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Cancer Policy Monitor: January 12, 2021

Appropriations Update from Capitol Hill

-Marc B. Johnson, MPP

After months of negotiations between the White House and congressional leadership, Congress has concurrently passed a $900 billion COVID-19 relief package in addition to omnibus funding legislation. The omnibus funding bill which consists of all 12 FY21 appropriations bills, is needed to keep the government operating through the end of September 2021. In the omnibus, the National Institutes of Health (NIH) will receive $42.9 billion, a $1.25 billion increase from the 2020 enacted level. Of the aforementioned amount, $6.56 billion will be allocated to the National Cancer Institute (NCI), an increase of $120 million over the 2020 enacted level. NCI’s FY21 total includes $195 million in Cancer Moonshot funding from the 21st Century Cures Act, which passed in 2016. House Appropriations Committee Chairwoman Nita Lowey, who is retiring at the end of this session, stated she was “…pleased that we are concluding this Congress with a bipartisan agreement to provide the certainty of full-year funding for all of government and urgently-needed coronavirus relief to save lives and livelihoods.”

The COVID-19 relief package, the second of two enormous relief packages this year, will primarily address the needs of small businesses, vaccine distribution, the enhancement of unemployment benefits, and sending out stimulus checks to individuals and households, among other needs. NIH will receive $1.25 billion “…to support research and clinical trials related to the long-term effects of COVID-19, as well as continued support for Rapid Acceleration of Diagnostics for COVID-19.” The AACR and the broader medical research community have been advocating for research relief funding and will continue to do so in the 117th Congress as Congress considers additional supplemental bills. In a joint statement, House Speaker Nancy Pelosi and Senate Minority Leader Chuck Schumer said the relief package (as well as the omnibus package) will deliver “…urgently needed funds to save the lives and livelihoods of the American people as the virus accelerates.” Senator Majority Leader Mitch McConnell stated on the Senate floor that the relief package is “…targeted toward our fellow Americans who need help the most.”   

Below are links to the full bill text and section by section summaries of both the COVID relief package and FY21 Appropriations. H.R. 133 Full Bill Text, (NIH FY21 language begins on page 963) FY21 Appropriations Section by Section Summary, COVID-19 Relief Package Section by Section Summary.

AACR COVID-19 and Cancer Taskforce Recommends Prioritizing Vaccines for Patients with Cancer

-Nicholas Warren, PhD

This December, the U.S. Food and Drug Administration (FDA) granted Emergency Use Authorizations to two vaccines for COVID-19. These incredible milestones provide light at the end of the tunnel for the devastating COVID-19 pandemic. However, supplies of the vaccines will remain limited for several months. This scarcity poses important questions of who should be prioritized for vaccination. In the United States, individual states and territories have ultimate authority over which of their residents are prioritized.

Also in December, the American Association for Cancer Research’s (AACR) COVID-19 and Cancer Taskforce published a perspective article in Cancer Discovery recommending patients with cancer be prioritized for COVID-19 vaccination. The recommendation is supported by more than two dozen studies finding patients with cancer have roughly double the risk of experiencing severe symptoms and dying from COVID-19 than patients without cancer, even after accounting for other co-morbidities like obesity or smoking history. The risk is especially pronounced for patients with blood cancer and patients on active anti-cancer treatment. Very few patients with cancer have been included in randomized clinical vaccine trials for COVID-19 thus far. Therefore, it is important to further elucidate the safety and efficacy of vaccination in this vulnerable population through additional research.

In October 2020, the AACR also organized a Virtual Patient Advocate Forum to discuss the impact of COVID-19 on patients with cancer and progress on therapies and vaccines to treat COVID-19. Avrum Spira, MD, global head of the Lung Cancer Initiative at Johnson and Johnson, and member of the AACR COVID-19 and Cancer Task Force, discussed the lead COVID-19 vaccines being developed, including Johnson & Johnson’s vaccine. He relayed that the current phase III clinical trials are not designed to measure safety and efficacy in patients with cancer, but hopefully additional trials will study subpopulations of vulnerable patients after the first authorizations are granted. Dr. Spira also encouraged patient advocates to speak up and demand transparency and inclusion in vaccine trials. The speakers encouraged patients to discuss specific concerns about vaccines with their doctors.

The AACR will continue to support patients with cancer by providing timely and trusted information about the pandemic and advocating for policies that will benefit their well-being. As part of this commitment, all COVID-19-related articles in AACR journals are available for free on the COVID-19 and Cancer Resource Center.

The Future of Telemedicine in Cancer Care Post-COVID-19

– Tod Guidry, PhD

The COVID-19 pandemic has escalated the implementation of telemedicine, the remote provision of health care services, across the landscape of cancer care. The use of telemedicine has removed substantial barriers to accessing care and improved both patient and provider satisfaction, opening the door to its more optimized application to cancer care in the future. An article published in Clinical Cancer Research highlights key priorities in optimizing telemedicine utilization toward improving the lives of cancer patients post-COVID-19.

Even short periods of disruption in care for cancer patients can result in significant increased mortality risk. The challenges and dangers of in-person cancer care brought on by the COVID-19 public health emergency this year have thus led to an escalated implementation of telemedicine, particularly virtual visits via phone or video and remote patient monitoring. Several National Cancer Institute (NCI)-designated cancer centers across the nation have reported significant increases in telemedicine utilization over the past year. Some centers have reported as many as 30 to 40 percent of cancer patient visits occurring via telemedicine, with reports of high levels of satisfaction among patients and providers. However, key challenges in access to telemedicine and questions around its impact on patients remain, representing opportunities for optimizing future telemedicine use in cancer care.

Of particular concern is the need to mitigate inequalities in access to telemedicine services that could exacerbate already existing cancer health disparities. Durable solutions to the geographic digital divide must be devised to overcome the ~40 percent gap in high speed Internet access among rural and tribal areas, compared to urban. A better understanding of the disparities in digital health literacy among patients of low socioeconomic status, racial/ethnic minorities, and advanced age is also needed to develop solutions toward more equitable telemedicine utilization. Additionally, many patients, despite having access to the technology required for telemedicine, face challenges in accessing the necessary software. The article advocates for the development of a streamlined portal for accessing telemedicine services, compatible with multiple operating systems. Oncology care teams may also need to develop strategies to improve patient knowledge and skills for telemedicine use.

The pandemic has also escalated the use of telemedicine for cancer patients enrolled in clinical trials. Changes to clinical trial conduct necessitated by the pandemic have included remote informed consent, virtual follow-up visits, and home delivery of medical products. The impacts of telemedicine utilization on patient access to and participation in clinical trials should be examined, and positive changes should be more permanently adopted in the conduct of clinical trials in the future.

The impact of telemedicine on clinical outcomes, patient-reported outcomes, and health care utilization measures will need to be tracked, including via wearable devices already seeing increased utilization within cancer care. Gathering longitudinal data on patients utilizing telemedicine could be used to improve its effectiveness. In addition to outcomes, efforts to better understand the impact of telemedicine on patient and provider satisfaction are warranted, including any negative impacts from the loss of in-person encounters.

The use of telemedicine in cancer care delivery throughout the COVID-19 pandemic has provided a path forward toward innovative means of improving access to cancer care. However, challenges remain in ensuring equitable access to telemedicine and gaining a fuller understanding of its impact on patient outcomes and satisfaction. The cancer community stands tasked with optimizing telemedicine’s implementation and ensuring its effectiveness in improving the lives of all cancer patients.

FDA’s Project Renewal Establishes a Framework for Updating Outdated Cancer Drug Labeling

– Tod Guidry, PhD

Project Renewal was launched in October 2018 by the U.S. Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) with the goal of updating outdated labeling for off-patent cancer drugs. Since its launch, the initiative has built a set of procedures by which decisions on drug labeling revisions are made by leveraging scientific expertise through a collaboration with the AACR. An article recently published in Clinical Cancer Research details the process developed by which Project Renewal fosters appropriate regulatory decision-making around potential labeling updates for cancer drugs.

FDA labeling provides scientifically rigorous information on the safe and effective use of medical products used to treat cancer patients. While new information including new usages and dosing regimens become available over time, drug companies have few incentives to revise drug labeling in the post-marketing setting. However, surveys of oncology professionals conducted through Project Renewal, with engagement facilitated by organizations including the AACR, have demonstrated trust in the scientific integrity of FDA-approved product labeling and a reliance on the information therein in caring for patients.

Project Renewal has begun with the identification and prioritization of a list of marketed oncology drug products based on their importance in current clinical practice and time since initial approval, prioritizing those having spent 15+ years on the market. FDA has contracted Deloitte Consulting, LLC, to form external scientific teams in collaboration with the AACR to identify potential off-label uses or dosing regimens that may need to be included in labeling updates for the prioritized drugs.

The AACR also supports the assembling of research team members (RTMs), consisting of healthcare providers across oncology sub-specialties and clinical pharmacologists, in addition to other scientific experts. RTMs review and evaluate the evidence that may support the inclusion of off-label uses, dosing regimens, or other pharmacologic information in revised product labeling. After meetings between FDA, Deloitte team members, and RTMs to evaluate the evidence and address regulatory requirements over several months, the Project Renewal team produces a drug-specific Product Report summarizing the team’s evaluation of the evidence supporting labeling considerations.

The Product Report is submitted to FDA along with considerations for proposed labeling revisions and serves as a resource for the agency in deciding whether labeling updates are warranted. If so, the FDA will send draft labeling to the appropriate drug company, recommending it be included in a requested supplemental marketing application.

Project Renewal is a collaborative effort leveraging the expertise of the oncology community to support updates to outdated cancer drug product labeling. This initiative represents an important step forward in improving public health, by maximizing the utility of labeling for cancer drugs as a crucial resource for oncologists prescribing them. Looking ahead, lessons learned during the pilot phase of Project Renewal will be used to optimize the current procedures, with the ultimate goal of a standardized process for evaluating scientific information in support of necessary cancer drug labeling updates.

Apply for the AACR Annual Meeting Virtual Scientist↔Survivor Program®

The AACR is now accepting applications for cancer survivors and patient advocates to enroll in the Virtual 2021 AACR Annual Meeting Scientist↔Survivor Program®. 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.

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 virtual special interest sessions about cancer research that are tailored to a lay audience;
  • Discuss relevant and timely cancer topics during Zoom meetings with scientific mentors;
  • Communicate to scientists the key issues, questions, and concerns of the survivor and patient advocacy communities;
  • Showcase their programs on a digital poster in the virtual Exhibitor Hall and on the AACR patient advocacy webpage.
  • Attend the AACR 2021 Virtual Annual Meetings scientific sessions and access on demand sessions at no cost.

Participants are accepted through a competitive application process. The deadline to submit an application is January 25, 2021. Please share this announcement with cancer survivors and patient advocates with an interest in cancer research and policy. Learn more.

Project Livin’ Label

In November, the American Association for Cancer Research, in collaboration with the U.S. Food and Drug Administration released the first episode of Project Livin’ Label, a new educational initiative that aims to foster broad understanding of specific oncology product labels and increase awareness of recent oncology drug FDA approvals among physicians, cancer patients and survivors, industry representatives, and others.

For each oncology product to be featured as part of this initiative, the FDA’s Oncology Center of Excellence (OCE) will moderate a panel discussion to include the FDA reviewer(s), a clinical trial investigator from academia, a patient who has benefitted from the therapy, and a representative from the company that developed the drug. This interaction about the product’s development and FDA’s review process will provide essential information for the safe and effective use of the product.

The first two episodes featuring tucatinib and pemigatinib are available now.

Learn more about Project Livin’ Label and tune-in.

Youth E-Cigarette Use Levels Remain High in 2020

-Nicholas Warren, PhD

Tobacco use is responsible for over 480,000 deaths every year in the United States. Since 1965, there has been a steady decline in tobacco use from almost half of the adult population to only 14 percent in 2019. The youth e-cigarette epidemic threatens to reverse decades of progress in tobacco use rates by addicting a new generation to nicotine and increasing the likelihood youth will progress to smoking tobacco.

This September, the CDC released new data on youth tobacco use rates from the 2020 National Youth Tobacco Survey (NYTS). The NYTS found that the rate of high school students who currently used e-cigarettes declined from 27.5 percent in 2019 to 19.6 percent in early 2020. While these results are promising, there is concern confounding factors could have impacted honest responses to the NYTS, such as: changes in e-cigarette regulations and corresponding use patterns; increased public awareness and stigma associate with vaping, and; an emerging global pandemic.

The Monitoring the Future (MTF) survey is a separate effort to measure youth use of tobacco and other drugs since 1991. This December, results of the 2020 MTF survey were published in JAMA Pediatrics. In 2019, the MTF survey found 22.5 percent of 12th and 10th grade high school students used e-cigarettes in the past 30 days. In 2020, 21.8 percent used e-cigarettes, which was not statistically different from 2019 rates. The discrepancies in use rates between the 2020 NYTS and MTF could be due to several factors, including: MTF only surveyed 10th and 12th graders, compared to the NYTS surveying all high school grade levels; shortened 2020 MTF data collection due to the COVID-19 pandemic, and; differences in willingness of students to respond truthfully to an academic or government-sponsored survey. The MTF survey also found a significant decrease in the ease of obtaining nicotine-containing e-cigarette liquids; 92 percent of respondents said it was easy to obtain nicotine-containing liquids in 2019 compared to 83 percent in 2020. This finding suggests increased regulations like increasing the minimum legal age to purchase tobacco products has made it more difficult to buy nicotine-containing products.

Despite the discrepancies in short-term trends, both the NYTS and MTF surveys suggest that approximately one fifth of high school students are actively using e-cigarettes; this translates to over 3 million high school users. While e-cigarettes have lower levels of carcinogens compared to combustible tobacco, they still pose significant health risks, such as: interfering with brain development; raising blood pressure; reducing immune function, and; causing inflammation that promotes cancer cell growth. The AACR will continue to advocate for evidence-based policies and engage with policy makers to reduce all tobacco use.

Most Favored Nation Rule Could Threaten Access to Cancer Drugs for Patients on Medicare

– Tod Guidry, PhD

On November 20, the Trump Administration published the Most Favored Nation (MFN) Rule, establishing a new model to decide Medicare Part B payment rates for certain drugs. According to the rule, a new methodology will be implemented to calculate reimbursements for drugs administered by health care providers at outpatient settings, based on discounts received by other countries for the same products. The model will be implemented over the course of four years starting with a cohort of 50 drugs/biologics that compose a high percentage of Medicare Part B spending, more than half of which are used to treat cancer.

While the MFN model is an effort by the administration to reduce Medicare expenditures, the rule is receiving pushback over concerns it would cut access to critical cancer therapies for Medicare beneficiaries. Indeed, the rule itself states that “a portion of the savings is attributable to beneficiaries not accessing their drugs through the Medicare benefit, along with the associated lost utilization.” Cutting Medicare reimbursement rates for outpatient drugs would force providers to decide between paying the difference or discontinuing offering the products to their patients.

Critics also point out the differences in drug development, regulatory, and pricing systems in other countries compared to the United States. Medicare patients may lose access to novel cancer therapies, which are often approved first in the United States, that are not yet approved elsewhere. Also, while the model represents an effort to coax pharmaceutical companies to lower drug prices, it is unclear whether the cost burden would simply be shifted onto privately insured patients.

In addition to its prospective impacts on access to Part B drugs, critics argue that the rule is an unconstitutional attempt to rewrite a reimbursement system laid out in statute and exceeds the authority granted to the Center for Medicare and Medicaid Innovation outlined in the Affordable Care Act. The Trump administration’s Department of Health and Human Services is now facing multiple legal challenges over the MFN Rule, filed by pharmaceutical and biotechnology industry groups, healthcare professional groups, and cancer patient advocacy organizations.

Strategies to Increase HPV Vaccination Rates are Cost Effective

-Nicholas Warren, PhD

The Human Papillomavirus (HPV) is a very common virus that is responsible for more than 35,000 cases of cancer in the United States every year. Fortunately, several HPV vaccines are available that effectively prevent infection and subsequent cancer diagnoses. The vaccines are most effective if patients are vaccinated before the age of 17 years. However, in 2019 only 56.8 percent of U.S. girls aged 13-17 years old and 51.8 percent boys were fully vaccinated against HPV. In 2019, the AACR joined over 80 other organizations in a call to eliminate all HPV-related cancers, in part by vaccinating 80 percent of teenagers. State-level vaccination mandates for other deadly diseases, like measles, have been effective strategies to increase vaccination rates. Unfortunately, similar mandates for HPV vaccination have faced strong political opposition and only Hawaii, Rhode Island, Virginia, Puerto Rico, and the District of Columbia have successfully passed HPV vaccination mandates. These hurdles have led to increased research and attention to innovative strategies to increase vaccination without mandates.

A recent cost-effectiveness study found that innovative strategies to increase HPV vaccination at the state level were highly cost effective compared to treating subsequent HPV-related cancers. The study, published in Pediatrics analyzed three initiatives to improve HPV vaccination rates, such as: centralized reminder and recall systems to remind patients and/or their parents about vaccination; in-school vaccination clinics, and; quality improvement visits at primary clinics to help improve implementation of best practices for vaccination. The study incorporated efficacy and cost data from randomized clinical trials on each intervention and epidemiology data on incidence of HPV-related illness and current vaccination rates from national databases. The authors then estimated the cost per HPV-related cancer prevented by each intervention. All three interventions were found to be significantly less expensive than standard treatment for HPV-related cancer. For example, the cost to treat patients with cervical cancer in just the first year following diagnosis is approximately $50,000. Vaccination clinics in schools were estimated to be the most effective at increasing HPV vaccination rates, and had the second highest cost at $17,691 per HPV-related cancer prevented. Reminder and recall systems were second most effective at increasing vaccination rates and had the highest cost per HPV-related cancer prevented at $28,333. Quality improvement visits to primary care clinics led to the lowest increase in vaccination, but only cost $1,475 per HPV-related cancer averted. The results of this study demonstrate that innovative state-led strategies to increase HPV vaccination could be powerful tools to help save patients from a devastating cancer diagnosis and the resulting costs.

Save the Dates: AACR Virtual Patient Advocate Forums 2021

Mark your calendars! The dates for the 2021 AACR Virtual Patient Advocate Forums have been released. Patient advocates from across the cancer community join these free virtual events to get the latest information on cancer research, new discoveries, and potential cancer treatments. Speakers include patient advocates, researchers, and clinicians. Learn More.

Oncology Approval Recap

Between November 24 and December 20, the U.S. Food and Drug Administration granted one accelerated approval for an existing therapy and approved new companion diagnostic indications.

  • The Sonalleve MR-HIFU system was approved for the treatment of osteoid osteoma in the extremities.
  • Pralsetinib was approved for adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy or RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).
  • Margetuximab-cmkb was approved in combination with chemotherapy, for the treatment of adult patients with metastatic HER2-positive breast cancer who have received two or more prior anti-HER2 regimens, at least one of which was for metastatic disease.
  • Selinexor was approved in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.
  • The first oral gonadotropin-releasing hormone (GnRH) receptor antagonist, relugolix, was approved for adult patients with advanced prostate cancer.
  • Osimertinib was approved for adjuvant therapy after tumor resection in patients with non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test.

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.