September 2017

The ASPHO Advocacy Brief

Welcome to the Advocacy Brief, a quarterly offering from the American Society of Pediatric Hematology/Oncology. The goal of this e-newsletter is to inform members about legislative and regulatory issues impacting the profession of pediatric hematology/oncology and the patients we serve.

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Special Features

ASPHO Bill Tracker

Visit ASPHO’s Legislative Tracking Chart to see a list of bills that ASPHO is monitoring.

Childhood Cancer STAR Act Update

From the Alliance for Childhood Cancer

House: We now have 60% of the House signed on to the STAR Act. We have 261 representatives signed on (109 Republicans and 152 Democrats). If we can sign on 10 more Republicans, we will have a majority of the Republican Caucus on board. Fortunately, we already have 80% of the Democratic Caucus signed on.

Senate: In the Senate, we now have 21 senators signed on (11 Democrats and 10 Republicans.) An additional nine Democratic senators have asked to sign on but we are waiting for additional Republicans to join before we add them.

Status of Negotiations

In the Senate, the staff of Senators Capito and Reed have been meeting with the Senate Health, Education, Labor, and Pensions (HELP) Committee to negotiate some of the details of the STAR Act before the Senate moves the bill to a mark-up and then to the floor for a vote. HELP Chairman Lamar Alexander (R-TN) has pledged to move the STAR Act though the Senate this year.

As you know, the House approved the STAR Act in December. Since this is a new Congress, the House will have to approve the bill once again. Congressman McCaul has already discussed the STAR Act with House Energy and Commerce Chairman Walden. They are discussing the possibility of moving the STAR Act through the House in September for Childhood Cancer Month. We will report on more details of these negotiations as they become available.

How can ASPHO members get involved?

Contact these key senators and ask them to support the STAR Act:

• Bob Corker (R-TN)
• Joni Ernst (R-IA)Deb Fischer (R-NE)
• Cory Gardner (R-CO)
• Dean Heller (R-NV)
• Ron Johnson (R-WI)
• Lisa Murkowski (R-AK)
• Rob Portman (R-OH)
• Pat Roberts (R-KS)
• Tim Scott (R-SC)
• Pat Toomey (R-PA)

There are currently 18 Republicans and 15 Democrats in the House that cosponsored the STAR Act last Congress but have not signed back on yet (despite repeated requests). We need to reach out to the following to ask why they have not signed back on:

• Adams, Alma (D-NC)
• Bilirakis, Gus (R-FL)
• Bishop, Mike (R-MI)
• Buchanan, Vern (R-FL)
• Bucshon, Larry (R-IN)
• Burgess, Michael (R-TX)
• Castro, Joaquin (D-TX)
• Collins, Chris (R-NY)
• Crawford, Rick (R-AR)
• Cuellar, Henry (D-TX)
• Davis, Danny (D-IL)
• Dent, Charles (R-PA)
• Doggett, Lloyd (D-TX)
• Esty, Elizabeth (D-CT)
• Fortenberry, Jeff (R-NE)
• Hill, French (R-AR)
• Hudson, Richard (R-NC)
• Johnson, Eddie Bernice (D-TX)
• Lee, Barbara (D-CA)
• Jackson Lee, Sheila (D-TX)
• Lynch, Stephen (D-MA)
• Murphy, Tim (R-PA)
• Reed, Tom (R-NY)
• McMorris Rodgers, Cathy (R-WA)
• Meadows, Mark (R-NC)
• Meeks, Gregory (D-NY)
• Meng, Grace (D-NY)
• Neal, Richard (D-MA)
• Paulsen, Erik (R-MN)
• Rokita, Todd (R-IN)
• Ruppersberger, C.A. (D-MD)
• Sanford, Mark (R-SC)
• Schrader, Kurt (D-OR)

RACE Act Signed into Law

The Research to Accelerate Cures and Equity (RACE) for Children Act was signed into law on August 18, 2017, as part of the Food and Drug Reauthorization Act. The RACE for Children Act modifies the Pediatric Research Equity Act by requiring companies developing cancer drugs to conduct studies in children when the molecular target of their drug is relevant to a children’s cancer. Read a press release from Kids v Cancer.

Take Action on the Deferment for Active Cancer Treatment Act

Contact your representative and ask him or her to support the Deferment for Active Cancer Treatment Act (H.R. 2976), which allows for the deferment of certain student loans during a period in which a borrower is receiving treatment for cancer and 6 months after such period.

ASPHO Concerned with Proposed Changes to Medicaid

ASPHO joined other organizations in a letter to Senate leadership outlining concerns about potential changes to the fundamental structure and purpose of Medicaid.

ASPHO Supports Expanded Access Principles

As policymakers debate changes to the way in which unapproved therapies are accessed, ASPHO agrees that changes should adhere to certain principles.

2017 Childhood Cancer Awareness Month Activities

Sept 13–14: Rally for Medical Research
Sept 14: Congressional Childhood Cancer Caucus
Sept 14: Alliance Luncheon and Art Show (call for exhibits below)
Sept 14: Golden Toast
Sept 16–17: Cure Fest

Calling all childhood cancer families! You're invited to submit artwork for the 3rd Annual Alliance for Childhood Cancer Art Exhibit. The exhibit will showcase the work of children with cancer, survivors, and siblings and will be featured on Capitol Hill immediately following the Congressional Childhood Cancer Summit in September. Click here for more information.

Spending Bill Clears First Hurdle

The House Appropriations' Labor, Health, and Human Services (LHHS) Subcommittee passed their Fiscal Year (FY) 2018 spending bill, which includes:

FDA Commits to Reducing Orphan Drug Backlog

The Food & Drug Administration (FDA) released a strategic plan to ensure timely review of orphan drug designation requests. The goals of the plan are as follows:

1. In 90 days, the FDA will complete reviews of all orphan drug designation requests that are older than 120 days (the backlog) while maintaining consistent, scientifically rigorous reviews.
2. Within 90 days of receipt, all new orphan drug designation requests will receive a response by the agency. 

Specific to pediatrics, the Office of Orphan Products Development (OOPD) will collaborate with the Office of Pediatric Therapeutics (OPT) to jointly review rare pediatric disease designation requests. In these cases, OPT will conduct the pediatric review and OOPD will conduct the rare disease review. This policy began as of May 15, 2017.

Orphan status for drugs and biologics is defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the United States, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

FDA Launches Expanded Access Navigator

The Food and Drug Administration (FDA) has launched a new online tool called the Expanded Access Navigator to educate patients and physicians about the FDA’s expanded access process including a directory where companies can submit public links to their expanded access policies, criteria used by companies to determine whether to make a drug available through expanded access, and contact information.

FDA to Host Pediatric Trial Design Workshop

On September 8, 2017, the FDA is hosting a public workshop titled “Pediatric Trial Design and Modeling: Moving into the Next Decade.” The purpose of this 1-day workshop is to

1. review the current best practices in designing pediatric drug development trials using the knowledge and tools available
2. discuss problems and potential solutions presently encountered with pediatric drug development trials
3. discuss strategies related to design and evaluation that have the best chance of facilitating and optimizing the use of pediatric drug development trials to achieve the labeling of products for pediatric indications.

More information can be found on the FDA website.

National Academies of Science, Engineering, and Medicine Releases Pain Management Study

National Academies of Science, Engineering, and Medicine (NASEM) recently released Pain Management and the Opioid Epidemic: Balancing Societal and Individual Benefits and Risks of Prescription Opioid Use, which provides a state-of-the-science on pain research, care, and education and identifies actions the Food and Drug Administration and others can take to respond to the opioid epidemic.

Cancer Moonshot Funding Opportunities

The 21st Century Cures Act, which was signed into law in December 2016 made $300 million available in Fiscal Year 2017 for the National Cancer Institute (NCI) to accelerate research efforts aligned with the Cancer Moonshot. The NCI has updated the Cancer Moonshot webpage to list all funding opportunity announcements aligned with the goals of the Moonshot.

For more information on ASPHO's coalition partners and activities, here are some helpful links:

Alliance for Childhood Cancer
American Academy of Pediatrics
American Society of Hematology
Sickle Cell Disease Coalition

Please visit our Health Policy & Advocacy website section for past issues of Advocacy Brief.