CLC Provides FDA
Guidance on Drug Shortages Strategic Plan
March 14, 2013
Assistant Commissioner for Policy
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, Maryland 20993
Re: Food and Drug Administration Drug Shortages Task Force and Strategic Plan, FDA-2013-N-0124
Filed Electronically at http://www.regulations.gov
Dear Ms. Kux:
The undersigned cancer patient, provider, and research organizations appreciate the opportunity to provide advice regarding the development of a strategic plan on drug shortages. Our comments will focus on improving the methods and timeliness of notice regarding shortages, identifying and assessing alternative manufacturing sites and entities for drugs and biologicals in shortage situation, evaluating possible incentives for entities to begin or expand manufacturing of drugs in short supply, and developing strategies to assess and address the impact of shortages on clinical trials.
During the last several years, diverse cancer community stakeholders have described the significant impact of cancer drug shortages on cancer care. Physicians have reported the pressure to find alternative treatment options when critical cancer drugs are unavailable; physicians, pharmacists, and nurses have described hours spent managing shortages and finding substitute drugs; patients have expressed their fears about receiving a therapy that is described as less than optimal; and clinical researchers have reported abandonment of studies because a drug crucial to the trial was simply not available.
Pediatric researchers have recently described the impact of drug shortages on long-term health outcomes. A December 27, 2012, study in the New England Journal of Medicine found that the substitution of cyclophosphamide for mechlorethamine in the Stanford V regimen for children diagnosed with high-risk and intermediate-risk Hodgkin’s lymphoma was significantly less effective. Cyclophosphamide was substituted only when shortages of mechlorethamine forced the change. The authors found that 75% of those treated with cyclophosphamide enjoyed 2-year event-free survival, compared to 88% with mechlorethamine. The authors warn, “Our results suggest that even promising substitute regimens should be examined carefully before adoption; what might appear to be a suitable alternative regimen may result in an inferior outcome – an intolerable situation for young people with curable diseases.”
Shortages of drugs and biologicals result in intolerable situations for both children and adults, and we urge that the development of the strategic plan be approached with a sense of urgency, creativity, and aggressiveness to find solutions to the problem of drug shortages.
We will address several of the specific topics identified by the Food and Drug Administration (FDA) in its Request for Comments.
1. New ideas to encourage high-quality manufacturing and to facilitate expansion of manufacturing capacity.
We understand that in a number of cases, FDA has been able to identify drug manufacturers who could respond to drug shortages by expanding manufacturing capacity for current products, opening facilities to begin manufacture of a drug or biological, or resuming production of a drug or biological in cases where manufacturing had been interrupted. These solutions have been achieved primarily through aggressive and creative management by FDA, combined with rapid action by responsive manufacturers. We urge the agency, in developing its strategic plan, to consider incentives that might provide additional encouragement for manufacturers to resolve shortages. These might include waivers of application fees for new drug applications or abbreviated new drug applications and additional (and possibly transferable) exclusivity for products in the same therapeutic areas as the drug in shortage. These options may be beyond the current authority of FDA, but they might be evaluated in a strategic plan to ascertain whether legislation would be necessary to provide FDA authority to implement these reforms.
2. Incentives that federal agencies, separately or in partnership with FDA, might provide to prevent shortages.
We recommend that FDA evaluate steps that it and other federal agencies might take to form partnerships not simply with each other but with potential manufacturers to address or mitigate shortages. The federal government has experience in partnering with or funding industry partners for the manufacture of vaccines. We urge that, as part of the strategic plan, the past experience related to vaccine manufacturing be evaluated and any promising models be considered for replication to address shortages of drugs and biologicals.
3. Communications to alleviate potential or actual shortages.
We recommend changes in the methods for communicating shortages, and we urge that special attention be directed to 1) communication with patients, and 2) collaboration with patient organizations to strengthen outreach to patients who need information about shortages. In developing the strategic plan on drug shortages, FDA should consider the following recommendations.
To assist in its decision-making related to management and mitigation of the impact of drug shortages on clinical trials, FDA should seek input from a number of agencies and organizations. We recommend that FDA seek advice related to drug shortages and clinical trials from NCI, which has deep knowledge about clinical trials supported with NCI funds; the FDA Office of Hematology and Oncology Products; the Coalition of Cancer Cooperative Groups; cancer centers; and the American Society of Clinical Oncology and other professional groups. These organizations and agencies and others can provide FDA advice about the impact of drug shortages on clinical trials to date as well as guidance about how to manage drug supplies going forward to ensure completion – rather than abandonment – of clinical trials. We are recommending that these entities be consulted during the development of the strategic plan and that a mechanism be created for obtaining ongoing input regarding clinical trials from these groups.
We understand that economic solutions, including possible changes in the reimbursement for drugs that are in short supply, are beyond the scope of this study. We believe that changes in third-party payment may be necessary to address drug shortages in a comprehensive and conclusive way, and we will encourage those changes to the appropriate decision-makers.
We appreciate the opportunity to comment on the FDA drug shortages strategic plan.
Cancer Leadership Council
American Society for Radiation Oncology
American Society of Clinical Oncology
Bladder Cancer Advocacy Network
The Children's Cause for Cancer Advocacy
Fight Colorectal Cancer
International Myeloma Foundation
The Leukemia & Lymphoma Society
Lymphoma Research Foundation
Multiple Myeloma Research Foundation
National Coalition for Cancer Survivorship
National Lung Cancer Partnership
Pancreatic Cancer Action Network
Prevent Cancer Foundation
Sarcoma Foundation of America
Susan G. Komen for the Cure Advocacy Alliance
Us TOO International Prostate Cancer Education and Support Network
 Metzger ML, Billett A, and Link MP. The impact of drug shortages on children with cancer – the example of mechlorethamine. N Engl J Med 2012; 367:2461-2463.
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