Guest Column | September 5, 2019

Patient Group Advances Clinical Trials, Diverts Patients From Unregulated Therapies

By Gregory Cosgrove, M.D., Chief Medical Officer, Pulmonary Fibrosis Foundation


For a patient with a debilitating disease, such as pulmonary fibrosis (PF), moments of hope can be few and far between. Many of these patients search desperately for new therapies to improve their quality of life, or possibly cure their condition, sometimes outside of the approved treatments prescribed by their healthcare providers.

Patients put their lives in our hands to help them deal with a wide array of conditions. As healthcare professionals, we must ensure they receive accurate information regarding all their treatment options and still increase their access to the latest regulated therapies. It is imperative that the medical community work together to accelerate the development of new treatment options to improve quality of life while eliminating the necessity for patients to seek potentially unsafe unregulated therapies.

Clear The Path To Approval

Recent reports of stem cell therapy tragedies across the country have shone a light on the need to address patient demands with more available treatment options. Without safe options, patients facing a devastating diagnosis may turn to unregulated stem cell therapies that promise a miracle but may actually do more harm than good. The regulatory agencies established standards to thoroughly evaluate therapies, and while the process can be time-consuming, it leads to the development of the safest treatments for patients with proven efficacy.

Can we accelerate this process safely and efficiently? We must. Through partnerships within the healthcare industry, we must expedite the evaluation of promising agents. This will not only encourage and support innovation, but also help enhance patients’ trust in a healthcare system focused on quality and results.

Through partnerships among patients, providers, regulatory agencies, and pharmaceutical organizations, we can fast-track the evaluation and approval of potential therapies, while reinforcing the benefits of regulatory agencies dedicated to safety and efficacy. When a treatment shows significant promise to positively impact a patient population — as was the case with HIV treatments — it should be prioritized and moved swiftly through the appropriate regulatory channels. Currently, there are two pathways — accelerated approval and prior review — both of which speed the evaluation.

Increase Access To Data For Research

The Pulmonary Fibrosis Foundation (PFF) actively engages with the PF community to develop essential programs for those living and working with the debilitating disease. This community is eager to help healthcare providers and researchers learn more about PF causes and treatments through sharing de-identified medical information and participating in clinical trials. Finding the best channels to do so, however, can be difficult.

To simplify the process, the foundation created the PFF Patient Registry, which provides clinicians and scientific researchers with detailed medical information voluntarily provided by more than 2,000 patients with PF. The registry contains information about how each person’s diagnosis was made, their symptoms of PF, medical test results, medications used for treatment, and regularly updated health assessments.

All of the data comes from patients being treated at facilities within the PFF Care Center Network, a group of 68 medical centers nationwide dedicated to improving the lives of individuals with PF. The PFF Care Center Network supplies the infrastructure to facilitate high-quality clinical and translational research to help providers better understand PF and how to care for patients. Providers at the Care Center sites use a multidisciplinary approach to deliver comprehensive patient care, engage in patient-oriented PF research, and work with the PFF to provide educational materials and engage the local community through PFF programs.

Those in the pharmaceutical industry researching PF causes, symptoms, and treatments can utilize the PFF Patient Registry to review thousands of detailed data points as they search for answers to key questions about PF. Having access to a specific patient population’s data will help advance research and development of new therapies to improve quality of life. Further expansion of the registry will lead to the discovery of the causes of PF and, hopefully, a cure.

Expand The Research Pool

It is equally important to increase clinical trial participation within the PF community. To engage more patients in the clinical trial process, the PFF recently launched its Clinical Trial Finder, which helps patients with PF connect with investigators in their communities to get more information about upcoming trials and how to join. By making it easier and faster for patients to access clinical trial information, the tool is helping to accelerate the development of new therapies.  All PF clinical trials on the site are registered with

Pharmaceutical companies, educational institutions, and research organizations conducting clinical trials for the treatment of PF will appear in the Clinical Trial Finder tool, allowing patients to review the information and contact the investigative team directly. Pharmaceutical organizations should promote their clinical trial opportunities and provide as much information as possible, as many patient populations are ready to participate but unsure how to get involved.

The PFF Clinical Trial Finder is the first step toward creating the PFF Therapeutics Network, where dedicated healthcare professionals from across the industry will pool their resources and expertise to reach the shared goal of finding a cure for PF.

To improve patient care, we must advance the research, evaluation, and approval processes to respond to needs faster and communicate to patients the dangers of unregulated therapies that often fail to live up to their promises. By leveraging available patient data and increasing clinical trial participation, the healthcare community can work together to develop new, innovative therapies for PF.

About The Author:

Gregory P. Cosgrove, M.D., is the chief medical officer of the Pulmonary Fibrosis Foundation, associate professor of medicine in the Interstitial Lung Disease Clinic at National Jewish Health, and in the Division of Pulmonary and Critical Care at the University of Colorado School of Medicine.