New PFF Registry Announced

At the ILD Support Group meeting yesterday, there were five members present who have received lung transplants. It was heartening to see them all doing so well. The beautiful Susan has begun the transplant process and was sick of feeling so sick. As a very self sufficient, independent, strong woman, she hated being tethered to supplemental oxygen. We did have a lovely lunch and a great chat before the meeting.

Sally told us about the recent Pulmonary Fibrosis Foundation's (PFF) conference and about the famous Dr. Beall. He is the genus behind major research, buying orphan drugs, buying and selling pharmacies and fundraising for the Cystic Fibrosis Foundation.  The prognosis for a patient with CF went from 5 years to 49 years due to his efforts. One of his brilliant ideas was to develop a registry to input variables and to analyze the data. An example of what was discovered with this method was that CF kids used to have summer camps together and lots of meet and greets. They realized that they would become ill afterwards, discovered that they carry a bad bacteria that they passed to other CF kids, so now the goal is to keep them in separate classrooms, separate waiting rooms, each examination room must be wiped down between patients, that sort of thing. All of this was discovered because of the data in the registry. Here is the CF Foundation web site: http://www.cff.org/aboutcffoundation/presidentmessage/

Dr. Beall was invited to speak at the conference because of this successful process and PFF is beginning to follow his examples. It was announced at the conference that PFF will be beginning a patient registry at a number of hospitals in the US beginning in 2014. Information about the registry is below. If you wish to read more about PFF, their web site is: http://www.pulmonaryfibrosis.org/

PFF PATIENT REGISTRY

WHAT IS THE PFF PATIENT REGISTRY?

The PFF Patient Registry is an online database of “de-identified” patient information that will be managed by an independent Data Coordinating Center (DCC). The registry data will be available to all qualified investigators. The Registry will provide access to accurate, validated, and standardized patient information. A patient registry can provide important information for physicians and researchers that will enable them to better understand the disease and develop more effective therapies.

Where is the information in the PFF Patient Registry collected?

Patients can enroll in the Registry at designated PFF Care Centers. This helps to ensure that patients are accurately diagnosed and that their information will be collected in a standard manner.

What kind of data is collected?
Data in the Registry will include:

• Demographics (e.g., age, sex, race)
• Medical history
• Treatment history
• Co-morbidities
• Physical exam findings
• Test results: pulmonary function, six-minute walk test, laboratory tests, bronchoscopy, HRCT, pathology, cardiac tests
• Quality of life questionnaires
• Hospitalization history
• Diagnosis

What does the data tell researchers?

Data can help researchers make new observations about who gets a disease and why. In a similar way, data can also help health care providers assess the effectiveness of specific interventions including pulmonary rehabilitation, nutritional counseling, and new medications.

Some of the questions the PFF Patient Registry will help answer are:

• What is the frequency of a specific diagnosis by geographic region?
• Does the natural history vary by geographic region?
• What are the benefits and risks of specific interventions (e.g., bronchoscopy, pulmonary rehabilitation, lung biopsy, medications, etc.)?
• How uniformly are patient care guidelines followed?
• Validation of quality of life questionnaires and patient reported outcomes.
• Assessment of an intervention or medication on an individual's quality of life.

How will the PFF Patient Registry benefit patients?

Develop "Best Practices" for Patient Care
The PFF Patient Registry will help the Care Centers develop treatment protocols for managing PF patients. Registry data will allow Care Centers to assess current practices, patterns, and eventually determine what are evidence-based "best practices."

Collect Accurate Patient Information and Clinical Data
Over time the PFF Patient Registry will become the largest collection of PF patient data in the world. Importantly, data will be collected in a standard fashion at all sites. This will ensure that accurate and meaningful results are obtained when the data are analyzed.

Facilitate Clinical Trial Enrollment
Clinical trials help prove to regulators that a drug or medical device is beneficial to patients and that the benefits outweigh the risks. Therefore, it is important that companies test the drug or device on the types of patients the product is meant to benefit. Finding a population of patients who meet the trial criteria can be a difficult task, especially for those who work with rare disease. The PFF Patient Registry will help facilitate enrollment in and expedite completion of clinical trials.

Where can I find PFF Patient Registry sites?
PFF Patient Registry sites are located at the following Care Centers:

• University of California, San Francisco
• University of Chicago
• University of Louisville
• University of Michigan
• National Jewish Health
• University of Pittsburgh
• Vanderbilt University
• University of Washington
• Yale University

How is the PFF Patient Registry funded?
The PFF Patient Registry is funded through the generous support of corporations, trusts, foundations, and individuals who realize that the creation of a registry is a fundamental to improving the lives of those affected by PF.

For more information, call 888.733.6741 or +1 312.587.9272 from outside of the US.