Cancer Policy Monitor: June 12, 2018      

AACR Supports Goal of Eliminating HPV-related Cancers in the United States       

On June 7, the AACR joined 70 National Cancer Institute (NCI)-designated cancer centers and five national cancer organizations in urging increased human papillomavirus (HPV) vaccination and screening access to prevent the human devastation caused by HPV--related cancers. The organizations collectively recognized insufficient vaccination as a public health threat and called upon the nations' physicians, parents and young adults to take advantage of this opportunity to eliminate several different types of cancer in men and women.

According to the Centers for Disease Control and Prevention (CDC), HPV is responsible for almost all cervical cancers, a substantial proportion of head and neck cancers, and the majority of anal, vulvar, vaginal, and penile cancers. CDC data show that approximately 41,000 new HPV-related cancers are being diagnosed in the United States each year, including roughly 17,300 men and 23,700 women. In the United States, the overall annual direct medical cost burden of HPV-related diseases is at least $8 billion.

Since its introduction in 2006, more than 100 million doses of the HPV vaccine have been distributed nationally. The CDC recommends all 11- to 12-year-old girls and boys receive the HPV vaccine. The Department of Health and Human Services, through the Healthy People 2020 initiative, has introduced a goal of reaching 80 percent vaccination rates by the year 2020. However, as of 2016, only 49.5 percent of girls and 37.5 percent of boys in the U.S. had completed the HPV vaccine series, according to the CDC.

"Thanks to decades of research that has produced the HPV vaccine and important screening methods, we have safe and effective tools at our disposal to help eliminate cervical and other HPV-related cancers," said AACR President, Elizabeth M. Jaffee, MD, deputy director of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. "However, we know that these innovative prevention approaches are not reaching all Americans, and that questions remain on how to enable access to these interventions. Eliminating HPV-related cancers will require researchers, health care providers, patients, community leaders, government officials, and scientific society personnel to work together to solve the impediments to implementation."

Research shows there are many barriers to overcome in improving vaccination rates, including a lack of strong recommendations from physicians and many parents not being aware that this vaccine protects against several types of cancer in both men and women.

To begin discussions on how to jointly address these barriers, the AACR joined HPV experts from the nation's top cancer centers, along with partners from the NCI, CDC, the American Cancer Society, and the National HPV Roundtable for a meeting in Salt Lake City in June, where experts discussed ways to collaborate on the goal of eliminating cancers caused by HPV.

At the meeting, participants agreed to an approach of highlighting the progress made thus far with cervical cancer as well as the states and localities that are making great strides in improving HPV vaccination rates. This optimistic framing would be paired with messaging about the urgency of addressing rising HPV-associated head and neck cancer rates in men, in order to highlight the potential impact of further improving HPV-vaccination rates. Broad consensus was reached regarding future collaboration on two specific activities:

  1. Supporting states' inclusion of HPV elimination in their cancer control planning efforts, and
  2. Working toward inclusion of HPV-elimination as an explicit goal of the upcoming Healthy People 2030.

Overall, participating organizations agreed that the time for eliminating HPV-cancer has come and that this goal is achievable with coordinated and persistent action.

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Childhood Cancer STAR Act Becomes Law     

The AACR commends Congress for working in a bipartisan fashion to pass the Childhood Cancer Survivorship, Treatment, Access & Research (STAR) Act of 2018. The STAR Act passed in the House of Representatives May 22 after having passed in the Senate March 22. It was signed into law June 5.

The STAR Act, which had 55 co-sponsors in the Senate and 370 co-sponsors in the House, is the most comprehensive childhood cancer legislation ever passed by Congress. It seeks to improve the lives of pediatric cancer patients and survivors through supporting pediatric cancer research, improving surveillance of childhood cancers, and enhancing resources for survivors.

“This important legislation supports the expanding number of scientific opportunities that exist today to advance pediatric cancer research and develop child-focused cancer treatments, while also helping childhood cancer survivors cope with the long-term impact of their previous treatments,” said AACR President, Elizabeth M. Jaffee, MD, deputy director of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. “For example, the entire field of pediatric cancer research is moving forward because we are currently seeing an ever-growing number of innovative clinical trials specially designed for childhood cancers. We are taking advantage of numerous cutting-edge technologies to get to the root cause of these cancers, and we are realizing the promise of immunotherapy and combination therapies for an expanding number of pediatric cancer indications.”

Among other provisions, the STAR Act:

  • Authorizes the National Cancer Institute (NCI) to expand existing efforts to collect biospecimens for childhood cancer patients enrolled in NCI-sponsored clinical trials and to collect and maintain relevant clinical, biological, and demographic information on all children, adolescents, and young adults with cancer
  • Authorizes grants to state cancer registries to identify and track incidences of childhood, adolescent, and young adult cancers
  • Authorizes grants for pediatric cancer survivorship research and guides the Secretary of Health and Human Services to facilitate the identification of best practices for childhood and adolescent cancer survivorship care
  • Requires that the National Cancer Advisory Board include at least one pediatric oncologist to ensure pediatric cancer research concerns are represented in recommendations to the NCI.

“Finding cures for pediatric cancers is a major priority for the AACR, as is ensuring that childhood cancer survivors are able to overcome many of the late health effects associated with their prior treatments, including secondary cancers and organ failure,” said Margaret Foti, PhD, MD (hc), chief executive officer of the AACR. “During the past few years, the AACR has been especially focused on making an important difference in the lives of children with cancer by bringing together the brain trust of the field to identify actionable steps that will accelerate progress against pediatric cancer.”

The newly passed legislation will complement current efforts of the AACR’s Pediatric Cancer Working Group (PCWG) in accelerating progress for kids with cancer and for childhood cancer survivors. The PCWG and its nearly 3,000 members works to establish childhood cancer research as a global priority supported by improved funding, the very latest technologies, and best educational strategies. The PCWG strives to provide a forum for communication and collaboration among basic, clinical, and translational researchers in academia, industry, and government on all aspects of pediatric cancer research. Additionally, the group serves as an interface, bridging the AACR with advocacy and legislative groups to promote the prevention and treatment of all childhood cancers.

Ongoing PCWG efforts are dedicated to recommending treatment and surveillance practices for childhood cancer predisposition syndromes, supporting the generation, characterization, and distribution of pediatric cancer models, facilitating the development of novel therapeutics for pediatric cancer indications, and advocating for increased research efforts dedicated to pediatric cancer survivorship.

Additionally, the AACR has sponsored Special Conferences in Pediatric Cancer during the past decade that have addressed the unique issues and challenges in investigating the biological basis of childhood cancers and translating recent findings into new treatment approaches. The most recent of these conferences, “Pediatric Cancer Research: From Basic Science to the Clinic,” took place in Atlanta in December 2017. Presentations examined the most recent research on novel strategies for modeling pediatric cancers, genomics and clinical trials, epigenetics, treatment resistance and toxicity, and disease relapse. Technological advances and strategies for translating basic science findings into treatments with a clinical impact, were also featured, including the most up-to-date findings on targeted therapeutics and immunotherapy. 

The AACR is a proud member of the Alliance for Childhood Cancer, which represents more than 25 national patient advocacy groups and professional medical and scientific organizations. The Alliance has been a leader in advocating for the passage of this legislation.

In addition, the AACR wishes to recognize the leaders in Congress whose diligent work has led to this important legislation, including the original co-sponsors of the bill: Senators Jack Reed (D-Rhode Island), Shelley Moore Capito (R-West Virginia), Chris Van Hollen (D-Maryland), and Johnny Isakson (R-Georgia); and Representatives Michael McCaul (R-Texas), Jackie Speier (D-California), Mike Kelly (R-Pennsylvania) and G.K. Butterfield (D-North Carolina).

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Right-to-try Bill Signed into Law      

Providing potentially lifesaving investigational drugs to terminally ill patients has long been a concern for patient and government groups. The U.S. Food and Drug Administration (FDA) has facilitated access to investigational drugs since the late 1980s. Since then, improvements have been made to this expanded access program through legislation, guidance, and collaboration on projects like the Expanded Access Navigator.

In recent years, the FDA has received over 1,000 applications annually to treat patients with investigational therapies. The agency authorizes greater than 99 percent of these requests, usually within hours or a few days depending on the level of emergency. While the FDA approves almost every application they receive, part of the current application process requires physicians to first request the use of an investigational therapy from that therapy’s sponsor. At this stage of the application, drug sponsors may choose not to provide a requested treatment.

Despite the improvements and high rate of approvals under the FDA's expanded access process (also called "compassionate use"), there has been a recent push by some organizations and advocates to pass "right-to-try" laws allowing terminally ill patients to bypass the FDA in seeking experimental treatments. Such laws have been passed in 40 states, and President Trump as well as many members of Congress prioritized federal right-to-try legislation this year.

In August of 2017, the U.S. Senate passed a right-to-try bill sponsored by Sen. Ron Johnson (R-WI). The House later passed an amended version of this legislation which limited the scope of patients who would qualify for the program and included other measures intended to protect patient safety. Ultimately, the Senate failed to pass this version of the legislation, so the House considered the original Senate bill and passed it May 22. President Trump signed the bill into law May 30. 

Under the legislation, terminally ill patients who have exhausted FDA-approved treatment options and cannot participate in a clinical trial may request access to an experimental treatment from the manufacturer, with their doctor's approval. As with the expanded access process, the manufacturer may approve or deny such an application.

Now that right-to-try legislation has become law, the FDA must determine how to implement it. FDA Commissioner Scott Gottlieb has stated that the agency remains committed to making sure patients are "adequately protected."

"We're committed to the underlying goals here of trying to get promising products to patients who are out of options," said Gottlieb, while speaking at a recent event in Washington, D.C. In a statement on the law’s signing he said, “Our implementation of the Right to Try Act will build on our long-standing efforts to help patients and families who are facing life-threatening diseases or conditions, in a way that seeks to protect their autonomy, their safety, and the safety of others following in their paths. The decisions we reach related to products that can serve as an effective treatment for a terminal illness, or that can arrest a devastating and debilitating condition, are among the most important and carefully considered judgments that we make. We recognize the important balance between making sure patients have the assurances Congress intends, while enabling timely access to promising treatments in these devastating circumstances. And we’ll implement this new law consistent with these longstanding values.”

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Appropriations Update from Capitol Hill      

NIH Appropriations

In late May, the Appropriations Committees in both the House and Senate approved the Fiscal Year (FY) 2019 allocations for each of their subcommittees. These amounts, referred to as 302b allocations, specify the total amount of funding that an Appropriations subcommittee can designate for all of the agencies and programs under the subcommittee’s jurisdiction.

Appropriations for the National Institutes of Health (NIH) fall under the Subcommittee on Labor, Health and Human Services (HHS), Education and Related Agencies. In the House, this subcommittee received an allocation equal to FY 2018 levels, while its Senate counterpart received an allocation that is approximately $2 billion higher than the FY 2018 level.

The House Appropriations Subcommittee on Labor-HHS-Education will hold the “mark up” of its spending bill soon, with the Senate Subcommittee to follow later in June. At that time, more information will be available regarding the specific allocations to agencies including the NIH. The AACR, along with many other organizations, is advocating for NIH funding of at least $39.3 billion for FY 2019. This reflects an increase of at least $2 billion for base NIH funding, in addition to funding designated under the 21st Century Cures Act in FY 2019 for specific initiatives including the Beau Biden Cancer Moonshot.

FDA Appropriations

In mid-May, the full House Appropriations Committee approved the FY 2019 Agriculture, Rural Development, Food and Drug Administration (FDA) and Related Agencies spending bill. The bill provides a robust increase of $308 million to the base budget of the FDA for a total funding level of $3.1 billion. This includes $20 million for the FDA Oncology Center of Excellence (OCE), a $5 million increase above FY 2018 that aligns with the AACR’s request.

In late May, the Senate Appropriations Committee also advanced its FY 2019 Agriculture, Rural Development, FDA and Related Agencies spending bill. This bill provides the FDA base budget with a 5 percent increase above current levels ($2.97 billion) and fully funds the Oncology Center of Excellence at $20 million. The Agriculture-Rural Development-FDA spending bills will likely be considered as part of an appropriations package in both the House and Senate.

The AACR continues to closely monitor report language in these bills related to the regulation of tobacco products. An amendment to the House version includes language that prohibits the FDA from regulating large and premium cigars under the Deeming Rule. However, the same amendment also includes language that is supportive of the FDA’s efforts to regulate electronic cigarettes, including a requirement to issue a proposed rule on multiple standards for vaping products, including flavors. The Senate version of the spending bill did not include such language.

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Capitol Corner: AACR Interviews Members of Congress      

Every month, the AACR will be interviewing several members of Congress from both sides of the aisle to get their personal story and views on cancer research. This month, we interviewed Senator Johnny Isakson (R-Georgia) and Congressman Joseph Crowley (D-New York).

Read our previous interviews with Senator Jon Tester (D-Montana), Representative Dave Reichert (R-Washington), Senator Gary Peters (D-Michigan), Representative Charlie Crist (D-Florida), Senator Susan Collins (R-Maine), Representative Dwight Evans (D-Pennsylvania), Senator Chuck Grassley (R-Iowa), Senator Tammy Baldwin (D-Wisconsin), Representative Brett Guthrie (R-KY) and Representative Suzan DelBene (D-WA).

Senator Johnny Isakson (R-Georgia) 

Can you share with our readers, many of whom are cancer survivors, advocates, and researchers, your personal connection to cancer?

We have all been touched somehow by cancer. I am personally a skin cancer survivor. I have had very dear friends, family and colleagues who have valiantly fought the disease, with some who have beat their cancer and others who have lost the battle. I have of course heard from countless advocates and worked with many to advance the fight against various forms of cancer.

Many of their stories are heart wrenching. It takes incredible strength, willpower and help from caregivers, family, friends and neighbors, no matter the ultimate outcome in each case.

How has the experience of dealing with cancer in your community, both personally and from stories you’ve heard from your constituents, shape your views as a policymaker and a public official?

Anytime you have been personally affected by a disease or an event, it makes a difference in your basic understanding of it. The stories I’ve heard from so many Georgians and Americans are often very personal and always extremely helpful as my aides and I work to understand how our policy decisions will affect outcomes.

I have taken on a number of policy positions and legislative initiatives dealing specifically with cancer, and others affecting patients who have multiple chronic conditions, because I understand the gravity of the disease.

It also makes a difference that the Centers for Disease Control and Prevention (CDC) is in my home state. There are a number of government and advocacy organizations that have helped increase my knowledge about cancer and other health-related issues.

What would you say to your colleagues in the legislative branch about the role of federal investment in medical research and cancer research in our nation?

The 21st Century Cures Act that was signed into law in 2016 is a great example of what the federal government can do to help the medical and research communities when we are able to work together. There were a number of provisions in this bill that I worked on for several years that are already paying off. This bipartisan effort is spurring development of medical treatments and cures as well as supporting the success of hospitals and research universities. I would suggest that when we work together, we can accomplish important things.

How can groups like the AACR and patient advocates best communicate the importance of medical research to the members of Congress? Do you think we have made progress in terms of raising awareness of the importance of National Institutes of Health (NIH) funding to saving lives and helping the American economy?

Patient advocacy groups should continue to meet with members and staff, sharing stories and with them and supporting legislative initiatives are important. It is always helpful for advocacy groups to coordinate efforts as much as possible so that you are speaking with one voice.

Americans are great advocates for the issues they care most deeply about, so keeping them informed and engaged through your own programming and through the media is worthwhile. I believe we have made progress, and that there is yet more progress to be made. Although there is widespread support in Congress for devoting resources to NIH, it is also important to consider the role of CDC and other key agencies that help save lives and fight disease.

Can you tell us more about other efforts—legislation and otherwise—that you have worked on in support of better prevention, detection and treatment of cancer?

As co-chair of the Senate Cancer Coalition, and as a member of the Senate Committee on Health, Education, Labor and Pensions and the Committee on Finance, which share jurisdiction over the American health care system, there are a number of legislative initiatives I’ve introduced or directly supported.

I mentioned the 21st Century Cures Act previously. That bill included my Advancing Hope Act, a three-year extension of the Food and Drug Administration (FDA) Priority Review Voucher program for rare pediatric diseases, including childhood cancer, to continue to spur investment in the development of cures for these diseases. It will ultimately help make lifesaving treatments available more quickly through an expedited review timeline. In August, the Food and Drug Administration (FDA) approved the first cell-based gene therapy available in the U.S. The approval came thanks to the Advancing Hope Act.

I am a proud co-sponsor of the bipartisan Childhood Cancer Survivorship, Treatment, Access, Research (STAR) Act, which would advance childhood cancer treatments through research and program development.

I also worked on the Sunscreen Innovation Act, which requires the Food and Drug Administration (FDA) to speed up the review process for new and innovative sunscreen ingredients and help clear the bureaucratic hurdles that have stood between American consumers and the newest sunscreen products. Many of these products are already available in many other countries and would better protect our skin from cancer causing UV exposure.

I have introduced Senate resolutions that have passed, including resolutions designating May of each year as "Melanoma Awareness Month" and also as "National Cancer Research Month."

The AACR is the world’s first and largest organization dedicated to every aspect of high quality cancer research. The AACR has 40,000 members across all states, as well as members in over 119 other countries. Do you have anything you would like to say to the AACR and our scientists and physicians who have dedicated their careers to making progress against cancer?

I, and more importantly, millions of Americans and individuals worldwide, appreciate your dedication. Countless individuals have benefited from the progress you have helped make in advancing research as we continue working to find cures.

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Representative Joseph Crowley (D-New York)  

Can you share with our readers, many of whom are cancer patients, advocates, and researchers, your personal connection to cancer?

Like millions of Americans, my family and I have lost a loved one to cancer. In 1985, my Uncle Walter passed away after a brave fight against the disease. I grew up looking up to Walter. He taught me that we have a responsibility to look out for one another and is one of the reasons I decided to dedicate my life to public service. This was a painful experience and one that reinforced my admiration for all those courageous patients fighting against the disease.

How has the experience of dealing with cancer in your community, both personally and from stories you've heard from your constituents, shape your views as a policymaker and a public official?

My wife Kasey, a nurse, and I are both active with the Congressional Families Cancer Prevention Program, a bipartisan effort to increase the general public's understanding and awareness of cancer prevention, early detection, and healthy living. This experience has taught how critical early detection and preventive health care are and has provided me a better understating of the importance of investing as a nation in cancer research to address curative treatments, and preventive efforts to stop cancer in its tracks.

You have supported efforts to provide the National Institutes of Health (NIH) with the largest increases in funding in more than a decade. Why do you believe that robust funding increases for NIH are so important at this time? What would you say to your colleagues in the legislative branch who may not understand as you do how important federal investment in medical research is to our nation?

I believe that the work of the NIH is invaluable toward the national goal of understanding and defeating many illnesses. Thanks to investments in the NIH and the Cancer Moonshot Initiative, we have been able to make advancements in chemotherapy and immunotherapy and reduce the rate of cancer mortality.

At a time when cancer continues to be the second-leading cause of death in the country, we must continue investing in research to treat and prevent all forms of cancer and other diseases. This issue has broad-based bipartisan support; both Democrats and Republicans in Congress believe in the value of the NIH and have resisted the Trump administration proposed harmful cuts to the agency.

How can groups like the AACR and patient advocates best communicate the importance of medical research to the members of Congress? Do you think we have made progress in terms of raising awareness of the importance of National Institutes of Health (NIH) funding to saving lives and helping the American economy?

The AACR and patient advocates have done a tremendous job communicating the importance and value of medical research with members of Congress. My advice is to continue what you're doing. Share your personal stories on how you or your loved ones have benefited from cancer research. You are the voice and face of our national investment in the NIH, and it's important for us in Congress to learn exactly where that investment goes and how it impacts the lives of millions of cancer patients. 

Can you tell us more about other efforts, legislation and otherwise, that you have worked on in support of better prevention, detection, and treatment of cancer?

As a New Yorker, I have worked hard to honor the sacrifices of the thousands of 9/11 first responders and survivors by championing the James Zadroga 9/11 Health Act, which established the World Trade Center Health Program. A number of these survivors contracted cancer in the aftermath of 9/11 and this program provides affordable medical and cancer treatments and financial compensation those exposed to toxins at Ground Zero, the Pentagon, and Shanksville, Pennsylvania.

Also, I'm a co-sponsor of the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act of 2017. No child should ever die from cancer, that's why I'm proud to support the most comprehensive legislation addressing pediatric cancer. This bill would authorize the NIH to collect data and medical information from children, reauthorize the national childhood cancer registry for states tracking childhood cancers, and allow the agency to support research on outcomes for pediatric cancer survivors in medically underserved populations.

Do you have anything you would like to say to the AACR and our scientists and physicians who have dedicated their careers to making progress against cancer?

Thank you to all the AACR members, scientists, and physicians who devote their lives and careers to help Americans prevent and overcome this disease. Your contributions and innovations give hope to millions of cancer patients and their families and provides us with better tools to diagnose, treat and prevent cancer. I'm proud of your work, and I'm proud to be an ally of the AACR in finding cures together.

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USPSTF Updates Recommendations on PSA-based Tests for Prostate Cancer Screenings    

In May, the U.S. Preventive Services Task Force (USPSTF) updated its recommendations on the use of prostate-specific antigen (PSA)-based tests for prostate cancer screenings in adult men who have no symptoms or a previous diagnosis of prostate cancer. For men aged 55-69, the USPSTF now recommends that the decision to undergo periodic PSA-based screening should be made by individuals after discussing the possible benefits and harms with their doctor.

In its previous recommendation published in 2012, the USPSTF had recommended against PSA-based prostate cancer screening. After reviewing evidence published since 2012, the task force updated its recommendation in a paper published in the Journal of the American Medical Association (JAMA).

"Before deciding whether to be screened, men should have an opportunity to discuss the potential benefits and harms of screening with their clinician and to incorporate their values and preferences in the decision," the recommendation states. "In determining whether this service is appropriate in individual cases, patients and clinicians should consider the balance of benefits and harms on the basis of family history, race/ethnicity, comorbid medical conditions, patient values about the benefits and harms of screening and treatment-specific outcomes, and other health needs."

For men aged 70 and older, the USPSTF continues to recommend against using PSA-based screenings, where the harms likely outweigh the benefits. The task force did not make any specific recommendations for African-American men or men with a family history of prostate cancer, but it encouraged further research on these populations who are at higher risk of prostate cancer.

"[Additional research is needed in] screening for and treatment of prostate cancer in African-American men," the USPSTF statement reads. "Given the large disparities in prostate cancer mortality in African-American men, this should be a national priority."

According to the USPSTF, some of the risks associated with PSA-based screening for prostate cancer include "false-positive results that require additional testing and possible prostate biopsy; overdiagnosis and overtreatment; and treatment complications, such as incontinence and erectile dysfunction."

Aside from additional research into the effectiveness of PSA-based screenings in higher-risk populations, the USPSTF also lists several areas where more research is needed to improve screening for and treatment of prostate cancer, such as:

  • Comparing different screening strategies, including different screening intervals, to fully understand the effects on benefits and harms;
  • Developing, validating, and providing longer-term follow-up of screening and diagnostic techniques; and,
  • Refining active prostate cancer treatments to minimize harms.

The published final recommendations are available here.

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Regulatory Science and Policy Subcommittee Welcomes New Chair Kenneth C. Anderson, MD    

The American Association for Cancer Research is pleased to welcome Kenneth C. Anderson, MD, as the new chair of the Regulatory Science and Policy Subcommittee of the Science Policy and Government Affairs Committee. The Regulatory Science and Policy Subcommittee is dedicated to bringing together and providing an open forum for stakeholder engagement and is actively engaged in the development and implementation of programmatic and policy initiatives to improve the development, evaluation, and regulation of cancer drugs, biologics, and diagnostics.

Anderson is the Kraft Family professor of medicine at Harvard Medical School as well as the director of the Lebow Institute for Myeloma Therapeutics and Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute. Anderson was inducted as a fellow of the AACR Academy in 2015 and has been an active leader in the AACR for many years serving as a member of the AACR board of directors from 2012-2015 and as editor-in-chief of Clinical Cancer Research from 2007-2017. He served as the 2017 president of the American Society of Hematology.

Perhaps most notably, Anderson transformed medical practice and markedly improved outcomes for multiple myeloma patients worldwide. His research emphasizing the role of the tumor microenvironment in carcinogenesis led to the preclinical and clinical development of the proteasome inhibitor bortezomib and the immunomodulatory drug lenalidomide. These agents, used in combination as front-line treatments, more than doubled the median survival time of patients with multiple myeloma. His lab also aided in the advancement of the monoclonal antibodies, elotuzumab and daratumumab, which are now used in the treatment of some multiple myeloma patients who have relapsed or are not eligible for stem cell transplants. Anderson’s experience shepherding novel therapies through clinical trials and the FDA-approval process and overall leadership will be invaluable to fulfilling the subcommittee’s goal of advancing regulatory policy to the benefit of cancer patients.

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Watch Live: FDA-AACR-SGO Drug Development in Gynecologic Malignancies Workshop    

On Thursday, June 14, the FDA, AACR and Society of Gynecologic Oncology are cosponsoring the Drug Development for Gynecological Malignancies workshop at the FDA Campus in White Oak, Maryland. Registration is open to view and participate in the workshop live via online webcastBoth the agenda as well as the speaker bios are available. The below guest post by Gwynn Ison, MD, medical officer, U.S. Food and Drug Administration, first appeared on the Cancer Research Catalyst, the official blog of the AACR.

Like medical oncology as a whole, the field of gynecologic oncology has undergone a remarkable transformation in recent years with the approval of bevacizumab and three PARP inhibitors for patients with cervical and ovarian cancers, respectively.

However, gynecologic malignancies continue to be a major cause of morbidity and mortality in the United States, resulting in an unmet medical need. In particular, ovarian cancer is usually diagnosed at an advanced stage, with no more than 20 percent of women being cured following initial treatment, which includes surgery and conventional platinum-based chemotherapy. It is the fifth leading cause of death in women in the U.S.

Endometrial and cervical cancers are gynecologic cancers that are treatable when diagnosed at an early stage, but for women with advanced or recurrent disease, the efficacy of available therapies is poor, and patients ultimately die from their disease within a matter of months. Rare gynecologic tumors such as low-grade serous carcinomas of the ovary continue to represent an area lacking in therapeutic options as well.

Given the recent advances in oncology, including the promise of immunotherapy and biomarker-driven approaches in multiple tumor types, it stands to reason that these successes in other tumors could be realized in the treatment of rare and advanced gynecologic malignancies. The potential for novel combinations and trial designs in the treatment of gynecologic cancers exists, but the approach needs to be well thought out.

To continue to improve clinical outcomes for patients with gynecologic malignancies, the U.S. Food and Drug and Drug Administration (FDA), the American Association for Cancer Research (AACR), and the Society of Gynecologic Oncology (SGO) have come together to co-sponsor a workshop to address furthering drug development in this area. Bringing together key stakeholders in the field of gynecologic oncology including industry, regulatory agencies, patient advocates, and academic oncologists, we seek to build on the discussions and progress initiated at the 2015 FDA-AACR-SGO-ASCO workshop focused on ovarian cancer endpoints.

The goal of this year’s workshop is to broaden our scope and engage in robust discussions on the most practical aspects of drug development including developing strong rationale for combination therapies and how to appropriately focus when dealing with rare malignancies that have low patient populations. The FDA-AACR-SGO Workshop on Drug Development in Gynecologic Malignancies will take place  June 14, 2018, at the FDA’s White Oak campus in Silver Spring, Maryland.

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FDA-AACR Workshop: Non-clinical Models for Safety Assessment of Immuno-oncology Products    

Date: Sept. 6, 2018. 
Location: Marriott Wardman Park, 2660 Woodley Rd NW, Washington, D.C. 20008

Purpose: Discuss current challenges and opportunities for non-clinical models to predict toxicities of immune checkpoint inhibitors and stimulators in patients. The workshop will provide a forum for academic, industry, and regulatory experts to discuss developing non-clinical models that are both predictive and practical for human safety assessment.

Background:  The development of non-clinical models that can predict toxicities of immune checkpoint inhibitors and stimulators in patients is an extremely important and timely issue for the cancer community. There are 50 agents targeting PD-1 or PD-L1 in clinical development, and more than 1100 trials combining anti-PD-1/L1 agents with other therapies, yet retrospective analyses indicate that animal toxicity for many of these agents is minimal and does not predict adverse effects in patients. In addition, the recent observation of worse overall survival in two randomized trials that evaluated anti-PD-1/L1 agents in combination with immunomodulatory drugs underscores the need for cross-sector collaboration in this area. With advances in nonclinical models to study the pharmacodynamics of immune checkpoint inhibitors and stimulators, the question remains whether any of these models could be adapted to assess the safety of immuno-oncology products.

Register for in-person or webcast participation.

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NGS Oncopanels Session Discusses CMS National Coverage Determination    

On March 16, 2018, the Centers for Medicare and Medicaid Services (CMS) issued a final decision memo on “Next Generation Sequencing (NGS) for Medicare Beneficiaries with Advanced Cancer.” The memo stated that "CMS will provide coverage for U.S. Food and Drug Administration (FDA) approved or cleared companion in vitro diagnostic tests that specify treatment options for patients with recurrent, relapsed, refractory, metastatic, or advanced stages III or IV cancer that has either not been tested previously with the same NGS test or for repeat testing using the same NGS test when a new primary cancer diagnosis is made by the treating physician." The CMS decision memo for this National Coverage Determination (NCD) is available.

At the 2018 Annual Meeting in Chicago, Illinois, the AACR presented a Regulatory Science and Policy Session called "NGS Oncopanels: Regulatory Considerations" to discuss NGS tests, their role in cancer diagnosis and treatment, and the implications of the National Coverage Determination. A free webcast of this session is available. Available webcast presentations include Reena Phillip, PhD, and You Li, PhD, from the FDA; Yali Li, PhD, from Foundation Medicine; and Ahmet Zehir, PhD, from Memorial Sloan-Kettering Cancer Center. The panel discussion also included perspectives and insight from CMS.

As discussed during the recent NGS Oncopanels session, the final NCD removed non‐coverage language and allows regional Medicare Administrative Contractors (MACs) to determine coverage of other NGS tests, not approved by the FDA, so long as patients meet the same coverage criteria listed above. The non-coverage language found in the draft decision memo would have meant any test not approved by the FDA would not be covered by CMS or its affiliates. This change relieved some concerns of medical centers who believed the draft decision would effectively prevent them from ordering tests not approved by FDA. Patient groups were also pleased that the final decision allowed for repeat testing coverage in certain circumstances and expanded the eligible patient criteria compared to the draft NCD.

Certain concerns remain for stakeholders, including that locally determined coverage for tests not approved by FDA could lead to regional variations in standards or patient access. The final decision also removed some requirements for data collection, also known as Coverage with Evidence Determination (CED), which were included in the draft NCD and would have allowed FDA approved or cleared tests without a companion diagnostic indication to be covered so long as either a) administrators of those tests collected certain data on the use and effectiveness of their test and enrolled patients in a registry to track patient outcomes or b) the test was a part of a NIH-NCI National Clinical Trial Network clinical trial. Not collecting this data could mean losing an opportunity to develop new insights into cancer.

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Register Today: Rally for Medical Research 2018    

Registration is now available for the sixth annual Rally for Medical Research, which will be held Sept. 12-13, 2018, in Washington, D.C. For those who cannot participate in person, the online Rally National Day of Action will take place Sept. 13.

We have reserved a block of rooms at the Rally headquarters hotel at a negotiated rate. Contact to make a reservation at the lower rate.​​

Learn more about 2018 Hill Day Sponsorship Opportunities​​. Stay up-to-date on registration information by visiting the Rally website, Facebook, and Twitter pages.

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