash the restraints, not just at the FDA, but across our government, then we will be blessed with far more miracles like Megan.
“In fact, our children will grow up in a nation of miracles.”
Trump’s comments on FDA come shortly after reports that the White House’s FY18 budget request would cut $54 billion from non-defense federal agencies to boost the defense budget. Earlier, on Jan. 30, the president signed an executive order requiring agencies to cut two regulations on businesses for each new regulation introduced.
“We are facing some serious and challenging fiscal headwinds in 2017, especially as we advocate for and seek to secure robust, sustained, and predictable annual funding increases for the NIH and FDA,” said Jon Retzlaff, managing director for science policy and government affairs at the American Association for Cancer Research. “These challenges were underscored by President Trump’s announcement this week that he plans to propose boosting defense spending by $54 billion in his FY 2018 budget by offsetting it by an equivalent cut from the rest of the government’s discretionary budget.
“It’s very concerning that the Trump Administration appears to be geared up to use the non-defense, discretionary accounts of the federal government, as, in essence, a bank to pay for the president’s proposals to increase military and veterans spending, pay for a massive infrastructure program, build a wall along the southern border, and cut taxes for all income groups,” Retzlaff said to The Cancer Letter. “Since the NIH and FDA are both funded from the non-defense, discretionary side of the budget, the Trump administration’s FY 2018 budget proposal would very likely slow the rate of progress in our understanding of the hundreds of diseases that afflict millions of people, and reduce the number of safe and effective treatments available to patients.
“It’s just paramount that if we are to help build on our nation’s prior investments in medical research, ensure that our nation is able to respond to emerging health and research needs, and train the future generation of scientists, the Trump administration, as well as our leaders in Congress, must prioritize funding for the NIH and FDA.”
Questions about Trump’s remarks on FDA should be directed to the White House, an agency spokesperson said to The Cancer Letter.
We were very disappointed when we heard President Trump state during his speech to a joint session of Congress on Tuesday night that the ‘slow and burdensome approval process at the U.S. Food and Drug Administration keeps too many advances from reaching those in need.’ We strongly disagree with this characterization about the FDA.
In 2016, the FDA Center for Drug Evaluation and Research approved 22 novel drugs, which is less than the average number—30—approved annually during the past decade, but the “number of applications for these drugs that sponsors have submitted over time has remained relatively stable,” the agency said in its 2016 Novel Drugs Summary.
As of Dec. 14, 2016, almost all—95 percent—of the novel drugs approved in calendar year 2016 were approved in the first review cycle, and met their PDUFA goal dates for the approval review cycle, according to a 2016 CDER new drug review update.
FDA officials said in the document that the center has granted 141 breakthrough therapy designations since the FDA Safety and Innovation Act was signed in July 2012.
Almost half of these designations were granted for drugs that are indicated for the treatment of cancer and hematologic conditions, which fall under the purview of the Office of Hematology and Oncology Products.
Until recently, OHOP was led by the agency’s “cancer czar,” Richard Pazdur, who is now director of the FDA Oncology Center of Excellence. An acting director for OHOP is yet to be named.
One of the landmark achievements of then-Vice President Joe Biden’s National Cancer Moonshot Initiative, Pazdur’s OCE aims to consolidate the agency’s cancer portfolio and serve as an incubator for developing new regulatory frameworks for cross-center review of cancer-related products.
Trump’s characterization of FDA is disappointing, said AACR CEO Margaret Foti, CEO.
“We were very disappointed when we heard President Trump state during his speech to a joint session of Congress on Tuesday night that the ‘slow and burdensome approval process at the U.S. Food and Drug Administration keeps too many advances from reaching those in need,’” Foti said to The Cancer letter. “We strongly disagree with this characterization about the FDA.
“For the past six years, the AACR has had the special opportunity to work even more closely with the oncology team at the FDA, and what we’ve witnessed is that Dr. Pazdur and his highly qualified colleagues at the FDA’s Office of Hematology and Oncology Products are working tirelessly to speed the availability of therapies for cancer patients, especially when the drugs are the first available treatment or have advantages over existing therapies.
“Dr. Pazdur, who has led that FDA Office since 2005, and who was most recently appointed as Director of the FDA Oncology Center of Excellence, has recruited a remarkable team of oncologists and other experts who are extremely committed to gaining a better understanding of the needs of cancer patients, clinical oncologists, physician-scientists, and developers of novel cancer therapies so as to speed the delivery of innovative cancer treatments. Dr. Pazdur and his colleagues are respected in the oncology community for their creative and groundbreaking methods to ensure a flexible, evidence-based regulatory approach to expediting the approval of promising new cancer drugs.
“It’s important to also point out that the FDA is significantly underfunded given the scope of the cancer problem and the challenges and complexities surrounding the review and approval of safe and effective cancer therapies. Additional funding for the FDA is sorely needed to incorporate the latest scientific breakthroughs into the approval of cancer therapeutics.”
The agency’s cancer experts have been able to approve drugs quickly while maintaining quality, said Clifford Hudis, CEO of the American Society of Clinical Oncology.
“ASCO supports efforts to continuously increase efficiencies to ensure the delivery of safe and effective treatments to patients with life-threatening diseases,” Hudis said to The Cancer Letter. “A robust and stable commitment to research and regulation that supports drug discovery and development leading to the efficient approval of safe and effective treatments offers the greatest hope to millions of Americans patients and families coping with cancer.
In format provided by Blumenthal and Pazdur (doi:10.1038/nrclinonc.2017.15)
Source: FDA, Nature Reviews | Clinical Oncology
“Within the FDA, the oncology division has demonstrated that it can increase the pace of drug approval and make more treatments available faster while maintaining critical safety and efficacy standards. We look forward to working with the administration to build on these successes while ensuring that the FDA continues to protect the health and well-being of Americans.”
The number of cancer drugs approved is a testament to FDA’s ability to review and rapidly bring therapies to the U.S. market, said Nancy Davidson, AACR president, executive director of oncology for Fred Hutchinson/University of Washington Cancer Consortium, and president of Seattle Cancer Care Alliance
“We’ve seen firsthand how the FDA has worked for years to build collaborative partnerships with academia, industry, other government agencies, scientific societies, and patient advocacy organizations to improve both the pace and quality of new cancer drugs reaching patients,” Davidson said to The Cancer Letter.
“On an annual basis, approximately 30 percent of all new drugs approved by the FDA are oncology products, and Dr. Pazdur, along with the team at the FDA, has led the approval of many innovative treatments for cancer patients, such as the recent approvals of immune-check point inhibitors, immune modulators, and many of the targeted therapies that have extended the lives of patients and greatly improved their quality of life.
“Additionally, Dr. Pazdur and colleagues have embraced regulatory science to truly inform and improve the way in which new cancer medicines are evaluated for their safety and efficacy. For example, they are effectively employing a variety of regulatory tools such as master clinical trial protocols, expedited approval pathways, including the agency’s new breakthrough therapy designation, and clinical trial enrichment strategies for approving targeted therapies in oncology.
“In the case of the breakthrough therapy designation, the FDA received statutory authority in 2012 to designate medical products as a “breakthrough therapy” if the therapy treats a serious or life-threatening disease or condition and if preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies.
“Since that time, the FDA has received over 500 breakthrough therapy designation applications and granted 170, of which more than 60 therapies have received accelerated approval, a majority being oncology products. These successes are a testament to the accomplishments of the FDA in bringing cancer medicines rapidly to patients.”
