Advocating on behalf of those impacted by sarcoma has been a core principle of the Sarcoma Foundation of America’s mission since the organization was founded in 2001. The SFA continues to be actively engaged in the public policy arena, urging legislators and regulators to place a high priority on rare cancer research, drug development, and access to quality patient care.
The SFA joins with other coalitions and patient advocacy organizations to work on public policy efforts related to cancer and rare disease issues.
Current Legislative Priorities
H.R. 820/S. 292, the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act of 2017
The SFA is a supporter of H.R. 820/S. 292, the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act. The STAR Act would expand opportunities for childhood cancer research, improve efforts to identify and track childhood cancer incidences, enhance the quality of life for childhood cancer survivors, and ensure publicly accessible expanded access policies that provide hope for patients who have run out of options.
Status: The STAR Act has been introduced in both the House of Representatives and the Senate.
H.R. 3381/S. 1883, the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act of 2015
The SFA is a supporter of H.R. 3381/S. 1883, the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act. The STAR Act would expand opportunities for childhood cancer research, improve efforts to identify and track childhood cancer incidences, enhance the quality of life for childhood cancer survivors, and ensure publicly accessible expanded access policies that provide hope for patients who have run out of options.
Status: The STAR Act passed in the House of Representatives but was not taken up by the Senate. The legislation will need to be reintroduced in the 115th Congress.
21st Century Cures Act
The SFA is a supporter of H.R. 6, the 21st Century Cures Act. HR 6 accelerates the discovery, development and delivery of life saving and life improving therapies, and transforms the quest for faster cures by: removing barriers to increased research collaboration; incorporating the patient perspective into the drug development and regulatory review process; measuring success and identifying diseases earlier through personalized medicine; modernizing clinical trials; providing new incentives for the development of drugs for rare diseases; helping the entire biomedical ecosystem coordinate more efficiently to find faster cures; and investing in 21st century science and next generation investigators.
Status: The 21st Century Cures Act passed in the House of Representatives and Senate and was signed into law on December 13, 2016.
NIH and FDA Funding
The SFA strongly urges Congress to fully fund the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Drastic and significant cuts in the budgets of health and science programs put funds for cancer research in jeopardy and could slow down the process for drug approval.
Petitions have become an important tool to mobilize stakeholders to advocate for policy changes and other actions. The Sarcoma Foundation of America does, from time to time, participate in petitions. One example is the petition to request a Presidential Proclamation designating July as Sarcoma Awareness Month. Typically, the SFA stays neutral on most petitions as we have a responsibility to represent all sarcoma patients. We do feel it is important to provide a venue for stakeholders in the sarcoma community to share their petitions with others. For this reason, we have created a webpage where these petitions can be share.
If you are interested in recent sarcoma petitions, please follow this link.
Past Legislative Priorities
Creating Hope Act
Past Regulatory Priorities
FDA Review of the Treatment of Rare Diseases
The Agriculture, Rural Development, Food and Drug, and Related Agencies Appropriations Act for 2010 directed the Food and Drug Administration (FDA) to review the treatment of rare diseases. The language in the bill required the FDA to establish “a review group which shall recommend to the Commissioner of Food and Drugs appropriate preclinical trial design, and regulatory paradigms and optimal solutions for the prevention, diagnosis, and treatment of rare diseases.” The FDA must deliver a report on this issue to Congress in March 2011 and must issue guidance in September 2011.
The SFA has been urging the FDA to include language in the report and guidance that acknowledges FDA acceptance of clinical endpoints other than overall survival. Specifically, we are asking the FDA accept progression-free-survival as a meaningful endpoint for evaluating treatment efficacy in smaller clinical trial populations.
Federal Citizen Petition
In 2007, the SFA filed a Citizen Petition with the FDA asking that the Agency issue a formal “Guidance Document” outlining the process by which a drug company could gain approval for their products for rare cancer use. In November 2005, the FDA asked for, and received, input from their Federal Advisory Committee for suggestions on making approval easier and faster for rare cancers like sarcoma. Our petition merely asked for a codification on what would be satisfactory clinical data to gain FDA approval. While there was Congressional support for the SFA’s Citizen Petition, the FDA chose not to act on our request. However, thanks to language in the Agriculture, Rural Development, Food and Drug, and Related Agencies Appropriations Act for 2010, the FDA has been tasked with providing a report to Congress in March 2011, and industry guidance in September 2011, that outlines ways in which the Agency can help people with rare diseases get better treatment. We are hopeful that many of the issues raised in our 2007 Citizen’s Petition will be addressed in the FDA’s report and guidance.
Letters of Support:
Representative Jim Cooper (D-TN)
Representative Patrick Kennedy (D-RI)
Representative Edolphus Towns (D-NY)
Representative Steve Israel (D-NY)
Representative Nydia Velazquez (D-NY)
Representative Tim Ryan (D-OH)