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Sucraid Shortage and QOL Response Summary
SUCRAID® SHORTAGE UPDATE
Conference Call with QOL Medical CEO Derick Cooper on April 13, 2016 at 2 p.m. (EST)
For current information on Sucraid and QOL updates, visit the following websites:
The following are highlights and follow-up on Q&A from the Conference Call
The listed questions were provided to Mr. Cooper in advance. Some were answered in conjunction with others during the conference call so not every question is answered precisely, but is addressed at one point or another. I’ve noted a reference to the answer for another question when it applies.
(Note: I’ve done my best to present this information in an objective manner and to state the information provided by Mr. Cooper as accurately as possible for informational purposes only.)
- Could QOL Medical clarify the reason for the shortage? Is it related to a combination of factors or primarily one?
Information known prior to the conference:
a) Ingredient related: The FDA website statement mentions “shortage related to unavailable approved active pharmaceutical ingredient (API) from a supplier due to requirements related to complying with good manufacturing process.”
b) New facility and production related: Letter by QOL Medical CEO March 2016 mentions the construction of an entire new facility and transfer of production to entirely new building.
ANSWER: Mr. Cooper further explained the change in facilities was to transfer the production of the primary ingredient in Sucraid – sacrosidase – from a food-grade manufacturing facility to a pharmaceutical-grade facility per FDA request.
- Reason behind the decision to change production which resulted in the shortage: Sucraid has been produced for years and used by patients for 17+ years. If the formulation hasn’t changed at all, what happened now, to get us to where we are now?
ANSWER: Sacrosidase originally made in a food-grade facility that no longer met pharmaceutical standards. QOL has rebuilt a new facility to meet all current standards. They are working on details for final approval to begin manufacturing with FDA. Although the production of the ingredient met FDA requirements prior to this change, QOL is now “migrating manufacturing to meet modern standards”. Mr. Cooper mentioned that there are “items” to finalize with FDA within the week, but did not mention what those details were.
According to Mr. Cooper, the requirements to manufacture pharmaceutical-grade products involve two steps: 1) Tests of ingredient itself after processing to ensure that the product still effective, and no contaminants were introduced during the process. 2) Manufacturing process itself is very complicated. Mr. Cooper did his best to describe the general steps involved as an example to demonstrate the details and steps involved in the migration. They start with a lot of yeast, then extract the sacrosidase from the yeast and filter multiple times to ensure only sacrosidase is left in a pure form. The filter must be checked by outside vendor who has valid methods for checking filter. Each part of this process needs to be documented and submitted to the FDA. QOL has made valiant efforts to follow these procedures and paperwork requirements the entire time. FDA regulations are ever-changing and do not always apply to each specific product produced. These guidelines are subject to interpretation, and QOL has done their best to interpret the guidelines according to the specific process and manufacturing of sacrosidase (Sucraid). The FDA and QOL are working together to expedite the process as much as possible.
- What does QOL Medical see as the current status of the shortage? Can you expand on the below information?
Information known prior to the conference:
a) Letter by QOL Medical CEO March 2016 mentions “we have built this new facility, transferred the manufacturing process, begun production, and filed the necessary regulatory application with the FDA for approval. We have produced Sucraid® in the new facility and are awaiting final regulatory approvals for distribution.”
b) Calls to Sucraid Assist / One Patient Services mentioned new facility has already produced Sucraid but that those lots have not been FDA approved.
c) Accredo representative has stated the shortage prolongation is due to the FDA not approving an ingredient in Sucraid.
To clarify, it was the production of the main ingredient, sacrosidase within a food-grade facility the FDA decided needed to take place in a pharmaceutical-grade facility which promoted the changes. QOL did not anticipate the building and transfer to new facilities taking so long, which is the assumed reason they did not manufacture enough Sucraid prior to the transfer to meet the demand. This is my interpretation of what seemed to be implied by Mr. Cooper.
- FDA states they are awaiting information from QOL in order to approve Sucraid lots. What are they waiting for? As of today, all paperwork has been turned in and inspection of facility is complete. There are a few minor details (not specified by Derek) that will be address tomorrow (April 14, 2016) in a meeting between QOL and FDA for final resolution.
ANSWER: At the time of the phone conference, FDA had completed a full inspection of the new facility and QOL had completed and submitted all required paperwork.
- Does QOL anticipate a foreseeable end date to shortage?
ANSWER: Though this could not be answered precisely, Mr. Cooper said it could take up to 2-3 months before Sucraid is available again, but he was hopeful it would be sooner.
- Can QOL Medical provide an estimated timeline for a resolution of the shortage in terms of weeks, months or longer? Patients would like to know if they should plan to be without Sucraid for a short, medium or long period of time. They are cognizant any specific timeline is unlikely as it will invariably change.
See ANSWER TO #5.
- How can the most vulnerable patients be prioritized to receive any available, or first available, Sucraid? For the families who have quit using Sucraid to make it more available to more at risk patients, how will it be communicated to us that there is enough to begin using it again?
There are some units left they are saving for these patients according to dire need, however they will not use this supply if they are able to begin manufacturing a new batch soon.
- Does QOL have any data about other medications that experienced a similar facility approval related shortage situation? If so, what was the timeframe for those similar cases to be resolved? When it is released, it will be available within a week of FDA approval. NA
- Can QOL confirm that all Sucraid that has been currently released is only done with full FDA approval?
Information known prior to the conference:
a) That there are NO unapproved lots in circulation.
b) That Sucraid lots requiring an informed consent listed additional possible risks but were still approved as safe to be released by the FDA.
c) That current Sucraid is safe to use and that all possible risks have been fully disclosed in writing to patients. That neither QOL nor the FDA has hidden any important information from them.
d) FDA stated this clearly in the teleconference, but the point seems to have still been confused by other external statements on the call and noise distraction.
Details related to this question were not addressed specifically enough for me to confirm or deny the details.
- Even though it was FDA approved as safe to use with informed consent, What were the specific contaminants that were identified or posed as possible risks?
ANSWER: I asked Mr. Cooper to clarify this once the call was opened to questions. If I understood him correctly, it was the “manufacturing of sacrosidase” that no longer met FDA requirements. There was NOT any contamination of the ingredient.
It’s possible the FDA was concerned the food-grade facility could lead to a risk which is why they prompted the move to new pharmaceutical grade facilities, but this inference was not made by Mr. Cooper himself.
ADDITIONAL DETAILS SHARED DURING THE CALL
To clarify the frustration of one parent on why they were not informed of the possible shortage in advance, Mr. Cooper explained that QOL doesn’t have the ability to directly contact patients due to HIPPA.
Possible Solutions to Communicating with Parents and Patients in the future Not Explored during the call: Communicating in general with pharmacies, doctors, or insurance companies OR placing a notice in the packaging of Sucraid when this process started, letting patients know that a shortage was possible.
In answer to another caller’s question, Mr. Cooper clarified that this transfer to new facilities would not impact the cost of Sucraid or change any current programs designed to assist families in need.
Mr. Cooper ended the call by stating that QOL would love to hear stories about how Sucraid has made a difference. Again, they are doing all they can to expedite this approval process as soon as possible, and they are deeply sorry for the issues this shortage has caused to families.