Advocating on behalf of those impacted by sarcoma has been a core principal 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 has endorsed a bill introduced by Reps. Cliff Stearns (R-FL) and Ed Towns (D-NY). The bill, H.R. 3737 – Unlocking Lifesaving Treatments for Rare-diseases (ULTRA) Act, amends the Food, Drug, and Cosmetic Act to allow the Food and Drug Administration to approve an application for a drug designated both as an orphan drug and as a fast track product using a surrogate endpoint. The orphan disease or condition needs to affect a “small number of patients in the United States.” The SFA believes that this kind of approach will enable the Food and Drug Administration to exercise flexibility in approving drugs for rare diseases like sarcoma.
H.R. 3737 – ULTRA Act
Letter of Support for the ULTRA Act
ULTRA Act Resources from EveryLife Foundation
Creating Hope Act
The SFA has endorsed a bill proposed in the U.S. Senate that would create incentives for the development of treatments for rare diseases that disproportionately impact children. The bill, S. 606 – Creating Hope Act, was introduced by Sen. Robert Casey (D-PA) in March.
S. 606 – Creating Hope Act
Sen. Robert Casey (D-PA) Press Release on Creating Hope Act
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.
Legislation introduced in the 111th Congress contained language that would have directed the FDA to conduct a review and improve policies related to oncology products, including considering alternatives or surrogates to traditional clinical trial endpoints. The SFA firmly believes that FDA acceptance of clinical endpoints other than overall survival could remove many of the present barriers in the development of drugs to treat rare cancers such as sarcomas.
In 2011, a record number of drugs were reported to be in short supply. A large number of the drugs impacted by this shortage are drugs used by cancer patients. The SFA is highly concerned by this shortage and has been working with other advocacy organizations to address this complicated issue.
President Obama issued an Executive Order related to the drug shortage problem in October of 2011. This Executive Order directed the FDA to “take steps that will help to prevent and reduce current and future disruptions in the supply of lifesaving medicines.”
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)
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.