MitoAction Submits Comment to FDA on Proposed Changes to Compounding Pharmacy Regulations

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On Monday, July 20, 2015, MitoAction submitted a comment to the FDA on its proposed Memorandum of Understanding concerning the distribution of products from compounding pharmacies. The proposed Memorandum of Understanding would substantially limit the ability of compounding pharmacies to distribute compounded prescriptions across state lines. Since many Mito families rely upon high-quality compounding pharmacies that often are located in states other than their own for preparation of the Mito cocktail and other supplements, the proposed Memorandum of Understanding would have a significant detrimental effect on the Mito community. MitoAction submitted the following comment to make the FDA aware of the proposed Memorandum of Understanding’s disproportionate impact on the rare disease community.
Agency: Food and Drug Administration (FDA)
Document Type: Nonrulemaking
Title: Memorandum of Understanding Addressing Certain Distributions of Compounded Human Drug Products Between the States and the Food and Drug Administration; New Proposed Draft; Availability
Document ID: FDA-2014-N-1459-0001


On behalf of MitoAction, a non-profit organization that represents patients suffering from mitochondrial disease and their families, I would like to express my deep concern over the proposed changes to the FDA Memorandum of Understanding Addressing Certain Distributions of Compounded Human Drug Products Between the States and the Food and Drug Administration.

MitoAction has a strong interest in ensuring that its patients are able to access high-quality compounded medications no matter where they reside in the United States. Patients with mitochondrial disease, which affects energy production within the cells, often use compounded preparations consisting of high-dose vitamins and antioxidants to combat the symptoms and slow the progression of the disease. Since mitochondrial disease is rare, only a handful of compounding pharmacies specialize in preparing these compounds, each of which must be specially tailored to the particular patient.

The FDA’s proposed Memorandum of Understanding (“MOU”) would limit the distribution of compounded medications to 5% of sales for compounding pharmacies in a state that does not sign the MOU, and 30% of sales in states where the state government has signed the MOU. These limits are concerning to the mitochondrial disease community for the following reasons:

(1) There is a disincentive for states to sign the MOU as it requires increased state oversight without the allocation of additional funds to state governments for this extra work. Compounding pharmacies located in states that do not sign onto the MOU have no recourse and stand to lose significant business if their state does not agree to the MOU.

(2) The limits on sales imposed on compounding pharmacies will be more burdensome for high-quality pharmacies with expertise in a particular patient population and customers throughout the country. Since compounding pharmacies that specialize in preparations for rare diseases and cater to widely dispersed patients will be disproportionately impacted, patients with rare diseases are more at risk of losing access to vital compounded medications.

(3) The stated limits would be difficult to comply with as pharmacies cannot predict what percentage of total sales will be comprised of out-of-state sales until the end of the year. Since it will be impossible to tell what percentage of sales are out-of-state until the end of the year, some compounding pharmacies may decide to cease interstate sales altogether. This would have a terrible impact on families dealing with rare diseases, such as mitochondrial disease, who rely heavily on well-established compounding pharmacies for life-sustaining medications and supplements. As long as pharmacies are licensed in the states to which they are shipping, there should be no limit on the number of sales shipped out of state.

In addition to serving as Director of Outreach & Advocacy for MitoAction, I am also the parent of a child with a mitochondrial disorder. My child takes two compounded preparations, one of which comes from a compounding pharmacy in Massachusetts, and the other of which is delivered from a compounding pharmacy in Ohio. We sought out these compounding pharmacies after failing to find the expertise we needed among compounding pharmacists in our home state of Georgia.

With these compounded prescriptions as part of our medical regimen, my child has gone from multiple hospital visits per year to zero hospital visits since March 2014. I am extremely concerned that if our current compounding pharmacies are unable to send our compounded preparations to Georgia, my child will suffer from increased symptoms and additional hospitalizations.

Our family’s situation is representative of many other mitochondrial disease patients and families. The MOU as it currently stands would penalize most harshly the compounding pharmacies that have been the most successful and to whom patients turn because of their specialized knowledge. Without access to knowledgeable compounding pharmacists, some of whom may be in states different than their own, the patients ultimately pay the price with their health and associated increases in medical costs.

On both a personal and professional level, I urge the FDA not to place these limits on patient access to compounded medications and to find a different way to oversee and regulate compounded preparations that travel through interstate commerce. The health and welfare of rare disease patients like my child hinge very much on taking a different approach than the one found in the current Memorandum of Understanding.

Respectfully submitted,

Christine S. Cox, J.D.
Director of Outreach and Advocacy
P.O. Box 51474
Boston, MA 02205
1-888-MITOACTION (1-888-648-6228)