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What's in the Data?

Project to collect and analyze information about people with hemophilia and other bleeding disorders

By Neil Versel | 03.15.2010
Originally Published September 2007 | Updated March 2010
Data about hemophilia

A lot has changed in the more than 30 years since hemophilia treatment centers   (HTCs) 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 128 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 determining the best way to care for each of their patients. New medical research and guidelines from organizations like the National Hemophilia Foundation’s (NHF’s) Medical and Scientific Advisory Council (MASAC) can certainly help doctors make the best treatment decisions. But more data about which medical decisions work—and which don’t—would also be helpful. In addition, staff at HTCs have long been concerned that they don’t have enough information to draw upon for the Food and Drug Administration’s required post-marketing surveillance of blood products.

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 complications, including joint disease, in people with hemophilia. The mandate currently is broad-based and includes monitoring of bleeding complications as well as clotting disorders in all ages of both sexes.

In 2005, the regional core centers, overseeing the network of HTCs, adopted Lab Tracker, an electronic medical record and disease-management program by Ground Zero Software. It can collect clinical data and submit required UDC data to the CDC. To ensure long-term viability of Lab Tracker, the regions, in collaboration with the CDC, started development of WebTracker, a Web-based version of Lab Tracker. Having data centrally located on the Web relieves the HTCs of the burden of installation, maintenance, security and data storage. This is important, because some of the HTCs are small, independent operations lacking sophisticated information technology staff.

But CDC staff admit that the UDC database was initiated specifically for public health surveillance to monitor the extent of complications such as blood-borne infections and joint disease. “It doesn’t fulfill the clinical needs [of hematologists and the HTCs],” says Roshni Kulkarni, MD, former director of and current senior medical advisor to the Division of Blood Disorders at the CDC’s National Center on Birth Defects and Developmental Disabilities in Atlanta.

In 2006, with support from the CDC and a grant from drug-maker Novo Nordisk, a new nationwide data collection project was formed called the American Thrombosis and Hemostasis Network (ATHN). ATHN was founded out of a desire by the HTC network to leverage the Lab Tracker national data infrastructure to support the data collection capacity of the HTCs and to develop a research engine that could benefit a broad bleeding and clotting disorders community.

Nearly 23,000 patients with bleeding disorders have consented to participate in the UDC Project, but the database does not contain detailed treatment data that could determine why some patients do better than others.

“There has been a need in this community to collect not only outcomes data, but treatment data,” says Kulkarni. “It is my hope that in the future, collaborations between federal agencies such as the CDC, HTCs and ATHN would encourage more public health research; ATHN through the National Heart, Lung, and Blood Institute [NHLBI] has the potential of doing clinical and basic science research with the Hemophilia & Thrombosis Research Society [HTRS]. Continued support for the basic infrastructure of the HTC by the Health Resources and Services Administration [HRSA, in the US Department of Health and Human Services] for access by this population will allow this community to survive and thrive and be a role model for other chronic diseases.”

Because 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, there’s no good way to share data among the treatment centers,” explains Ray Stanhope, former NHF board chair and current board member. “A large, unidentified community is involved.”

The plan is for ATHN to help make treatment data more accessible and usable.

The Time is Now

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 Foundation, an independent, nonprofit enterprise that forges effective partnerships between the CDC and others, 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, president and CEO of ATHN. “The overall purpose of ATHN is providing stewardship to the HTC national database.”

Input from the clinical and administrative committees, composed largely of HTC physicians, 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.

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 immediate past 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.”

Participants chose Shapiro, along with Jeanne Lusher, MD, director of the Hemophilia, Hemostasis and Thrombosis Program at Children’s Hospital of Michigan in Detroit, as board co-chairs to guide the fledgling organization through its first year of operation. They helped form committees, developed a governance structure, hired executives and set the scope of work for each committee.

“A variety of people from diverse backgrounds weigh in on ATHN’s activities. Its board, for instance, is composed of 19 voting members, some of whom are hematologists, researchers, and staff from the CDC, NHLBI and NHF’s chapters. In addition, there is a community liaison group, with representatives from NHF, Hemophilia Federation of America, the Committee of Ten Thousand (COTT), HTRS and others. “The group makes sure our goals are aligned in using resources efficiently,” says Ashman. “That way we’re not overlapping or working at odds with each other, but rather in a collaborative manner.” Members of the industry liaison group, from pharmaceutical and biopharmaceutical companies, serve as checks and balances. “They make sure that the things we’re doing are useful to them and the data can be used to support their research initiatives,” Ashman says.

Resources Ready

ATHN already has some resources to share with consumers. Among these are the ATHNready Personal Health Report, electronic health information for use in an emergency. Stored on a wallet-sized flash drive, the personal health data are provided to the patient by the HTC and are secure and ready when needed. “It provides key medical information, along with patients’ medical and drug history, which is particularly helpful for a treater who is not familiar with the patient,” says Aschman.

ATHN’s HTC finder is an interactive map of all of the HTCs across the country. After clicking on one of the HTC pushpins on the map, the user is provided with the center’s name, address, contact personnel with phone numbers and directions. “When traveling on vacation or for business or in case of emergency, this is a very helpful resource,” Aschman says.

With the donation of Baxter’s Advoy database system in late 2009, ATHN will assume training and stewardship for its version, ATHNAdvoy. The year 2010 will be one of transition, says Aschman, as HTCs and patients submit consent to have their data moved to and managed by ATHN. “We can help treatment centers more directly use the combined information—what they collect clinically and the patient-reported bleed and infusion information—more efficiently,” says Aschman. In addition, all three of the commercial bleed and infusion logs now interface with WebTracker, saving time that used to be spent manually entering information.

Another ATHN accomplishment is the creation of a patient registry for plasminogen deficiency, a rare clotting disorder. Working with the Indiana Hemophilia & Thrombosis Center, with funding from Baxter, ATHN helped develop the online registry, which will help document such unknowns as patients’ natural history and how many people have the disorder.

Privacy Concerns

Despite the potential benefits of such a large-scale data collection effort, there are people in the bleeding disorders community who have posed serious questions about the project. Some worry that the information in the database won’t be completely secure and confidential.

“Why are we privatizing hemophilia data?” asks Corey Dubin, president of COTT, a group whose core constituents have hemophilia and contracted HIV/AIDS 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 infrastructure—Dubin 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. He says ATHN officials have not been forthcoming with answers. “We have some very strong concerns,” he adds.
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 ensure that no patient’s 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 we’re 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. “We’re going to stay engaged with them,” he says.

ATHN’s Data Privacy, Data Access and Security Committee has taken its marching orders seriously. “Our patient authorization process for patients to opt into the database goes above and beyond HIPAA requirements,” Aschman assures. The patient data are nonidentifiable, she says. According to the HIPAA privacy rule, Aschman says that 16 categories of identifiers are stripped from the health data, including the patient’s name, home and e-mail addresses; phone and fax numbers; and social security and medical records numbers. “We’ve put in place a process to make sure that patient data are safe, secure and appropriately portable.”

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 organization’s mission.

“We will all be working together for a common cause,” Lusher says. “For the good of the bleeding disorders 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.

Patient data will be collected at HTCs during routine appointments and sent electronically to the central server. Although some data points to be collected and analyzed are identical to those in the CDC’s UDC Project, this will not require duplicate data entry by the HTC staff. Information in WebTracker can be divided into data sets for different purposes. From the national dataset of information gathered through the HTC network ATHN will manage its own smaller data subset, called the ATHNdataset.

The type of information that will be sent to ATHN includes:

  • Diagnosis
  • Date when first diagnosed
  • Family history
  • Types of bleeds
  • Medications and treatments, including adverse effects and allergies
  • Routine care
  • Trauma

In the future, the ATHNdataset may be able to answer such research questions as:

  • Based on patient age and dosing regimen, what’s the optimal use of prophylaxis to prevent bleeds?
  • Is any product associated with a higher or lower incidence of inhibitors?
  • For patients who track and self-report bleed and infusion information, is the number of hospitalizations reduced?
  • How many patients with hemophilia are covered by the state’s high-risk insurance pool?

Prototype Potential

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 HRSA and NHLBI, may be able to access and augment the hemophilia and thrombosis database.

Aschman describes ATHN as transitioning from a development organization to one that can deliver value to the community. “The Web-based infrastructure is now a reality,” she says with pride. “It is being implemented and deployed to HTCs around the country. It will provide a robust community resource.”