Cancer Policy Monitor: November 2019
Cancer Policy Monitor – Nov. 12, 2019
- Appropriations Update from Capitol Hill
- Hahn Nominated to be FDA Commissioner
- AACR Submits Comments to FDA on Proposed Graphic Cigarette Health Warnings
- 2020 AACR Early-Career Hill Day: Applications close November 25
- Apply Today: Scientist↔Survivor Program at the AACR Annual Meeting
- Vincristine Shortage Highlights Increasing Drug Shortfalls in the U.S.
- FDA Moves to Require Digital Submission of Safety Reports for Investigational New Drug Applications
- FDA Finalizes New Pathway for Select 510(k) Devices
- Guest Post: Early-Career Scientists Partner with Patients
- Registration Available for FDA-AACR Workshop to Examine Under-representation of African Americans in Multiple Myeloma Clinical Trials
- Oncology Approval Recap
-J. Tod Guidry, PhD
With a continuing resolution (CR) currently funding the government at fiscal year (FY) 2019 levels, fears of additional CRs loom as Congress works to complete the FY 2020 appropriations process. The Senate has recently moved several bills forward, while the House of Representatives passed most of their spending bills over the summer. However, numerous roadblocks threaten the likelihood of the two chambers reaching a compromise on all 12 spending bills by the Nov. 21 deadline.
On Oct. 31 the Senate passed a package of four spending bills to fund agencies including the U.S. Food and Drug Administration (FDA), Environmental Protection Agency (EPA), and National Science Foundation (NSF), among others. Senate Majority Leader Mitch McConnell (R-KY) has committed to bringing forward another package next that would incorporate the Labor-HHS-Education bill, which funds the National Institutes of Health (NIH). Chairman Roy Blunt (R-MO) of the Labor-HHS-Education subcommittee previously announced his intention to provide the NIH with a $3 billion budget increase over FY 2019 levels. The House of Representatives passed 10 of its 12 spending bills in June, including a $2 billion boost to NIH funding.
Each of the 12 bills will need to be finalized by a bicameral conference committee and subsequently passed through both chambers of Congress before reaching the president’s desk, though numerous obstacles lie in the way. The House and Senate have yet to agree on 302(b) allocations, which dictate how the spending cap totals agreed to in the Bipartisan Budget Act of 2019 are to be divvied up among the 12 bills. Passing the new four-bill package in the Senate could expedite 302(b) negotiations, according to Sen. Patrick Leahy (D-VT), ranking member of the Senate spending panel.
Partisan disagreements may also serve as significant roadblocks to completion of the appropriations process. While Senate Minority Leader Chuck Schumer (D-NY) expressed support of the first package of bills, he has warned that other bills will not pass the Senate without better satisfying Democratic priorities, including higher spending numbers for the Labor-HHS-Education bill.
The Defense and Homeland Security bills are a major source of contention among legislators, with disagreements over funding for the president’s proposed southern border wall. Senators Leahy and Richard Shelby (R-AL), chair of the Senate Appropriations Committee, have argued for getting less controversial bills to the president’s desk before tackling more partisan measures. However, McConnell may wait for an agreement on Defense before clearing any final conference reports. Furthermore, the president could refuse to sign any appropriations bills into law without assurance for border wall funding.
The ongoing impeachment proceedings could also stall the appropriations process. Impeachment of the president in the House would divert the Senate’s attention away from passing appropriations measures as it conducts an impeachment trial.
Neither Shelby nor Nita Lowey (D-NY), chair of the House Appropriations Committee, have ruled out the possibility of another CR that could extend into December. Congressional leaders, including McConnell, House Speaker Nancy Pelosi (D-CA), and House Majority Leader Steny Hoyer (D-MD) want all 12 spending bills passed by Dec. 31 at the latest. The possibility of a full-year CR has been floated, though according to Shelby, “the threat is not looming just yet.”
CRs hamper the progress of federally funded medical research by introducing unpredictability over funding levels. Along with the broader medical research advocacy community, the AACR advocates for an expedient completion of the appropriations process, with an increase of at least $2.5 billion (for a total funding level of $41.6 billion) for the NIH budget for FY 2020.
The AACR congratulates Stephen M. Hahn, MD, on his nomination as commissioner of the U.S. Food and Drug Administration (FDA). Hahn is a renowned expert in radiation oncology and research, an experienced and highly effective administrator, and an innovative leader. Read the press release.
-Audrey Jackson, PhD
The AACR submitted comments to the U.S. Food and Drug Administration (FDA) in response to the FDA’s proposed rule on new graphic health warnings for cigarette packages and advertisements. The warnings were required by the 2009 Family Smoking Prevention and Tobacco Control Act (Tobacco Control Act), but implementation has been delayed for a decade by litigation from the tobacco industry. The FDA had issued graphic cigarette health warnings in 2011, but those were invalidated by the District of Columbia Circuit Court.
The new health warnings consist of both text statements and color graphics that depict the negative health consequences of smoking. Prior research has shown that graphic health warnings are more effective than text-only statements in conveying the detrimental effects of smoking. The 2009 Tobacco Control Act directed FDA to update the current surgeon general’s cigarette smoking warnings, which have not been changed in 35 years and have become practically invisible to consumers. While the tobacco industry litigation stalled the process in 2011, public health groups recently won a lawsuit that requires FDA to issue new proposed graphic warnings by Aug. 15, 2019, and a final rule by March 15, 2020. In August 2019, FDA proposed a new set of 13 graphic warnings designed to inform and educate the public about the negative consequences of smoking.
The AACR submitted comments developed by the Tobacco Products and Cancer Subcommittee. In the comments, we strongly supported and applauded FDA’s actions in developing the panel of graphic warnings based on the agency’s consumer research. The FDA went through a careful, evidence-based, iterative approach to develop and consumer-test graphic warnings that would address the court’s prior criticisms. The AACR emphasized the need for new warnings to help reduce cigarette smoking, including among cancer patients, and provided supporting evidence for the greater effectiveness of graphic warnings compared to text-only warnings. We also provided a suggested rank-ordering if the FDA decides to select a subset of the 13 proposed warnings, and suggested consideration of a gain-framed graphic label, which would focus on the positive benefits of quitting smoking rather than the negative consequences of smoking. Finally, we urged the agency to advance the proposed rule to a final rule by the court-ordered deadline of March 15, 2020.
The AACR will once again offer a limited number of Associate Members the opportunity to get involved in advocacy by engaging and educating key congressional offices regarding the needs of cancer researchers, particularly those early in their careers. The online application is currently open and will close Nov. 25.
The 2020 Early-career Hill Day will take place Feb. 26-27 in Washington, D.C.
The AACR is now accepting applications for cancer survivors and patient advocates to enroll in the Scientist↔Survivor Program at the AACR Annual Meeting 2020. This unique educational program provides an opportunity for survivors and patient advocates to learn and discuss the latest findings in cancer research, regulatory science, and health science policy. Travel, hotel, and registration expenses will be covered for participants accepted into the Scientist↔Survivor Program.
The curriculum is designed to build enduring partnerships among the leaders of the scientific, survivor, regulatory, and patient advocacy communities. Participants have the opportunity to:
- Attend special lectures about cancer research that are tailored to a lay audience;
- Discuss relevant and timely cancer topics during small group meetings, roundtable discussions and meetings with scientific mentors;
- Explore the scientific sessions of the annual meeting on their own or with scientific mentors or other advocates;
- Communicate to scientists the key issues, questions, and concerns of the survivor and patient advocacy communities;
- Promote their organizations’ missions by participating in their own poster sessions; and,
- Network with scientists and fellow advocates from local, national, and international cancer organizations.
Participants are accepted through a competitive application process.
Learn more and access application materials here.
Please share this announcement with survivors and patient advocates with an interest in cancer research and policy.
-J. Tod Guidry, PhD
A shortage of the chemotherapy drug vincristine has disrupted the treatment of pediatric cancer patients across the country. The drug, which has been used for decades to treat a large portion of childhood cancers, is currently in short supply with no available substitute. The vincristine shortage highlights a growing problem of increasingly frequent drug shortages in the United States, to which the U.S. Food and Drug Administration (FDA) and others have offered recommendations.
Vincristine is used to treat more than half of all pediatric cancers, including brain tumors, lymphoma, and leukemia. It is a staple for treatment of acute lymphoblastic leukemia (ALL), the most common childhood cancer. Pfizer has been the sole supplier of vincristine since Teva Pharmaceutical Industries discontinued production of the drug in June 2019. Since then manufacturing delays at Pfizer have led to the current shortage, with doctors of pediatric cancer patients forced to decide whether to ration the drug or skip doses. The Children’s Oncology Group has recommended modifying clinical trial treatment protocols involving vincristine to consider limiting its dosage, skipping doses during the maintenance phase of treatment, or eliminating the drug from the treatment regimen altogether.
In response to the vincristine shortage, Pfizer plans to accelerate production output of the drug by three- to four-fold. On Oct. 18, the company issued a
statement stating their expectation of a full recovery by January 2020. Pfizer released the latest shipment of the drug in late October. The FDA stated that they are working closely with Pfizer to ensure availability of the drug to patients who need it.
Drug shortages have been a long-standing problem in the United States for years, though their frequency has increased over the past decade. A Drug Shortages Task Force at the FDA was established in 2018 to understand the causes of drug shortfalls and identify solutions. A report issued by the Task Force on Oct. 29 of this year revealed that drugs that went into shortage between 2013 and 2017 were cheaper and “financially unattractive” compared to similar drugs that did not go into shortage. Production of older, generic drugs that command low prices like vincristine is often limited to few manufacturers. Furthermore, drug prices and production rarely increase in response to such shortages.
The Task Force report offers recommendations to government and industry in their efforts to prevent and assuage future shortages, including increasing awareness of companies’ contracting practices that may contribute to drug shortages and the impact of those shortages on public health. The task force supports developing a system to rate drug manufacturing facilities’ ability to produce high-quality drugs with minimum disruption, which could be used to inform consumers and give highly rated companies a competitive advantage. Prioritizing contracting with companies boasting higher quality ratings could ensure a sustainable return on their products, resulting in a more reliable supply of drugs. The report also outlines legislative proposals, planned initiatives and guidances, and new requests in the president’s fiscal 2020 budget geared toward mitigating drug shortages.
In an open letter to the childhood cancer community, Chair of the Children’s Oncology Group Peter Adamson, MD, pressed the need to focus on solutions for today’s pediatric cancer patients. He proposed establishing a national stockpile of key pediatric cancer drugs and allowing importation of vincristine from other countries for patients affected by the current shortage. In the letter, Adamson also issued a call to action to boost advocacy efforts to prevent future shortages of pediatric cancer drugs stating, “While it is clear that the immediate concern and goal is to solve the issue surrounding vincristine, I believe the community should consider advocacy steps to help prevent future shortages of lifesaving cancer drugs for children.”
-Elizabeth KS Barksdale, PhD
In late October, the U.S. Food and Drug Administration (FDA) released
draft guidance announcing a pending requirement that sponsors of commercial investigational new drug (IND) applications submit safety reports directly to the FDA Adverse Event Reporting System (FAERS) via a digital framework. The FAERS database contains information on serious adverse events experienced by patients that may be related to drugs, biologics, and devices regulated by the agency.
Currently, IND sponsors submit safety reports either through the mail or electronically as PDF files. Under the new system, sponsors will submit these reports to the FAERS database as structured data elements in a format consistent with International Council for Harmonisation (ICH) guidelines. The new submission requirements will enable FDA to better track and analyze potential safety signals that occur during clinical trials.
Once finalized, the guidance will apply to IND safety reports as defined by
statute. That is, “The sponsor must report any suspected adverse reaction that is both serious and unexpected…if there is evidence to suggest a causal relationship between the drug and the adverse event.” Only events that meet the provided criteria need to be reported. Additionally, noncommercial INDs—for products that won’t be distributed commercially, investigator-sponsored INDs, and expanded access INDs—will be exempt from the new electronic submission requirements.
IND sponsors will have two years after the final guidance takes effect to comply with the new FAERS submission requirements. Sponsors will have the option to voluntarily submit safety reports as directed in the guidance before the effective date; the FAERS website will be updated to indicate this reporting method when it is available. Importantly, unlike most FDA guidance, the FAERS reporting requirements will be binding. This is because Congress explicitly granted FDA statutory authority to specify the submission format of IND safety reports in section 745(A) of the Federal Food, Drug, and Cosmetic Act (December 2014). Thus, FDA-issued requirements on this topic are legally enforceable.
This digital push is part of the FDA’s Technology Modernization Action Plan (TMAP) that was unveiled in September. The TMAP outlines steps the FDA will pursue to close the gap separating recent scientific advances and the technologies needed to translate them into new therapies and new ways to protect the public health. The three components of the plan include 1) modernizing FDA’s technical infrastructure; 2) enhancing its capabilities to develop technology products in support of its regulatory mission; and 3) communicating and collaborating with stakeholders to drive technological progress.
-Trevan Locke, PhD
In late September, the Center for Devices and Radiologic Health (CDRH) at the U.S. Food and Drug Administration (FDA) shared the Framework for the Safety and Performance Based Pathway, an expansion of the Abbreviated 510(k) pathway. Through this pathway, device developers have the option of showing a new device meets FDA-specific performance criteria rather than needing to perform direct comparisons to an existing “predicate” device.
The 510(k) pathway is a means of premarket submission to the FDA in which a new device is compared to an existing, legally marketed device known as a predicate. To be cleared for marketing, the new device must be determined by the agency to be substantially equivalent to the predicate device. Traditionally, developers directly compare their device to the predicate to establish equivalence.
Instead of direct comparisons to a predicate device, the new Safety and Performance Based Pathway provides an optional pathway based on pre-specified performance criteria set by FDA guidance. Along with the final guidance on the Safety and Performance Based Pathway, the agency released four draft guidances for the initial device types eligible for this pathway, which include spinal plating systems, cutaneous electrodes, conventional Foley catheters, and orthopedic non-spinal metallic bone screws and washers. The criteria in these guidances are based on characteristics of existing, legally marketed devices. Based on stakeholder feedback, the FDA will work to expand this pathway to other device types. Sponsors can begin using this pathway once the device criteria guidances are finalized.
CDRH Director Jeff Shuren, MD, explained the potential benefits of the new pathway, “The major benefit is that the pathway will benchmark modern technology against modern standards while, at the same time, offering a potentially more efficient way to demonstrate a new device is substantially equivalent to devices already on the market. This ensures patients have timely access to beneficial products.”
-Hillary Stires PhD, Christine Hodgdon, and Kimberly Richardson
Part 1 of a 2-part blog. This post is part of a special series on patient partnerships in research and policy.
The goal of cancer research is to improve the lives of those who have been or may be diagnosed with cancer and includes discovering new therapies that keeps cancer growth and spreading at bay, developing strategies to reduce the risk of cancer development, and understanding how a cancer diagnosis and cancer therapies influence survivorship. At the core of this goal is the person; however, many research scientists do not often interact with survivors (those who have ever been diagnosed with cancer). We are research scientists and cancer survivors who have found value in working with one another to improve research, to keep research patient-centric, and to ensure we are focusing on the most pressing challenges cancer survivors face. By working together, we have not only seen research improve, but also our individual understandings of cancer. As an added benefit, we have gained a satisfaction from building relationships with one another. We believe that all research scientists should develop these relationships, but that the best place to start is early in a scientist’s career when they are training as a graduate student, postdoc, or clinical fellow.
Part one of this two-part post will share the perspective of an early career scientist.
-Hillary Stires, PhD
As a postdoctoral fellow at Georgetown University’s Lombardi Comprehensive Cancer Center, I studied endocrine resistance in estrogen receptor-positive breast cancer using breast cancer cells in a culture dish as a model. While I was able to learn a lot about the way the cancer responds to various treatments, I was uncertain how the treatments would influence a person with cancer. After my best friend was diagnosed with breast cancer when we were both 28 years old, I realized how little I knew about people who have been diagnosed with cancer. After she started treatments, I started working on building relationships with cancer patient advocates by presenting to the Georgetown Breast Cancer Advocates (GBCA) and tweeting with #BCSM on Monday nights.
Slowly but surely, I saw my research change. I was more careful to ensure the doses of treatments I was using in my experiments would be tolerable if administered to a person. I considered interactions of the drug I was using with other aspects of treatment, such as radiation, a common procedure in people diagnosed with breast cancer. I wrote the purpose statement for my grants and papers with ease as I considered the impact of my research on the patient.
I saw a request for applications for an Elevate Ambassador for the National Coalition for Cancer Survivorship and immediately applied. The goal of the program is to improve survivorship within the ambassador’s community. I was selected to improve survivorship, or rather relationships with survivors, in my community, the cancer research scientists. I have been working with a group of advocate friends to build the Cancer Trainee Advocate Program (CTAP) in an effort to introduce patient advocates and research scientists while the scientists are in training (graduate students, postdoctoral fellows, or clinical fellows).
This past summer, we held the first Cancer Trainee Advocates Program workshop at Lombardi with the graduate students and postdocs in the Tumor Biology Program. The trainees organize an annual retreat during a Friday afternoon in July, which provided an opportune time to host the workshop. The advocates we invited were from GBCA, recommended patients of oncologists at Lombardi, and advocates I knew from Twitter. Following a lively roundtable discussion between the advocates and the trainees (with questions from advocates from #bcsm!), the trainees held a poster session to discuss their research with the advocates and provided a time for advocates to participate in lab tours between posters. Overall, the event was a raving success, and both the advocates and the trainees expressed the benefits they received from attending.
As cancer research scientists, we are not always given such unique opportunities, but I am here to advocate for more of these connections to be built. Next month, we will feature the perspectives of cancer survivors Christine Hodgdon and Kimberly Richardson, two of my friends that I have met from the patient advocate community who are interested in expanding relationships between patient advocates and research scientists. They both completed the Scientist↔Survivor Program, which offers a unique opportunity for cancer survivors to learn from cancer scientists at the AACR Annual Meeting. As you’ll see, their individual experiences in working with research trainees has not only benefited them; it also inspired them to continue building bridges.
Registration Available for FDA-AACR Workshop to Examine Under-representation of African Americans in Multiple Myeloma Clinical Trials
The American Association for Cancer Research (AACR) is partnering with the U.S. Food and Drug Administration (FDA) to present the FDA-AACR Workshop to Examine Under-representation of African Americans in Multiple Myeloma Clinical Trials on Feb. 13, 2020, in Washington, D.C. The FDA-AACR Workshop to Examine Under-representation of African Americans in Multiple Myeloma Clinical Trials is cochaired by:
- Kenneth C. Anderson, MD, FAACR, program director, Jerome Lipper Multiple Myeloma Center and LeBow Institute for Myeloma Therapeutics and chief, Division of Hematologic Neoplasia, Dana-Farber Cancer Institute; chair, AACR Regulatory Science and Policy Subcommittee
- ‘Lola A. Fashoyin-Aje, MD, MPH, clinical team leader, Division of Oncology Products 2, Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
- Nicole J. Gormley, MD, clinical team leader, Division of Hematology Products, Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
- Paul G. Kluetz, MD, deputy director, Oncology Center of Excellence, U.S. Food and Drug Administration
Registration for this one-day workshop is free and open to the public.
Oncology Approval Recap
In October, the U.S. Food and Drug Administration approved niraparib for patients with advanced ovarian, fallopian tube, or primary peritoneal cancer treated with three or more prior chemotherapy regimens and whose cancer is associated with homologous recombination deficiency (HRD)-positive status. HDR is defined by either a deleterious or suspected deleterious BRCA mutation, or genomic instability in patients with disease progression greater than six months after response to the last platinum-based chemotherapy.
To learn more about this approval of niraparib, see the Cancer Research Catalyst. To learn more about the approval of other cancer therapies, you can find more information on the FDA’s website, and an AACR journal, Clinical Cancer Research, regularly publishes FDA approval summaries.