Cancer Policy Monitor: September 8, 2020
- Join the Rally for Medical Research National Day of Action on Sept. 17
- AACR to Release Inaugural Cancer Disparities Progress Report in Sept. 16 Virtual Briefing
- Appropriations Update from Capitol Hill
- RACE for Children Act Provisions Take Effect
- FDA PMTA Regulatory Deadline is Quickly Approaching
- AACR to Release Cancer Progress Report 2020 in Sept. 23 Virtual Briefing
- Real-Time Oncology Review Pilot Accelerates Approval Timelines
- Recent Breakthroughs in Lung Cancer Treatment Dramatically Improve Survival
- FDA Finalizes Guidance for Developing Male Breast Cancer Therapies
- Vaping Linked to Higher Risk of COVID-19 in Teen and Young Adults
- Cancer Screenings and Treatment are Still Important During COVID-19
- Patient Advocate Dialogue: Cancer Disparities
- SAVE THE DATE: Virtual Patient Advocate Forum: COVID-19 and Cancer
- Oncology Approval Recap
Join the Rally for Medical Research National Day of Action on Sept. 17
The Eighth Annual Rally for Medical Research will be held Thursday, September 17, bringing patient advocates, caregivers, researchers, clinicians and other advocates together for virtual meetings with members of Congress in support of funding for the National Institutes of Health (NIH). You can join them by participating in the National Day of Action, also on September 17. Take a moment to contact your members of Congress that day via email or social media and tell them why federal funding for medical research is so important for you, your family and friends. Find out more about messaging and tools that you can use to contact your members of Congress by visiting the Rally for Medical Research website.
AACR to Release Inaugural Cancer Disparities Progress Report in September 16 Virtual Briefing
The AACR will release its inaugural Cancer Disparities Progress Report during a virtual briefing on Wednesday, September 16.
This report will feature the latest research on why disparities in cancer incidence, diagnosis, treatment, and survival exist, and what can be done to address them. The report has been developed by the preeminent cancer disparities researchers in the country and builds upon the AACR’s longstanding commitment to reducing cancer disparities through research, including our annual conference on the Science of Cancer Health Disparities.
Learn more information on the briefing.Register today
Appropriations Update from Capitol Hill
-Marc Johnson, MPP
As diagnosed cases and deaths of COVID-19 continue to rise, negotiations between the Democratic-led House and the Republican-led Senate (with support from the White House) regarding emergency supplemental appropriations and FY 2021 appropriations are stalled.
In addressing FY 2021 Appropriations, the House passed the “Defense, Commerce, Justice, Science, Energy and Water Development, Financial Services and General Government, Labor, Health and Human Services, Education, Transportation, Housing, and Urban Development Appropriations Act of 2021” known as a “Minibus,” which is a six-bill appropriations package, totaling $1.3 trillion in discretionary funding on July 31. See a summary of the bill. The Labor-Health and Human Services-Education, and Related Agencies (LHHS) portion of the bill calls for $196.5 billion in overall funding, with $96.4 billion of the amount dedicated to the Department of Health and Human Services (HHS). The National Institutes of Health (NIH) would receive a total of $47 billion, an increase of $5.5 billion above the FY 2020 enacted level. The bill provides for $42 billion in annual appropriations, an increase of $500 million above the 2020 enacted level and $8.6 billion above the president’s budget, as well as $5 billion in emergency appropriations. Each institute and center would receive no less than a 7 percent increase in funding, and the bill calls for additional “targeted investments” such as $5.5 billion in emergency funding to improve capacity at research institutions, $80 million for Research Centers in Minority Institutions, and although no specific amount was specified, continued robust investments in the Cancer Moonshot. LHHS Subcommittee Chairwoman Rosa DeLauro stated that the subcommittee’s portion of the bill “…will help develop new cures, support critical health services…” and provides a “…critical step to invest in beating this virus (COVID-19) and closing the gaps it has exposed.” Senate Republicans voiced their opposition to the Minibus and have not yet moved forward on any FY 2021 appropriations bills.
In addressing Emergency Supplemental Appropriations, Senate Republicans announced the “Health, Economic Assistance, Liability Protection, and Schools Act” or the “HEALS Act,” which is their $1.1 trillion alternative to the House-passed (Democrat-written) “HEROES Act.” The HEALS Act is combination of eight smaller bills referred to six Senate committees. Senate Majority Leader Mitch McConnell touts this legislation as a “bold framework to help our nation,” with a focus on reopening schools, the overall economy, and addressing the COVID-19 pandemic. One of the eight bills, S. 4320, the “Coronavirus Response Additional Supplemental Appropriations Act of 2020” sponsored by Senate Appropriations Committee Chairman Richard Shelby (R-AL), would allocate $306 billion to primarily address the COVID-19 health response. Labor-HHS-Education, and Related Agencies would receive almost 75 percent of the funds in the bill. See a breakdown of the appropriations in this bill. In the HEALS Act, NIH would receive a total of $15.5 billion in emergency supplemental funding available through September 30, 2024. These funds would go towards offsetting the costs related to reductions in lab productivity resulting from the coronavirus pandemic, accelerating research and development of therapeutic interventions and vaccines, and supporting additional scientific research. With internal disagreement among Senate Republicans and opposition to the bill by House Democrats, the bill hasn’t moved forward.
In the midst of August recess, Speaker Pelosi called the House back for an emergency session to vote on legislation addressing funding for the U.S. Postal Service (USPS). The House-Democrat led proposal would call for $25 billion in additional funding to counter actions to postal operations made by the Trump Administration. On August 22, the bill passed along party lines 257-150, with some Republicans voting with Democrats. As a counter to the Democrat’s funding plan for the USPS, Senate Republicans put forth the “Delivering Immediate Relief to America’s Families, Schools and Small Businesses Act” or what is being called a “skinny” version of proposed COVID-19 funding (prior to the House passage of the USPS funding bill). The draft bill included a decrease in USPS funding, but it did provide $29 billion in funding for a COVID-19 vaccine and drug development and distribution, and $16 billion for testing and contact tracing. There is not any mention of NIH funding in this proposal.
Emergency supplemental appropriations and FY 2021 appropriations discussions will occur after both the House and Senate return from August recess. An emergency supplemental appropriations package does have a chance of passing, but negotiations will become increasingly political as the 2020 campaign season ramps up. The FY 2021 appropriations process is at a stand-still and will likely not be resolved prior to the end of the current fiscal year on September 30. A continuing resolution (CR) would then be needed in order for the federal government to continue operations.
RACE for Children Act Provisions Take Effect
-Brandon Leonard, MA
On August 18, 2017, key provisions of the Research to Accelerate Cures and Equity (RACE) for Children Act were signed into law as part of the FDA Reauthorization Act of 2017. The RACE Act requires that drug developers study in pediatric populations their targeted cancer therapies developed for adult populations, if the therapies’ mechanisms of action involve molecular targets that are present in cancers that affect both pediatric and adult patients. To facilitate this process, the RACE Act obligated the development of a Pediatric Molecular Targets List detailing molecular targets that do and do not meet the criteria. As of August 18, 2020, applications submitted to the FDA for therapies meeting RACE Act criteria must have agency-approved pediatric study plans.
This policy change has the potential to significantly increase the number of new therapies studied in and ultimately approved for pediatric cancer patients by addressing a key challenge: because of the relatively small population of children with cancer, there has been little market incentive for the biopharmaceutical industry to develop new pediatric oncology drugs. Since the passage of the RACE Act in 2017, the FDA has worked with industry, researchers, clinicians, and advocates to prepare for its implementation.
The RACE Act provisions are an update to the Pediatric Research Equity Act (PREA) of 2003. Under PREA, drug companies were required to develop drugs to treat diseases for children as well as adults. This was not applied to cancer, however, because cancers develop in different organs in children and adults. Under the new law, companies developing a cancer treatment for adults would also undertake PREA studies in children when the molecular target of the drug is relevant to a pediatric cancer.
FDA PMTA Regulatory Deadline is Quickly Approaching
–Carmine Leggett, PhD
On July 12, 2019, the U.S. District Court for the District of Maryland ruled in favor of the plaintiffs and ordered the Food and Drug Administration (FDA) to require manufacturers of e-cigarettes, cigars and other new tobacco products that were on the market as of August 8, 2016, to submit applications for premarket review by May 12, 2020. However, due to the COVID-19 pandemic, on April 22, 2020, the FDA petitioned the court for an extension, asserting that the pandemic delayed the agency’s ability to adhere to the ruling. The extension was granted, moving the May 12 deadline to September 9, 2020.
To facilitate the submission of Premarket Tobacco Product Applications (PMTA), the FDA has created a webpage to provide manufacturers and importers with a single location for information and resources related to tobacco product applications for deemed new tobacco products. Marketing orders are given to PMTAs that have demonstrated that the new tobacco product is appropriate for the protection of the public health, which is determined with respect to the risks and benefits to the population as a whole, including users and non-users of tobacco products, and taking into account the increased or decreased likelihood that: existing users of tobacco products will stop using such products; those who currently do not use tobacco products will start using such products. Once an application is submitted, companies can keep selling their products one year, without official approval, or until a negative action is taken by the FDA.
AACR to Release Cancer Progress Report 2020 in September 23 Virtual Briefing
The annual AACR Cancer Progress Report to Congress and the American public is a cornerstone of the efforts of the AACR to educate the public about cancer and the importance of biomedical research, as well as to advocate for increased federal funding for the NIH, NCI, FDA, and CDC. This year’s report chronicles how federally funded research continues to save and improve lives, and it shows that our ability to fully capitalize on our ever-growing knowledge of cancer is dependent on robust, sustained, and predictable federal funding.
The AACR Cancer Progress Report 2020 will be officially released during a virtual briefing that is open to the public.
Learn more information on the briefing.Register today
Real-Time Oncology Review Pilot Accelerates Approval Timelines
– Trevan Locke, PhD
In August, the U.S. Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) published an update on the Real-Time Oncology Review (RTOR) pilot program in Clinical Cancer Research. The RTOR program, launched in February 2018, is intended to facilitate earlier submission of datasets to the FDA to allow reviewers to start the review of drug applications sooner. From February 2018 to April 2020, 20 oncology applications reviewed as RTOR submissions showed shorter median approval times than applications reviewed under normal priority review processes.
The 20 applications that used the RTOR program from February 2018 to April 2020 included 18 supplemental drug applications and two new molecular entity (NME) applications. The median time from application submission to approval was 3.3 months and all 20 applications received approval. Approval was granted ahead of the PDUFA date for 18 of the applications, with approval being granted a median of 2.9 months ahead of the PDFUA date. Compared to oncology approvals conducted under normal priority review in the same time period, RTOR applications had shorter median approval times for both NMEs (4.5 versus 6.7 months) and supplemental applications (3.1 versus 5.8 months).
RTOR allows FDA review staff an early look at the data prior to official application filing. This provides an opportunity for increased discussion between the agency and the applicant, which can allow for early identification of potential issues and the identification of additional analyses that might address those issues. Though productive, RTOR is resource-intensive, which may limit how many applications the agency can accept through the RTOR program.
Through the OCE, the review of oncology therapies has been a testbed for new regulatory science approaches, such as RTOR. Facing a pandemic, the RTOR pilot program has received more attention as the agency considers how to approach COVID-19 therapies. The lessons learned by the FDA oncology staff through RTOR and other OCE regulatory science initiatives will be instructive as similar efforts are developed in other parts of the agency to aid in the review of COVID-19 related products and other high priority applications.
Recent Breakthroughs in Lung Cancer Treatment Dramatically Improve Survival
-Nicholas Warren, PhD
Lung cancer has been the leading cause of cancer-related death in the United States for decades. According to the Surveillance, Epidemiology, and End Results Program, the average five-year survival rate for all patients with lung cancer was 20.5 percent from 2010-2016. However, the stage of lung cancer at diagnosis greatly impacts overall survival rates; when lung cancer is detected early, 59 percent of patients survived five years, but only 5.8 percent survived five years if they were diagnosed after the cancer spread throughout their body. Unfortunately, more than half of all patients with lung cancer were diagnosed with late-stage cancer between 2010-2016, when surgery and traditional treatment options are not effective. A new report in the New England Journal of Medicine by officials at the National Cancer Institute (NCI) detailed how lung cancer survival rates dramatically improved during 2010-2016 due to breakthroughs in new targeted therapies.
Nadia Howlader, PhD, et al. found improved lung cancer outcomes for both men and women, as well as every racial and ethnic group studied. The likelihood that male patients with lung cancer would survive two years increased from 26 percent in 2001 to 35 percent in 2016; for female patients, the likelihood of surviving two years increased from 35 percent to 44 percent. Although disparities still exist, there were similar increases in survival for non-Hispanic White, non-Hispanic Black, Hispanic, and Asian and Pacific Islander patients. Female Asian and Pacific Islander patients with lung cancer have the highest two-year survival rate, with >50 percent in 2016. Conversely, male non-Hispanic Black patients with lung cancer have the lowest two-year survival rate, with just under 30 percent in 2016. Additionally, lung cancer mortality decreased at faster rates than decreases in newly diagnosed lung cancers. This suggests that improved treatment options, like those targeting gene mutations driving lung cancer, are having a greater impact on lung cancer mortality than prevention efforts.
The authors suggested a major contributor to improved survival was routine screening of genetic alterations that sensitize lung cancer to targeted therapies. Mutations in the EGFR and ALK genes promote uncontrolled cellular growth to drive cancer, but cancer researchers have developed therapies which specifically target these mutated proteins. Screening for EGFR and ALK alterations became standard in cancer centers in the early 2010s, coinciding with a rapid increase in cancer survival. These advances highlight the importance of cancer research to help save the lives of thousands of patients.
The AACR recently hosted a Lung Cancer Dialogue with two EGFR positive lung cancer survivors, Ivy Elkins and Jill Feldman, who discussed their experiences with targeted therapies and creating the patient advocacy organization EGFR Resisters. Ms. Elkins relayed how she was surprised to even be diagnosed with lung cancer, because she never smoked tobacco and did not think she could get lung cancer. In fact, EGFR mutations are most common in patients with lung cancer who have never smoked, and non-smokers comprise 10-15 percent of all patients with lung cancer. Ms. Elkins was diagnosed after the cancer had spread to her bones and brain, but is now six years-post diagnosis with no evidence of disease thanks to this powerful new anti-EGFR therapy. Through EGFR Resisters, Ms. Elkins and Ms. Feldman are raising awareness of EGFR positive lung cancer and advocating against the stigma of lung cancer within pharmaceutical companies and research funding agencies, because no patient should be blamed for their disease.
Dr. Howlader, et al. only analyzed lung cancer outcomes through 2016, which included just the beginning of using new immunotherapies to treat advanced lung cancer. Survival rates have likely continued to improve as more therapies for lung cancer become FDA approved. Immunotherapies that activate patients’ own immune systems to fight cancer hold incredible promise and have become widely used in lung cancer treatment. As cancer researchers continue their critical work developing new therapies, we will hopefully be able to cure more types of cancer.
FDA Finalizes Guidance for Developing Male Breast Cancer Therapies
-Trevan Locke, PhD
Breast cancer is rare in males. This rarity has often resulted in males being excluded from breast cancer trials leading to a lack of data from prospective, randomized clinical trials and limited approved therapies. To help address this gap, the U.S. Food and Drug Administration finalized guidance in August that provides recommendations to sponsors on the development of drugs for male patients with breast cancer.
The FDA recommends that the eligibility criteria for clinical trials of breast cancer therapies allow for inclusion of both males and females. If the protocol proposes to exclude males, scientific rationale should be provided.
When males have limited to no inclusion in trials for a drug, the FDA suggests that it may be possible to extrapolate findings to include male patients if available data supports doing so. If additional data needs to be generated, small single arm trials or real-world data sources could be appropriate. An example of this was seen in 2019 when the approval of palbociclib to treat women with hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer (MBC) in combination with an aromatase inhibitor or fulvestrant was expanded to include men, supported in part by real-world data from electronic health records and insurance claims.
Finally, the guidance also recommends that when males are included in clinical trials for breast cancer drugs that nonclinical animal studies should be conducted in male and female animals.
Vaping Linked to Higher Risk of COVID-19 in Teen and Young Adults
-Carmine Leggett, PhD
According to the Centers for Disease Control (CDC), the number of middle and high school students using e-cigarettes rose from 3.6 million in 2018 to 5.4 million in 2019—a difference of about 1.8 million youth. Therefore, preventing tobacco product use, particularly that of e-cigarettes, among youth is critical to ending the tobacco epidemic in the United States. A new study led by researchers at the Stanford University School of Medicine, which was published in the Journal of Adolescent Health shows that during the COVID-19 pandemic it is more urgent than ever to end teen and young adult use of e-cigarettes. This recent study is the first to use U.S. population-based data collected during the pandemic to examine associations between teen and young adult vaping and COVID-19.
The researchers used online surveys of 4,351 participants ages 13 to 24 who lived in all 50 U.S. states, the District of Columbia and three U.S. territories. Participants were recruited and divided between those who had used e-cigarettes and those who had never used nicotine products. Study participants were asked whether they had ever used vaping devices or combustible cigarettes, as well as whether they had vaped or smoked in the past 30 days. They were also asked if they had experienced COVID-19 symptoms, received a test for COVID-19 or received a positive diagnosis of COVID-19. The results were adjusted for confounding factors such as age, sex, LGBTQ status, race/ethnicity, mother’s level of education, body mass index, compliance with shelter-in-place orders, rate of COVID-19 diagnosis in the states where the participants were residing, and state and regional trends in e-cigarette use. Results from this study show that among young people who were tested for COVID-19, those who vaped were five to seven times more likely to be infected than those who did not use e-cigarettes. These data also indicate that youth who had used both cigarettes and e-cigarettes in the previous 30 days were almost five times as likely to experience COVID-19 symptoms, such as coughing, fever, tiredness and difficulty breathing as those who never smoked or vaped. Among the participants who were tested for COVID-19, those who had ever used e-cigarettes were five times more likely to be diagnosed with COVID-19 than nonusers. Those who had used both e-cigarettes and conventional cigarettes in the previous 30 days were 6.8 times more likely to be diagnosed with the disease.
The use of e-cigarettes is unsafe for kids, teens, and young adults. Studies show that nicotine is highly addictive and can harm adolescent brain development, which continues into the early to mid-20s. Preventing tobacco product use among youth is critical to ending the tobacco epidemic in the United States, especially amid the COVID-19 pandemic.
Cancer Screenings and Treatment are Still Important During COVID-19
-Nicholas Warren, PhD
Routine screenings for common cancers greatly improve the chances of finding tumors before they spread throughout the body, when therapies are most effective. For example, when colon cancer is diagnosed early, 90.2 percent of patients will survive past 5 years, but only 14.3 percent will survive past 5 years when colon cancer is detected after it has metastasized. Unfortunately, cancer screenings decreased dramatically due to the COVID-19 pandemic. The electronic health record company, Epic, found breast and cervical cancer screenings decreased 94 percent and colon cancer screenings decreased 86 percent in April 2020 compared to prior years. While screening rates rebounded in July 2020, they are still below normal. Norman Sharpless, MD, director of the National Cancer Institute, estimated fewer screenings and delayed treatment will result in almost 10,000 extra deaths per year from colorectal and breast cancers alone by 2030. Understanding the reasons behind decreases in screenings and treatments is important to drive solutions that will save lives.
During AACR’s COVID-19 and Cancer Conference in July 2020, a session on “Cancer Prevention and Early Detection During the COVID-19 Pandemic” discussed factors affecting cancer screening and treatment during the pandemic. The session was chaired by Karen Knudsen, PhD, Director of the Sidney Kimmel Cancer Center and member of the AACR Board of Directors and the AACR COVID-19 and Cancer Taskforce.
Several speakers described how losing health insurance and financial costs are significant burdens for seeking preventative care. Both Lisa Richardson, MD, MPH, Director of the CDC’s Division of Cancer Prevention and Control, and Otis Brawley, MD, Professor of Oncology at Johns Hopkins University, reported that almost 27 million Americans have lost employer-provided health insurance due to the economic effects of the pandemic. Loss of health insurance leads to higher out of pocket costs for all forms of healthcare and lower likelihood of utilizing healthcare. Dr. Brawley was most concerned about patients who self-identified suspicious masses or symptoms of cancer and are delaying follow up screening and care. Dr. Brawley suggested those patients are likely to see worsened prognosis from delays as short as 3 months. Dr. Richardson described how financial issues trickle into small primary care offices: primary care visits have fallen by 50 percent; visits for chronic conditions and wellness exams fell by 60 percent; 39 percent of primary care clinics have laid off or furloughed staff, and; many clinics are worried they may permanently close. The financial issues affecting primary care clinics may impact the availability and access to healthcare well beyond the era of COVID-19.
Another significant roadblock to seeking screening and care during the pandemic is fear of contracting COVID-19 at the doctor’s office. All of the speakers have described efforts to ensure patient safety during cancer screening and treatment, including: separating COVID patients from other patients; dramatic increases in telehealth; no visitor policies; mask requirements, and; many more. Amy Leader, PhD, from Thomas Jefferson University, detailed a survey with 968 cancer survivors that found 40 percent had changes to their doctor appointments and 63 percent were somewhat or very worried about getting COVID-19. Erica Warner, ScD, MPH, from Harvard Medical School, discussed a survey of 534 patients that found approximately 40 percent of respondents were communicating with doctors via video conferencing and telephone; approximately 50 percent of breast cancer survivors in the survey were using these technologies to stay in touch with doctors. Dr. Richardson advised patients to reach out to their providers to see what precautions they are taking, as protocols vary between providers.
While the COVID-19 pandemic poses unprecedented challenges to the cancer community, many hospitals and clinics have adapted to safely provide cancer screenings and treatments. In order to save lives, it is important that these critical services continue. The AACR will continue to do everything we can to advocate for patients with cancer in these difficult times.
Patient Advocate Dialogue: Cancer Disparities
Patients with cancer provide invaluable insight on current issues impacting the cancer research community. The AACR Patient Advocacy Program developed the interview series, Dialogues to showcase unscripted discussions between patient advocates, cancer survivors, researchers, and physicians on current topics.
Dialogue: Cancer Disparities, is a discussion between advocates Dr. Russell J Ledet, co-founder of The 15 White Coats and Candace Henley, founder of The Blue Hat Foundation and colorectal cancer survivor. The Dialogue explores contributing factors to cancer disparities and the impact of cancer disparities on access to care, clinical trials, cancer treatments, and patient outcomes.
SAVE THE DATE: Virtual Patient Advocate Forum: COVID-19 and Cancer
Join AACR and patient advocates from across the cancer community on Thursday October 1, 2020 at 1-3:30 p.m. EDT for the AACR Virtual Patient Advocate Forum on COVID-19 and Cancer. To support AACR’s efforts to keep the cancer community informed and safe from infection, the virtual Forum will be free for all cancer patient advocates who wish to learn more about the state of COVID-19 research.
Moderated by Anna D. Barker, USC chief strategy officer and co-founder of the AACR Scientist↔Survivor Program®, the program will feature:
- Patient perspectives on COVID-19’s impact on cancer care and research.
- Current state of COVID-19 research.
- Development of COVID-19 vaccines and treatments
- Impact on healthcare for patients with cancer
The program will end with an extended live question and answer session to allow attendees to ask questions and share their perspectives. Learn more.
Oncology Approval Recap
Between July 27 and August 26, the U.S. Food and Drug Administration approved two novel oncology therapies and two expanded indications for oncology drugs:
- Atezolizumab was approved in combination with cobimetinib and vemurafenib for patients with BRAF V600 mutation-positive unresectable or metastatic melanoma.
- Tafasitmab-cxix, a CD19-directed cytolytic antibody, was granted accelerated approval in combination with lenalidomide for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant.
- Belantamab mafodotin-blmf was granted accelerated approval for adult patients with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent.
- Carfilizomib and daratumumab were approved in combination with dexamethasone for adult patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy.
Read more about FDA approvals on the AACR webpage and on the AACR 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.