The Ramazzini Institute
for Occupational and Environmental Health Research   

Who Are We?

Nancye Buelow
Unique, Urgent Need for Blood Plasma Availability
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     There are issues of safe and available blood plasma prevalent in every population, but there some issues unique to each plasma-using community. Over the last few years, there has been only one manufacturer of a plasma-based therapy licensed in the United States to treat Alpha-1. This is unique only to the Alpha-1 community. A sole manufacturer with a single product continues to make our community very vulnerable. We have experienced numerous shortages in the past due to a variety of reasons. As of a few weeks ago the Alpha-1 patient community is facing a total lack of any product at all. While we are being told that the manufacturer anticipates the distribution to resume in late May, thousands of people in precariously poor health are left without this life-sustaining product to see them through until then Any lung infection can cause loss of valuable lung function. Too many times in the past we have watched our friends loose their battle as a lung infection causes their death.
     In a community of less than 10,000, the last issue of the Alpha-1 News had six pages of memorials. We have young children losing their parents. We also have young, very sick adults unable to participate in parenting much less the workforce.
     This situation has reached critical mass. Supply does not meet demand. One manufacturer can no longer produce enough product for our community. With the awareness efforts of the Alpha-1 Association and the Alpha-1 Foundation, fortunately more Alphas are being identified. We can help these newly identified Alphas begin to monitor their health more aggressively, immediately seeking medical attention with any infection or exacerbation, hoping to stem the tide before too much damage is done to their fragile lung tissue. We can also help them understand how important it is for them to practice the best possible health habits. However, they need and deserve a treatment to sustain their lives.
     Can you imagine being told that you have a life threatening genetic disease that has a treatment but it is unavailable for you? That is what happened to me when I was diagnosed, a young mother with my children still at home. I was diagnosed after being treated for an unremitting case of pneumonia. I was given course after course of treatment and still could not recover. Once I was given a diagnosis of Alpha-1 I had to wait nine more months for treatment. Sadly and incredibly, over eight years later, this is still happening. Imagine what it is like to have to wait for someone to die so that you can get enough treatment to sustain your life.

We understand that there are two more IV products that have been yet to be released.

• We ask the committee to request that the FDA facilitate a rapid approval of this much-needed supply of augmentation therapy for our patients.
• We also ask that you support the development of aerosol A1PI therapy and work closely with the manufacturers to provide us with more products and more choices.
• Finally we ask that the committee recommend to the FDA to make A1PI aerosol a priority and to support clinical trials.

While we totally support and understand the diligence of the FDA to assure the public of safe products from compliant manufacturing facilities, please consider the impact of increasingly complex rules requiring plants to close for substantial periods of time on product availability. We do not wish to have safety compromised, but we advocate for ongoing dialogue to maximize the process and minimize the impact on the patient communities.
     The Alpha-1 community, cannot solve these problems alone. We need the help of the Secretary of Health and Human Services and the FDA. Our lives depend upon it.

*Nancye Buelow, a valued contributor to GEE!, is a member of the Board of the Genetic Alliance and former President of the Alpha 1 Association. This article is based on her testimony before the Food and Drug Administration Committee for Blood Safety and Availability on April 20, 2001