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A lot has changed in the nearly 30 years since hemophilia treatment centers (HTC) were started as a way to better diagnose and treat people with bleeding disorders. Patients have access to better-coordinated comprehensive and preventive medical care. But even with today’s nationwide network of more than 140 HTCs, doctors and other medical professionals want to do a better job of communicating best practices and standards with each other.

One challenge healthcare professionals face is knowing the best way to care for each of their patients. New guidelines from the National Hemophilia Foundation’s (NHF’s) Medical and Science Advisory Committee (MASAC) and cutting-edge medical research can certainly help doctors make the best treatment decisions, but more data about which medical decisions work—and which don’t would also help.

In 1998, the Centers for Disease Control and Prevention (CDC) established the Universal Data Collection (UDC) Project to monitor the safety of the nation’s blood supply for people being treated with blood products, as well as the occurrence of joint complications in people with hemophilia. In the past few years, HTCs have standardized their data collection for the CDC on Lab Tracker, a medical record and disease management program from Ground Zero Software. The company now claims the program tracks 92% of all US hemophilia patients. But CDC staff admit that the UDC database is incomplete. It doesnt fulfill the clinical needs [of hematologists and the HTCs], says Roshni Kulkarni, MD, director of the Division of Blood Disorders at the CDCs National Center on Birth Defects and Developmental Disabilities in Atlanta. The CDC has data on about 20,000 patients with hemophilia in the database, but no good estimate of the true size of the US population with bleeding and clotting disorders. You want to have a defined population for testing, Kulkarni says. There has been a need in this community to collect not only outcomes data, but treatment data. Since the HTCs operate independently, many cases on file at individual locations are not reported to a central authority. Right now, the way the system is set up, theres no good way to share data between the treatment centers, explains Ray Stanhope, NHF board chairman. A large, unidentified community is involved. Now, a nationwide data-collection project has been established to make treatment data more accessible and usable. This project is called the American Thrombosis and Hemostasis Network (ATHN).

The Time is Now

In this decade alone, crises such as Hurricanes Katrina and Rita in September 2005, as well as the August 2003 blackout that left 50 million people in the Northeast, Midwest and Canada without electricity, highlight the importance of access to the health and treatment information of those with bleeding and clotting disorders. In late 2004, the hemophilia and thrombosis community held meetings to determine whether to establish an organization to collect and analyze relevant information. The CDC provided funding to hire a consultant during the exploratory phase. The consensus was yes, it did make sense to have a new organization created to shepherd that effort, says Diane Aschman, RPh, the first president and CEO of ATHN. The overall purpose of ATHN is providing stewardship to this national database. Input from the clinical and administrative committees at those meetings led to the creation of ATHN in July 2006. As a public-private partnership, ATHN is officially a nonprofit entity incorporated in Georgia with headquarters in Riverwoods, Ill. With support from the CDC and a grant from drug-maker Novo Nordisk, ATHN plans to centralize clinical record keeping among HTCs with a high-tech database for physicians, public-health officials, researchers and advocacy groups. When fully operational, the ATHN project will be the largest effort to date to collect, analyze and share information about people with bleeding disorders. It is expected to help MASAC develop best practices for treatment. In its work, MASAC needs standards of care, says Aschman. It needs data to set standards. Aschman also hopes ATHN will be useful in screening patients for clinical trials and in developing research hypotheses. For instance, capturing data electronically will help researchers better monitor a cohort of patients over time. The infrastructure should help to standardize the collection of data needed for clinical trials, Aschman says. In the long term, patients will be contributing to quality improvements. The whole concept is to advance the science. Amy Shapiro, MD, ATHN co-chair and medical director of the Indiana Hemophilia and Thrombosis Center in Indianapolis, is optimistic. This allows us to ask questions on a national level for the first time, she says. This is the first step in even trying to look at outcomes. One of the big things that didnt exist at all is a totally confidential database of all hemophilia patients in the country, says Jeanne Lusher, MD, the other co-chair of ATHN and director of the Hemophilia, Hemostasis and Thrombosis Program at Childrens Hospital of Michigan in Detroit. If there is another disaster like Hurricane Katrina, ATHN will have this secure database, she says. Participants chose Lusher and Shapiro as board co-chairs to guide the fledgling organization through its first year of operation. Their tasks include forming committees, developing a governance structure, hiring executives and setting the scope of work for each committee. San Francisco-based Ground Zero Software is now working closely with the CDC and ATHN to convert the software into a format known as Web Tracker. It became evident that the tool, which was a client-server tool, would be more effective as a Webbased tool, Aschman says. Having all its data on the Web relieves ATHN and the HTCs of the burden of installation, maintenance, security and data storage, Aschman says. This is important, since some of the HTCs are small, independent operations. They dont all have sophisticated IT [information technology] staff, Aschman explains. By early fall 2007, Aschman expects some HTCs to start testing the Web-based software. Patients will have to give their consent to participate in the beta testing, in which versions of the new software will be released to a limited audience for further testing to ensure that the product has few faults or bugs. Our firmest date is to have the beta testing done by the end of this year, she says. Data collection will begin in January 2008. ATHN plans call for a permanent board to be in place by the end of 2007. It will include two-thirds of the current board, as onethird of members will rotate off each year. We are just now determining the scope of work for each committee, Shapiro says.

Privacy Concerns

Despite the potential benefits of such a large-scale data collection effort, there are people in the bleeding disorders community who are skeptical of the project. Critics worry that the information in the database wont be completely secure and confidential. Why are we privatizing hemophilia data? asks Corey Dubin, president of the Committee of Ten Thousand, a group representing HIV patients, including those with hemophilia who contracted the AIDS virus from contaminated blood products. In light of the five-year, unrestricted Novo Nordisk grant an undisclosed amount meant for ATHN to hire staff and build its network infrastructureDubin also wonders about the commercial value of the ATHN database. The issue is: Did Novo just buy access? he asks. We have asked these questions in public forums, says Dubin. According to Dubin, ATHN officials have not been forthcoming with answers. We have some very strong concerns, he says. Aschman is adamant that ATHN is not for sale. No organization, not even the CDC, will have carte blanche access [to the data], she says. Shapiro insists that unless a patient grants written permission, the organization will only share information that has been aggregated and stripped of individual identifiers. We have no Machiavellian plans, Shapiro says. NHF is taking precautionary steps to assure that no patients rights are violated. At the moment, we take them at their word that [ATHN] will abide by all Health Insurance Portability and Accountability Act [HIPAA] regulations, Stanhope says. What were being assured is that any question will be evaluated on its value as a research question rather than a marketing question, says Stanhope. He expects NHF to monitor the situation over time. Were going to stay engaged with them, he says. workiNg ouT The deTails ATHN organizers are talking to various constituencies associated with bleeding and clotting disorders, including clinicians, researchers, advocacy groups and patients, laying the groundwork for the organizations mission. We will all be working together for a common cause, Lusher says. For the good of the hemophilia community, we all have the same goals. Core values for ATHN include improving clinical outcomes, facilitating continuity of care, fostering collaboration, maintaining confidentiality and conserving resources through a common infrastructure, according to a presentation given to HTCs. ATHN is collaborating closely with the CDC to avoid wasted resources and duplicate data entry, says Aschman. Though the clinical and operational committees are working

To Learn More...

To learn more about ATHN in the coming months, visit its Web site: www.athn.org. out some of the finer points of ATHNs work, Aschman says that some data points to be collected and analyzed are identical to those in the CDCs UDC project. ATHN will add self-reported information on bleeding episodes from patient infusion logs. Its a little more subjective, Aschman says of the latter. Although ATHN has not finalized its complete list of data points, Aschman says patients will be asked about such things as family history, risk factors, co-morbidities like HIV and hepatitis, allergies, bleed status, diagnostic tests, medications and treatments past and present. Patient data will be collected at HTCs during routine contact with patients and sent electronically to the central server. Until that point, the process mirrors an electronic health record, Aschman says. Data then will be stripped of patient identifiers and aggregated for analysis. For patients who consent to be a part of the CDCs data project and ATHNs research, the database will retain a limited set of identifiers as HIPAA privacy regulations allow. Questions ATHN may try to answer by analyzing patient data include: o How many patients are using plasma-derived vs. recombinant product for immune tolerance therapy (ITT)? o How long is the average treatment for ITT? o What is the success rate of ITT? o Is any product associated with a higher incidence of inhibitors? o If something should happen to a person with a bleeding disorder while traveling, how can a local practitioner access that persons information for treatment purposes? Kulkarni is optimistic that ATHN could help define the full spectrum of bleeding and clotting disorders. I think it could be a prototype for other disorders, she says. She foresees that other federal agencies, including the Health Resources and Services Administration and the National Heart, Lung and Blood Institute, may be able to access and augment the hemophilia and thrombosis database. Kulkarni also hopes that members of Congress will take note of the project as a model for public health surveillance. Stanhope envisions the data bolstering clinicians bargaining position with insurers, including Medicare, as well as helping payers develop more appropriate drug formularies for affected patients. Right now, though, those remain wishes for the future. ATHN is currently formulating its rules and processes for accessing the information; the Data Privacy, Security and Access Committee will define the rules through a peer-reviewed process. Our intention is that the data will be available to address a wide range of issues, Aschman says.

HEMAWARE

SEPTEMBER/OCTOBER 2007

By Neil Versel