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Cancer Policy Monitor: April 10, 2018      

Key Policy Updates from Capitol Hill      

Congress passed and the President signed an omnibus appropriations bill for Fiscal Year 2018. The spending bill provides $37.084 billion for the National Institutes of Health, a $3 billion increase over current levels (8.8%), and a full $1 billion above the amount approved last summer by the Senate Appropriations Committee.

Congress Passes Final Spending Bill for FY 2018 with Large Increases for NIH, NCI

Late last month, just before another deadline, Congress passed a long-awaited omnibus appropriations bill for Fiscal Year (FY) 2018 that was subsequently signed into law by President Trump.  The final funding level for the NIH and NCI was outstanding news for medical research advocates.  The spending bill provides $37.084 billion for the National Institutes of Health, which is a $3 billion increase over current levels (8.8%), and a full $1 billion above the amount approved last summer by the Senate Appropriations Committee.

This NIH total includes the following:

  • The full $496 million designated by the 21st Century Cures Act in FY 2018, which includes $300 million for the Beau Biden Cancer Moonshot to the NCI;
  • A base funding level of $5.665 billion for the National Cancer Institute (NCI). When adding the Moonshot funds, the total funding level for the NCI is $5.965 billion, close to a 6% increase over FY 2017 enacted levels.
  • A provision requiring NIH to continue reimbursing grantee research institutions for facilities and administrative costs.
  • $12.6 million for the Gabriella Miller “Kids First” pediatric cancer research initiative.


A significant part of the $1 billion in additional funds for the NIH is for opioid research (a total of $500 million), split evenly between NINDS and NIDA.

Also of note, the new FDA Oncology Center of Excellence (OCE) was funded for the first time at a level of $15 million, which is aligned with what the AACR has advocated. 

The increase for NIH is a tremendous win for everyone who values medical research and would not have been possible without the hard work and dedication of advocates like you.  Your meetings, calls, emails, letters, tweets, and posts are making a difference, and our collective voice grows stronger by the day.  From everyone at the AACR, thank you for all that you do to advocate for cancer research.  Congratulations and let’s keep the momentum going!

STAR Act

The Childhood Cancer STAR Act passed the Senate unanimously in late March.  The STAR Act is the most comprehensive pediatric cancer bill ever considered by Congress. The bill now moves to the House, where over 80% of members are signed on as co-sponsors. For more information, see this press release from the Alliance for Childhood Cancer. 

Presidential Rescission Could Roll Back Federal Spending

President Donald Trump may resort to a procedural tool known as a presidential rescission in order to cut federal dollars that Trump has already signed into law. When the $1.3 trillion spending bill, H.R. 1625, was passed by Congress, Trump initially threatened to veto it but then signed it into law. Now, Trump administration officials are discussing the possibility of a rescission package with House Majority Leader Kevin McCarthy (R-Calif.), a procedure that has not been used in 25 years. Rescission would provide for government-wide spending cuts while requiring only a simple majority in the House and Senate.

Despite requiring only a simple majority, the use of rescission would still be politically difficult. Currently, it remains unknown which parts of the federal budget would be cut through rescission. The Trump Administration’s FY 2018 budget, which Congress ignored, called for severe cuts in several areas, including the National Institutes of Health (NIH). NIH has significant bipartisan support in Congress and it is unlikely that Congress would support a sharp spending cut to the agency after having approved a large increase in the FY 2018 omnibus.

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Regulatory Science and Policy Sessions at the AACR Annual Meeting 2018     

The Regulatory Science and Policy track provides an opportunity to interact with representatives from the FDA, industry, and academia. These informative sessions are designed to highlight recent regulatory developments and provide an open forum for the discussion of cutting-edge issues in cancer drug, biologic, and diagnostic regulation.

NGS Oncopanels: Regulatory Considerations

Saturday, April 14, 2018 | 10:15 AM - 12:15 PM
Room S401bcd (Level 4)

Reena Philip (Chair) - FDA, Silver Spring, MD

FDA has approved NGS tests in oncology. This education session will go over the requirements for analytical and clinical performance for the various intended uses.

Biosimilars: Biological Science, Regulatory Science, and Clinical Practice

Saturday, April 14th, 2018 | 1:00 PM – 3:00 PM
Room S105 (Level 1)

Sara A. Hurvitz (Chair) - UCLA, Santa Monica, CA

This session will provide an in-depth discussion of the preclinical development pathway of biosimilars, taking into account the biologic complexity of these agents compared to small molecule drugs. A detailed overview of the FDA regulatory statute and guidance on biosimilar production including key policy concepts and reimbursement considerations will be provided. Finally, the clinical development for biosimilars will be thoroughly examined.

Cancer Genomic Reference Samples - Sequencing Consortium Results and Beyond

Saturday, April 14, 2018 | 1:00 PM - 3:00 PM
Room S401bcd (Level 4)

Zivana Tezak (Chair) - FDA, Silver Spring, MD

Clinical oncology is being transformed by the use of next-generation sequencing (NGS) based diagnostics. This new technology can enable the rapid identification of potentially significant genetic variations across nearly the entire genome. However, lack of agreed upon well-characterized and community-validated reference samples and data benchmarks creates a potential challenge for development and understanding of NGS results. To address this challenge, a consortium of 60+ institutions and 160+ scientists have come together to characterize a triple-negative breast cancer cell line HCC1395, and its B lymphocyte derived normal cell line HCC1395BL as a community resource. The consortium applied machine learning algorithms to define high confidence mutation calls with datasets from several NGS platforms. The consortium is operated under the FDA-led Sequencing Quality Control Phase II (SEQC2) program. This educational session will go over the results from this effort, discussing the first release of high-confidence germline variants and somatic mutations on this breast cancer cell line pair, tumor purity benchmarking efforts, and false discovery investigation.

Has Pandora's Box Been Opened? The Site Agnostic Approval of Pembrolizumab

Sunday, April 15, 2018 | 1:00 PM - 2:45 PM
Room S401bcd (Level 4)

Steven J. Lemery (Chair) - FDA, Silver Spring, MD

On May 23, 2017, FDA approved pembrolizumab for the treatment of unresectable or metastatic, microsatellite instability-high (MSI-H) or mismatch repair deficient (MMRd) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options. The potential clinical benefit of checkpoint inhibition in these patients offers the potential for hope contained in Pandora’s box. Nevertheless, numerous questions and issues have arisen based on the pembrolizumab approval.

Scientific and Regulatory Challenges in Development of CAR-T Therapy for Solid Tumors

Monday, April 16, 2018 | 10:30 AM - 12:15 PM
Room S401bcd (Level 4)

Ke Liu (Chair) - FDA, Silver Spring, MD

The U.S. Food and Drug Administration has recently approved two CAR-T therapies for patients with hematological malignancies. These approvals represent an apex of intensive scientific and clinical research on this novel approach combining gene, cell and immunotherapy for the treatment of patients with hematological malignancies. However, the role of CAR-T therapy for solid tumor is less clear. This session will provide an overview of the recent advances and discuss scientific and regulatory challenges related to the clinical development of CAR-T therapy for solid tumors.

Real World Evidence in Oncology and its Implications

Monday, April 16, 2018 | 3:00 PM - 5:00 PM
Room S401bcd (Level 4)

Amy P. Abernethy (Chair) - Flatiron Health, New York, NY

Real-world evidence (RWE) is clinical evidence generated from datasets including electronic health records, administrative claims data, patient-reported data, and genomics/biomarker data when collected in the routine provision of care. We will examine a sample set of use cases in which “regulatory-grade” RWE is being utilized to improve and accelerate treatment development and patient care including: informing clinical trial design and feasibility, developing external control arms to support single-arm interventional trials, supporting regulatory submissions for label expansions, satisfying post marketing safety commitments, and generating evidence to support Health Technology Assessments.

Precision Therapy: When Is Better: Up Front or at Relapse?

Monday, April 16th, 2018 | 5:00 PM – 6:30 PM

Room S406 (Vista Ballroom - Level 4)

Gideon Blumenthal (Chair) - FDA, Silver Spring, MD

Precision therapy describes efforts to provide cancer patients treatment based on specific genetic mutation(s) contributing to a tumor, regardless of the cancer’s tissue of origin. In 2017, the U.S. Food and Drug Administration approved nine drug indications and two gene therapies designed to treat patients based on specific cancer mutations. Additionally, thirteen previously approved drugs received expanded indication approval based on precision therapy. In this cross-disease forum, leading scientists will engage in a broad discussion on the application of precision therapy in cancer. Discussion will focus on whether precision medicine should be utilized as a first line therapy or reserved for second line therapy following relapse.

Recently Approved Breakthrough Therapies and New Approval Endpoints

Stay tuned to the online program for recently approved drugs featured in this session.
Tuesday, April 17, 2018 | 10:30 AM - 12:15 PM
Room S401bcd (Level 4)

Ashley F. Ward (Chair) - FDA, Silver Spring, MD

The Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 gave the U.S. Food and Drug Administration (FDA) statutory authority to designate medical products as “breakthrough therapy” if the therapy treats a serious of life-threatening disease or condition and preliminary evidence indicates that the drug may demonstrate substantial improvement over existing therapies. This session will highlight the approval process for several recently approved breakthrough therapies and new approval endpoints and the impact that accelerated drug access is having on patient treatment in these disease areas. Drugs to be discussed in this session are: tisagenlecleucel and axicabtagene ciloleucel, midostaurin, apalutamide, and blinatumomab.

FDA Priorities: A Conversation with Deputy Commissioner Anna Abram

Tuesday, April 17, 2018 | 1:00 PM - 1:45 PM
Room S401bcd (Level 4)

Anna Abram - FDA, Silver Spring, MD

The U.S. Food and Drug Administration’s Deputy Commissioner for Policy, Planning, Legislation and Analysis, Anna Abram, will share the vision and goals of Commissioner Scott Gottlieb for how the Agency will harness the potential for new therapies and technologies to improve patient wellbeing in the coming years. Among the Commissioner’s goals for the Agency are improving the efficiency of approval processes, promoting generic drug competition, empowering patients to make smarter decisions about their health, and ensuring the FDA has a strong expert workforce to maintain the high standards of the Agency. The discussion will likely explore how health care in the United States is changing as a result of breakthroughs in cancer research, address the range of challenges faced by patients, and share how patients and advocates can help contribute to the FDA’s goals.

Implications of the 2017 FDA Reauthorization Act (FDARA) on Pediatric Cancer Drug Development

Tuesday, April 17, 2018 | 3:00 PM - 5:00 PM
Room S401bcd (Level 4)

Gregory H. Reaman (Chair) - FDA, Silver Spring, MD

This particular symposia will feature a diverse panel of stakeholders, including individuals from academia, government, and industry, as well as from patient advocacy organizations, to discuss the implications of the 2017 FDA Reauthorization Act (FDARA) on Pediatric Cancer Drug Development. Specifically, the RACE for Children Act was included in the 2017 FDA Reauthorization Act (FDARA) to support the early evaluation of potentially effective drugs by requiring pediatric investigation of appropriate new drugs intended for adults with cancer. This session will assess the promise of precision medicine in pediatric oncology while also examining the practical considerations of implementing the provisions included in FDARA, including considering the implications for global development and international collaboration.

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Science Policy Sessions at the AACR Annual Meeting 2018     

The AACR Annual Meeting 2018, taking place April 14-18 in Chicago, will include several events organized by the Office of Science Policy and Government Affairs. These include sessions in the Regulatory Science and Policy track and a town hall meeting on how the AACR is working for you in Washington. Click on each session title below to learn more.

How the AACR is Working for You in Washington: A Town Hall Meeting on Cancer Research Funding and Science Policy Initiatives

Sunday, April 15, 2018 | 3:00 PM - 4:30 PM
Room S401bcd - McCormick Place South (Level 4)

George D. Demetri (Chair) - Dana-Farber Cancer Institute, Boston, MA

The AACR works tirelessly to advocate for cancer research funding and science-based policies that affect cancer researchers and patients, from bench to bedside. This interactive session, moderated by the chair of the Science Policy & Government Affairs Committee, will feature a diverse panel of stakeholders and representatives to discuss AACR’s numerous initiatives in government relations and science policy. In addition to sharing more about current efforts to support cancer research funding and advance science for the benefit of researchers, cancer patients and the public at large, panelists will answer your questions about what the AACR is doing to support cancer research and biomedical science in Washington, DC., and hear your perspective on how the AACR can further leverage its expertise and that of its members.

The Cancer Survivorship Landscape: Potential Focus Areas for the Future

A special session in the memory of Jimmie C. Holland, MD
Sunday, April 15, 2018 | 5:00 PM - 6:30 PM
Room W190 - McCormick Place West (Level 1)

Anna D. Barker (Moderator) - Arizona State University, Scottsdale, AZ

Currently, there are over 16 million cancer survivors in the U.S. and this number is projected to rise to over 20 million within the next decade. This statistic underscores how the efficacy of oncology treatment has improved, as today there are now many more individuals living with their disease in remission or as a chronic condition than at any other time in history. In this session, panelists representing the entire continuum from cancer research to cancer care and patient advocacy will discuss and address issues affecting cancer survivors, including the psychosocial and lifestyle aspects of cancer survivorship, concerns regarding the development of second malignancies, and challenges such as comorbidities and the toxicities resulting from current and prior treatments. In addition, the state of current cancer survivorship research for improving the quality of care that cancer survivors receive will be examined.

A Town Hall Discussion of AACR's Priorities in Tobacco Policy

Monday, April 16, 2018 | 1:00 PM - 2:45 PM
Room S401bcd - McCormick Place South (Level 4)

Roy S. Herbst (Chair) - Yale Cancer Center, New Haven, CT

This interactive town hall session will discuss important topics in tobacco policy such as: the July 2017 announcement by U.S. Food and Drug Administration Commissioner, Dr. Scott Gottlieb, that the Agency will begin to explore ways of lowering the nicotine content of combustible cigarettes as part of their strategy for addressing the devastating addiction crisis that is threatening American families; the focus on addressing the continued high rate of tobacco use by rural, poor, and other marginalized populations; a review of local tobacco sale laws which would increase the minimum purchase age from 18 to 21; and the revision of cigarette warning labels to plain packaging that includes explicit health warnings.

NCI Director's Address & Fireside Chat with AACR Leaders

Monday, April 16, 2018 | 3:00 PM - 3:45 PM (This session will be livestreamed at Facebook.com/AACR.org)
Room W190 - McCormick Place West (Level 1)

Norman E. Sharpless - National Cancer Institute, Bethesda, MD
Fireside Chat
Michael A. Caligiuri - City of Hope National Medical Center, Duarte, CA
Elizabeth M. Jaffee - Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD

During this first major public address from the new Director of the National Cancer Institute, Dr. Norman E. Sharpless will outline his roadmap and goals for the institute. Following a brief presentation, Dr. Sharpless will be joined by AACR President, Dr. Michael A. Caligiuri, and AACR President-Elect, Dr. Elizabeth A. Jaffee, for a discussion. Questions from the audience may be submitted by email to NCIchat@aacr.org.

Biden Cancer Initiative Colloquium

Tuesday, April 17, 2018 5:00 to 6:30p.m.
Room S401bcd - McCormick Place South (Level 4)

Greg Simon - Biden Cancer Initiative, Washington, DC
Elizabeth M. Jaffee - Johns Hopkins Kimmel Comprehensive Cancer Center, Baltimore, MD

Join us during the AACR Annual Meeting for a special colloquium moderated by Gregory Simon, President of the Biden Cancer Initiative, featuring panelists who will discuss the status of their Cancer Moonshot efforts. Elizabeth M. Jaffee, MD, 2018-19 AACR President, will provide opening remarks.

Access to Health Care in the Era of Biologically Targeted Therapies

Wednesday, April 18, 2018 | 10:15 AM - 12:00 PM
Room N427 - McCormick Place North (Level 4)

Sara A. Hurvitz (Chair) - UCLA, Santa Monica, CA
Targeted therapies for cancer offer the promise of improved outcomes with lower morbidity, but also pose a danger that disparities will arise from differences in access to increasingly costly and complex treatments. In the first talk of the session, evidence of disparities in breast cancer subtypes that benefit from targeted therapies will be presented and specific data regarding differences in treatment by race and age will be reviewed. Possible interventions to improve disparities in the delivery of some these novel therapies for breast cancer will also be previewed. The second speaker will present the current state of disparities by race and class in lung cancer mortality, and will discuss issues surrounding the lack of data relating to access to care for patients of color, and those with low socioeconomic status. The final speaker will present several far-reaching solutions, focusing on the FDA, Medicare and clinicians, to promote access and innovation while reducing price growth for targeted cancer drugs.

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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 Gary Peters (D-Michigan) and Representative Charlie Crist (D-Florida).

Read our previous interviews with 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 Gary Peters (D-Michigan)  

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

A number of years ago, my mother was diagnosed with breast cancer. While I am fortunate to say my mom is a survivor and turned 93 this year, I have heard from too many Michiganders who have lost loved ones to cancer.

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?

Cancer is more than just a statistic: to millions of Americans, it is all too personal. I've heard the heartbreaking stories of children who lose a parent at far too young an age, spouses who lose a loved one far too soon and patients whose lives are turned upside down in an instant after receiving the devastating prognosis. Those are stories I take close to heart – and why I believe firmly that we must build on the groundbreaking cancer research done everyday to save families from enduring such hardships in the future.

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? 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?

We are living in an exciting time where new discoveries are revolutionizing how we approach cancer diagnosis, treatment and recovery – and that is a testament to the vital federal investments made at institutions like the NIH. For example, NIH-sponsored clinical trials are finding new drugs like trastuzumab hat can attack cancer cells more effectively than chemotherapy alone – and reducing the risk of reoccurring breast cancer by as much as 40 percent. Breakthroughs in gene editing technology like CRISPR offer even greater potential to treat cancer more precisely than current techniques.

With further research, this technology has the promise to eventually prevent cancer altogether by eliminating the disease at the genetic level. Continued strong federal funding for science research will ensure that these groundbreaking innovations can continue. 

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?

Advocates play an important role in bringing issues related to cancer research directly to policymakers, and it is absolutely vital for families impacted by cancer to educate their lawmakers about the need for strong medical research funding. As a member of the bipartisan NIH Caucus, I support increased federal investments in health research and have consistently called for robust funding of the NIH and the National Cancer Institute.

In March 2018, I voted in favor of a $3 billion increase to NIH funding, which follows a $2 billion increase the previous year. While major pieces of legislation, such as the 21st Century Cures Act, have helped bolster awareness and support for cancer research, we must continue supporting advanced medical research as new discoveries and breakthroughs emerge.

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

Last January, President Obama signed into law the American Innovation and Competitiveness Act – bipartisan legislation I introduced to update federal research and development policies, maximize basic research opportunity, and reduce administrative burdens for researchers.

I also support a number of bipartisan, commonsense bills dedicated to cancer treatment and diagnoses. The Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act, which recently passed in the Senate, would help support children diagnosed with cancer, particularly those suffering from the late effects of their treatment or disease.

The Removing Barriers to Colorectal Cancer Screening Act would waives coinsurance costs for colorectal cancer screenings under Medicare, raise awareness for screenings and support patients who do not have full coverage for a screening colonoscopy.

I have also consponsored the Firefighter Cancer Registry Act to better collect data on instances of cancer amongst firefighters, which is the leading cause of firefighter line-of-duty deaths in the United States. This data will help researchers understand the links between firefighting and cancer, and offer a stepping stone to find preventive actions and possible treatments for these public servants.

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?

Thank you for all you do. You are at the forefront of innovations and breakthroughs that directly impact on millions of lives and bring us closer to a cure for cancer. Please know that my door is always open, and I am committed to working with the AACR to increase awareness and strengthen support for cancer research.

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Representative Charlie Crist (D-Florida) 

You lost a family member to cancer. Can you share with our readers, many of whom are cancer patients, advocates, and researchers, more about your personal connection to cancer? What would you say to a family that is dealing with a cancer diagnosis of a loved one?

The most important thing is to leave no words left unsaid. When my sister Margaret was diagnosed with a brain tumor, we knew that her prognosis was tough. We wanted to make the best of every moment as a family, and I believe we did. 

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?

I've learned that often the best-informed advocates are patients, survivors, and families. When you sit down and listen to folks about what they're going through and what they need, they have a better understanding of what policies would benefit them, their situation, or their community than any lawmaker or lobbyist in Washington. We have to listen, and then we have to act.

In your first year in Congress, you supported a $2 billion funding increase to the National Institutes of Health (NIH) budget and opposed proposed cuts to the NIH. 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?

Any cuts to medical research handicaps our scientists and doctors, moving us backwards in our efforts to find more effective treatments and cures to serious medical conditions.

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?

It's important to show up where we live – in our districts. Nothing is more impactful than hearing directly from your neighbors on how a group, program, or issue is impacting their lives directly, impacting our community.  

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

Access to cancer detection, including procedures such as prostate exams and mammograms along with affordable treatment options, are a matter of life and death. This is why I continue to call for Medicaid expansion in my home state of Florida and have been fighting to protect the Affordable Care Act. I also recently called for increased funding for the National Institutes of Health and the Defense Department's medical research program. Congress must continue to prioritize funding for innovative treatments and new methods of detection.

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 for all you have done – and continue to do – to help those impacted by cancer, which affects millions of Americans regardless of age, racial background, gender, or geographical location. It is life-changing work that we all should be grateful for and support.

Is there anything we didn't discuss that you would like to add?

As the younger brother of a sister lost to cancer, this is an issue that touches me personally. I will continue to do all I can to support the work of those leading in the fight against cancer for Margaret and all others who have been affected by it.

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New FDA Approval Endpoint: Minimal Residual Disease      

On March 29th, the U.S. Food and Drug Administration (FDA) granted accelerated approval to blinatumomab to treat children and adults with minimal residual disease (MRD)-positive B-cell precursor acute lymphoblastic leukemia (ALL). ALL is a rare indication, with about 6000 new cases diagnosed yearly in the United States. MRD is the presence of cancer cells below levels that can be observed by a microscope, now measured by techniques including flow cytometry and next generation sequencing. For patients in remission, MRD can mean an increased chance for relapse. While MRD is a factor in all forms of leukemia, this approval marks the first time an MRD-targeted treatment has been approved and signals a shift in how industry and the Agency look at treating these cancers.

During a recent Oncologic Drugs Advisory Committee (ODAC) meeting in early March, questions were raised on what specific level of MRD is appropriate to use as a threshold to determine for which patients pre-emptive therapy should be considered. The study used to seek approval included patients with MRD ≥ 0.1%, but this cutoff level is determined primarily by the technical ability to reliably measure it. ODAC participants suggested that if lower levels can be measured more accurately in the future then the threshold for this treatment should likewise be lowered.

ODAC members were asked to vote whether they thought the presented trial data demonstrated a potential clinical benefit for ALL patients with MRD above the 0.1% threshold. While the overall vote was supportive (8 in favor, 4 against), four ODAC members voted ‘No’ to this question. Though MRD can serve as an indicator to guide treatment, concerns were raised over confounding data from subsequent treatments, such as bone marrow transplants, in the trial and were not convinced that meaningful clinical benefit was definitively shown. Vassilliki Papadimitrakopoulou, MD, explained her “No” vote, reasoning, “although I think there is potential benefit, and I think there is plenty of data, and a clear benefit for this patient to have some therapy in the setting of MRD, I do not feel that we have the exact definition of the popula­tion that benefits.”

Ongoing confirmatory trials will provide clarity on these concerns but ALL has a small patient population and these trials may take time to complete. Some of the involved studies may not end until 2020. Despite some uncertainty, this new approval endpoint is still important. This accelerated approval will provide patients in danger of relapse a treatment option while these ongoing studies assess long term survival of MRD-positive patients and other factors that might affect treatment success. As Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence said, “Because patients who have MRD are more likely to relapse, having a treatment option that eliminates even very low amounts of residual leukemia cells may help keep the cancer in remission longer.”

Attendees of the AACR Annual Meeting 2018 can hear directly from the FDA, and reviewer Emily Jen, MD, PhD, about this ground-breaking new approval endpoint and other recent breakthroughs at the Regulatory Science and Policy track session “Recently Approved Breakthrough Therapies and New Approval Endpoints,” taking place on Tuesday, April 17th, from 10:30am – 12:15pm in room S401bcd – McCormick Place South. Symposia of the Regulatory Science and Policy track provide attendees with an opportunity to interact with the FDA, National Cancer Institute, industry, and academia, including basic researchers and physician scientists. These informative sessions are designed to highlight recent regulatory developments and provide an open forum for the discussion of cutting-edge issues in cancer drug, biologic, and diagnostic regulation.

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Register Today: FDA-AACR-SGO Drug Development in Gynecologic Malignancies Workshop    

Date: June 14, 2018. Time: 8:00 a.m. - 5:00 p.m.

Gynecologic malignancies continue to be a major cause of morbidity and mortality in the United States, resulting in an unmet medical need. The ongoing revolution in oncology that has offered many new therapies to patients with a variety of cancer types has yet to reach these women. With the notable exception of the approvals of bevacizumab, and PARP inhibitors in ovarian cancer, we need to bring increased efforts to bear to spur the development of new treatment.

Goals and Objectives:

  • To provide a forum for open discussion between the FDA, clinicians, laboratory experts, and researchers on the way forward for diagnosis and treatment of gynecologic malignancies;
  • Accelerate the development of immunotherapy in gynecologic malignancies;Facilitate open discussions among all parties in the area of gynecologic malignancies;
  • Obtain input from multiple stakeholders on approaches to novel study designs to leverage rare subtypes in gynecologic malignancies; and
  • Discuss the impact of biomarkers in gynecologic malignancies.

Learn more about the workshop. Register for in-person or webcast participation.

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Save the Date: Rally for Medical Research 2018    

The sixth annual Rally for Medical Research 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.

Stay up-to-date on registration information by visiting the Rally website, Facebook, and Twitter pages.

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