May 2007 Advocacy Update (part two)

ASH Presents Comments on Appropriate Use of ESAs at FDA ODAC Meeting; ODAC Recommends Further Restrictions and Study
ASH Councillor and Chair of the Reimbursement Subcommittee, Samuel Silver, MD, presented comments on the appropriate use of erythropoietin-stimulating agents (ESAs) at the May 10 meeting of the Food and Drug Administration Oncologic Drugs Advisory Committee (ODAC).

ASH's comments emphasized four main points:

1. ESAs should continue to be used for treatment of anemic patients with hematologic malignancies not undergoing chemotherapy while Phase III studies need to be initiated to address this issue further;
2. The appropriate length of time for a patient to be considered under the umbrella of chemotherapy treatment;
3. Evidence to support the use of ESAs in anemic patients with low-risk myelodysplasia; and
4. Treatment recommendations.

ODAC made the following recommendations to FDA:

• Further marketing authorizations should be contingent upon additional restrictions in product labeling and additional trials.
• Labeling should specifically state that ESAs are not indicated for use in specific diseases studied in trials that showed adverse safety signals. This restriction would apply until adequate trials and subsequent data are reviewed by FDA. Tumor types that may be included would be breast cancer, head and neck cancers, and non-small cell lung cancer. ODAC recognized that MDS and other hematological malignancies should not be swept up into this and ODAC staff indicated they would consult with CMS.
• Product labeling should define a hemoglobin level in asymptomatic patients at which ESAs should be initiated.
• Product labeling should recommend discontinuation of ESAs following completion of chemotherapy and reevaluation of the degree of anemia with subsequent chemotherapy.

For more information about ASH's comments to Medicare, view "ASH Weighs in on Review of Medicare Coverage Policy for Erythropoietin-Stimulating Agents (ESAs)."

ASH Submits Comments to Medicare on Proposed Revisions to Clinical Research Policy
On April 10, the Centers for Medicare and Medicaid Services (CMS) proposed revisions to the Medicare National Clinical Trial Policy, to be renamed the Clinical Research Policy (CRP). Since the September 2000 issuance of a National Coverage Decision (NCD), Medicare has paid for the routine costs of beneficiaries enrolled in qualifying clinical trials. The revised policy would, among its many provisions, pay for investigational treatments and services if they are covered by Medicare outside the clinical trial or are required in order to manage the patient's health within the study.

ASH's comments to CMS on the revised policy took into account comments and input from the Reimbursement Subcommittee, the Committee on Government Affairs, and the general ASH membership.

Among other provisions, the proposed CRP:

• Adds a definition of research;
• Requires the research study to be registered on the ClinicalTrials.gov Web site prior to the enrollment of the first study subject;
• Renames "routine costs" to "routine clinical services" and recommends that this term be defined "as those items and services that are available to Medicare beneficiaries outside the study, but are not explicitly the item or service being evaluated for its effect on health outcomes (i.e., not the investigational item or services), only when those items and services are used for patient management within the study;" and
• Requires research studies to have written protocols that "specify and fulfill method and timing for public release of results," have "explicitly discussed inclusion criteria and considered relevant subpopulations (as defined by age, gender, race/ethnicity, socioeconomic, or other factors)," and "contain a discussion of how the results will generalize to the Medicare population."

Senate Passes FDA Authorization Bill
The Senate passed, by a vote of 93 to one, legislation that would, among its many provisions, reauthorize the Prescription Drug User Fee Act (PDUFA).

S. 1082, the FDA Revitalization Act, reauthorizes PDUFA through 2012. The legislation also includes a number of prescription drug safety provisions, additional oversight of prescription drug advertising, and additional civil penalties for noncompliance with FDA label revision requests.

Though many believed that provisions providing for an approval process for generic biologics would be included in S. 1082, such provisions were ultimately left out of the final bill that passed the Senate. Supporters of these efforts worked with Senate leaders to negotiate an agreement to have generic biologic legislation considered separately by the Senate later this summer.

The House has begun holding committee hearings on the issues of PDUFA reauthorization, drug safety, and approval of generic biologics, but no timetable has been set for consideration of legislation similar to S. 1082 by the full House.

National Institutes of Health (NIH) News and Information
View the latest issue of the "NIH Public Bulletin" for information on:

• Hematologists Barbara Alving and Griffin Rodgers, the newly appointed NCRR and NIDDK directors
• Registration for a teleconference about cancer clinical trials
• The new monthly podcast series on women's health issues
• The latest public events, activities, and health information resources

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