The ASPHO Advocacy Brief
Welcome to the Advocacy Brief, a quarterly offering from the American Society of Pediatric Hematology/Oncology. The goal of this newsletter is to inform members about legislative and regulatory issues impacting the profession of pediatric hematology/oncology and the patients we serve.
ASPHO Bill Tracker
Visit ASPHO’s Legislative Tracking Chart to see a list of bills that ASPHO is monitoring.
Special Feature Contributions
By Lori Luchtman-Jones, MD, ASPHO Advocacy Committee Member; Julie Jaffray, MD, ASPHO Advocacy Committee Member; and Natasha N. Frederick, MD MPH MS, Chair of ASPHO Advocacy Committee
This month’s advocacy spotlight focuses on inherited bleeding disorders and related patient rights and pending legislation. The mildest bleeding disorder is von Willebrand disease (VWD), affecting males and females equally, with an estimated prevalence of up to 1% of the population. Patients with VWD have decreased or dysfunctional von Willebrand factor leading to mucocutaneous bleeding such as bruising, gastrointestinal bleeding or heavy menstrual bleeding, as well as bleeding after procedures. Another well-known inherited bleeding disorder is hemophilia, which is due to the deficiency of either factor VIII or IX. Patients typically have bleeding into their joints or muscles, bleeding after trauma, dental extractions or surgeries and may also have mucocutaneous bleeding. Males predominantly are affected, due to the inheritance pattern being X-linked, but female carriers can have significant bleeding requiring medical treatment of their bleeding symptoms. Other rarer inherited bleeding disorders that cause mucocutaneous bleeding include platelet dysfunction disorders such as Bernard Soulier and Glanzmann’s Thrombasthenia, and rare deficiencies of other coagulation factors such as V, VII, X, XI, XIII and fibrinogen.
Unrecognized or inadequately-treated bleeding disorders result in unacceptably high morbidity and mortality rates and poor quality of life. As pediatric hematologist/oncologists, it is crucial that we understand what more is needed as well as the value of existing programs and legislation, lest they be sacrificed in the name of cost-cutting or efficiency. Some important resources for clinicians are: (1) the Foundation for Women and Girls with Blood Disorders (http://www.fwgbd.org), which promotes awareness and educates clinicians about bleeding disorders that affect females, (2) National Hemophilia Foundation (https://www.hemophilia.org/) that provides education to healthcare providers and the community about bleeding disorders and (3) Hemophilia Federation of America (https://www.hemophiliafed.org/) that supports and educates the bleeding disorders community.
High quality care facilities: Federally-funded hemophilia treatment centers (HTCs) provide comprehensive, multidisciplinary care for patients with various bleeding disorders. In a study of 3,000 hemophilia patients funded by the Centers for Disease Control (CDC), those enrolled in an HTC were 40% less likely to be hospitalized for bleeding problems (Soucie JM et al. Haemophilia 2001;7:198) or die of hemophilia-related complications (Soucie JM et al. Blood 2000;96:437). In addition, HTCs participate in data collection that is made available to researchers. HTCs have experienced substantial funding reductions in recent years. It is important that financial support for HTCs be maintained. More information about HTCs can be found here: https://www.cdc.gov/ncbddd/hemophilia/htc.html. The National Hemophilia Foundation (NHF) hosted its annual Washington Days event on February 27, where advocates visited their Members of Congress to discuss the need to adequately fund the HTC network across the country. ASPHO is monitoring advocacy efforts related to this meeting.
Access to effective, quality treatments: Patients with bleeding disorders often need expensive treatments such as factor replacement. Pharmacy providers must be knowledgeable about, and experienced in, bleeding disorders. The NHF Medical and Scientific Advisory Council has defined minimum standards for pharmacy providers to bleeding disorder patients, MASAC 188, but compliance with these standards is voluntary. Public and private payers face constant pressures to control health care costs by restricting provider networks, mandating prior authorization, using preferred drug lists and offering lower reimbursement rates. NHF and other groups are advocating for federal- and state-enforced uniform standards of care and service, modelled after the MASAC guidelines. ASPHO is monitoring the status of the MASAC guidelines and the impact of the guidelines at the federal and state levels.
The right to non-discrimination: An important milestone for patients with inherited medical disorders (and carriers) was the passage of the Genetic Non-Discrimination Act (GINA) in 2008. GINA prevents insurers from obtaining genetic information, denying coverage or offering higher premiums for people with a genetic disorder or predisposition. GINA also prohibits employers from discrimination, hiring or firing because of genetic information.
What’s happening with the Affordable Care Act (ACA)? On December 18, 2019, the US Court of Appeals for the Fifth Circuit declared that, without a tax penalty, the ACA’s mandate that most people have health insurance is unconstitutional. The District Court must now decide whether the entire ACA, or just some provisions are unconstitutional. For now, the ACA-mandated protections for those with pre-existing conditions, prohibition of lifetime caps and allowance for children to be covered on parental insurance until age 26 remain valid. It was announced in early March that the Supreme Court will hear an ACA appeal. The case will be taken up as part of the court’s next term, starting in October. Due to this, a decision is not expected until after the November election.
As the only professional organization dedicated solely to the development and interest of subspecialists in pediatric hematology/oncology, ASPHO is uniquely positioned as a health advocate.
ASPHO continues to work towards the advancement of pediatric hematology/oncology related health policy. Specifically, ASPHO focuses on policies that:
- Ensure adequate federal funding for research, surveillance and other legislation;
- Improve access to drugs, devices and biologics for children and adolescents; and
- Promote adequate payment for care of children by subspecialists in pediatric hematology/oncology to support needed access and workforce.
ASPHO is also focused on continuing to increase the profile of our organization as a trusted advocate. For more information on how to get involved, visit A Pediatric Hematologist/Oncologist Guide to Political Action.
The U.S. Response to Coronavirus: Summary of the Coronavirus Preparedness and Response Supplemental Appropriations Act, 2020
The Coronavirus Preparedness and Response Supplemental Appropriations Act, 2020 (P.L. 116-123), which passed with near unanimous support in both the House and Senate, was signed into law by the President on March 6, 2020.
ASPHO joined a large group of organizations and signed on to a letter spearheaded by the American Association for Clinical Chemistry which supports funding to help generate pediatric reference intervals (PRIs) from existing clinical samples. The letter was sent to House and Senate appropriations subcommittees in February and is an updated version of one that ASPHO signed on to last year. This funding would help create a more standard reference interval for a more accurate diagnosis. The CDC supports this initiative and some of the PRIs language from last year’s letter was included as part of the Further Consolidated Appropriations Act of 2020 which became law in December 2019.
By Dominic Sawaya, JD MPPA, Manager, Health Policy and Advocacy
The FDA has a new commissioner, and with the coronavirus looming, the drug shortage issue continues to become more complex. Dr. Stephen M. Hahn became the 24th Commissioner of the FDA on December 17, 2019, after being confirmed by the Senate with a bipartisan vote. Previously, Dr. Hahn was the chief medical executive at the University of Texas MD Anderson Cancer Center.
Upon entering the FDA, Dr. Hahn made assurances that he would guide the FDA towards addressing drug shortages and drug pricing issues. Drug shortages and increases in drug prices have impacted health care providers and patients across the care spectrum. Pediatric hematology/oncology has been specifically impacted by drug shortages, notably the shortage of the chemotherapy drug vincristine. In order to address this issue, Dr. Peter C. Adamson, Children’s Oncology Group Chair and ASPHO Advocacy Committee Member, provided recommendations aimed at altering clinical trial treatment protocols that utilize vincristine. While other drug shortage issues are still ongoing, the coronavirus and its impact on the drug supply chain globally present a serious test for the FDA and its new commissioner.
With the rise of the coronavirus epidemic, there was initial speculation that the impact on the drug distribution chain in China could cause drug shortages in the US. This speculation was confirmed, as Dr. Hahn issued the Coronavirus (COVID-19) Supply Chain Update on February 27 stating that there is a drug shortage as a result of the coronavirus and supply and shipping issues in China. The FDA did not identify the drug or manufacturers specifically but noted that the shortage is related to a site affected by the coronavirus. Alternative drugs are available that can be used by patients, and the FDA is working with manufacturers and others to mitigate the shortage. As part of the ongoing coronavirus response, the FDA has urged more than 180 drug manufacturers to evaluate their entire supply chain. The FDA has also identified approximately 20 other drugs that either solely source their active pharmaceutical ingredients or finished drug products from China to determine if drug shortage risks are present.
As the coronavirus threatens to increase drug shortages, advocacy on this issue is vital to ensure that the FDA follows through on the necessary steps to lessen drug shortages. It is also crucial that the FDA and other federal agencies take regulatory steps to prevent future shortages. ASPHO is continuing to follow drug shortage and pricing issues, with a strong focus on the coronavirus and the FDA’s response.
For more information on our coalition partners and activities here are some helpful links:
Please visit our Health Policy & Advocacy website section for past issues of Advocacy Brief.