Article - Plasma Therapies: Where Are We Now?

Date: May 3, 2011

Several key variables play a role in the promising possibilities for lifesaving plasma protein therapies.

By Kris McFalls

As the market for plasma protein therapies continues to grow at a steady pace, the possibilities for these precious lifesaving proteins seem endless. Patients long for that one treatment to return them to the quality of health and life they once had. Scientists, in turn, are dedicated to providing the safest, most effective product possible. And the manufacturers’ desire to make it all happen is indisputable. However, the plasma protein market contains difficult-to-control variables that have a skewing effect on the laws of supply and demand. Limited natural resources, long processing time, increasing costs for expensive plasma testing, few manufacturers, costly but necessary government regulations and low reimbursement rates can make the plasma market particularly vulnerable to prolonged and painful market swings.

The Plasma Variable

As the demand for plasma-based therapies continues to increase, several manufacturers are exploring new indications for these lifesaving therapies. But an increase in demand for these products first necessitates an increase in the raw product: plasma.

In the United States alone, there were roughly 22 million donations of plasma collected in 2009, according to the Plasma Protein Therapy Association (PPTA). This represents an approximate increase of 4.5 million donations over 2008. Most of the world’s plasma was collected in about 400 plasma donation centers scattered throughout the U.S., the majority of which are owned by Baxter’s BioLife Plasma Services, CSL Behring, Talecris Plasma Resources, Octapharma Plasma Inc. and Grifol’s Biomat USA Inc.

Increasing the number of plasma donors to meet the increased product demand is challenging at best. And, regulations set forth by the U.S. Food and Drug Administration (FDA) to ensure the safety of the blood supply limits how much plasma can be collected. This raises the need for exploring safe and reliable ways to increase the donor pool.

This past year, the Health and Human Services’ Advisory Committee on Blood Safety and Availability (ACBSA) considered relaxing the rules banning gay and bisexual men from donating blood or plasma. Current policy dictates that all men who have had sex with other men since 1977 be deferred as donors. Both patient and industry groups recognize that the HIV epidemic had disproportionally impacted both gay men and the plasma user community. The American Red Cross, America’s Blood Centers and Advanced Transfusion and Cellular Therapies Worldwide were in favor of changing the ban to a temporary 12-month deferral after the last sexual encounter. But, several patient organizations, including the PPTA, released statements in favor of retaining the ban until further research into the matter could be attained because current data did not sufficiently support a change to the donor deferral policy. On June 10, 2010, the ACBSA found that data did not support changing the current policy and, therefore, did not recommend a change. The committee, however, did recommend researching the matter further.

The Manufacturer Variable

Even when the plasma supply is robust, there is a risk of a sudden shortage of plasma protein therapies due to a limited number of manufacturers. This is especially true in the immune globulin (IG) market, where current science is unable to develop a production method for manufacturing a recombinant product. And, because most of the manufacturers provide products for the worldwide market, a shortage creates a domino effect on the worldwide supply.

Those who have been in the IG business for a long time remember the mid- to late-1990s, when IG products were in very short supply. Some patients were forced to go without treatment or extend the intervals between treatments. Brand choice was limited, and many patients were forced to take whatever product was available due to shortages caused by plant shutdowns, viral contamination and discontinued products due to mergers. Since that time, manufacturing processes and safety continue to improve. Most products are now liquid, the result of a manufacturing process that produces a higher yield. Safety procedures are more stringent, and the blood supply is safer as evidenced by the significantly fewer number of recalls. Yet, with so few manufacturers, recalls and withdrawals continue to pose a risk to the worldwide supply of IG. Recently, the market experienced a complete withdrawal of Octagam (immune globulin intravenous [human] 5% liquid preparation). Even though Octapharma is not one of the top-three producers of IG for the U.S., the voluntary withdrawal still affected the U.S. market since supplies from all markets were needed to fill the void of the loss of Octagam from the U.S., Australia and Europe. And, even with improved manufacturing efficiency, it still takes approximately nine months to transform a plasma donation into a vial of ready-to-infuse IG. Therefore, even a rapid increase of production by the other manufacturers may not be able to meet the demands caused by a sudden withdrawal of any one brand of IG.

While there is currently not a shortage of IG, history has shown that recalls and withdrawals can cause sudden and unexpected shortages at any time, and to be complacent and unprepared for such possibilities is to put patients’ lives in danger. “The wonderful thing about IVIG [intravenous IG] is its unwavering path toward dozens of undiscovered areas of therapeutic promise for thousands of patients globally,” says Chris Ground, FFF Enterprises’ vice president of national accounts. “This is precisely why we must make every effort to be vigilant surrounding the global supply-and-demand ratio of IVIG and to work to try to keep those factors in balance. Being out of balance creates challenges either way. This, of course, is easier said than done, given the always present possibility of manufacturing issues. Yet, this is an inextricable fact of the plasma industry. We must strive to nurture demand, while delicately balancing this with managed increases in manufacturers’ global capacity.”

The Reimbursement Variable

Even in stable markets, reimbursement rates continue to pose a risk to plasma-derived therapies. Several industry and patient organizations continue to press Medicare for a change in the current reimbursement model. In its public comments to the Centers for Medicare and Medicaid Services (CMS), the PPTA supported the “CMS proposal to set the payment level of separately payable non-pass-through drugs and biologicals, which include most plasma protein therapies, at average sales price (ASP) plus 6 percent. [However, the PPTA raised objections to] the policy of using hospital claims data that includes drugs and biologicals sold as part of the heavily discounted 340B Drug Pricing Program when setting Outpatient Prospective Payment System (OPPS) payment rates.” Additionally, several patient organizations continue to press hard for changes in the Medicare payment system to allow patients equal access to care both in a home and clinical setting.

Some states are trying to ensure patient access to care by targeting private insurers’ policies. A few states have either passed or are considering legislation to disallow private insurers from moving plasma therapies into a high tier-level program with copayments as high as 50 percent of the cost of the medication. Included in the same legislation is a proposal that also would limit the yearly out-of-pocket maximum patients must pay. With decreasing reimbursement rates and increased patient liability, legislation will continue to play a key role in patient access to care.

Possibilities and Responsibilities

As the plasma therapy market continues to evolve and grow, so will the possibilities and responsibilities. It is clear that the industry is excited about the promises these lifesaving products bring to patients worldwide. Yet, while focused on fulfilling this potential, all participants also are working very hard to not lose sight of their responsibilities for doing it right.

Kris McFalls is a staff writer for BioSupply Trends Quarterly and the patient advocate for IG Living magazine, distributed to patients who rely on immune globulin and to their healthcare providers.