How advocacy organizations support rare disease research

A columnist clarifies the role these organizations play in developing therapies

Samuel Kirton avatar

by Samuel Kirton |

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Are you angry? If so, who are you angry with and why?

I follow a number of social media groups associated with different rare disease communities. Lately, it seems that people are angry with the very groups advocating on their behalf. I must admit, that intrigues me a bit.

When I was diagnosed with idiopathic pulmonary fibrosis in January 2017, I gained my own membership to the rare disease community. Another rare condition, ataxia, had greatly affected my family, taking the lives of three of my stepsiblings. By the time of my diagnosis, I’d been serving on the board of directors for the National Ataxia Foundation for many years and involved in rare disease advocacy for many more.

Because I’ve worked with several advocacy organizations in the rare disease space, I want to help explain their role to improve understanding and minimize frustration. A common misconception is that these organizations develop therapies or cures for their patient populations. In general, they do not. That misunderstanding seems to be a primary source of frustration in some social media channels.

So what do these organizations do?

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We Can All Play an Important Role in Rare Disease Advocacy

The role of the PFF

The Pulmonary Fibrosis Foundation (PFF) provides a clear mission statement:

“The mission of the Pulmonary Fibrosis Foundation [PFF] is to accelerate the development of new treatments and ultimately a cure for pulmonary fibrosis [PF]. Until this goal is achieved, the PFF is committed to advancing improved care of patients with PF and providing unequaled support and education resources for patients, caregivers, family members, and health care providers.”

Nowhere does it say that the PFF is developing new drugs. Rather, the foundation is working to accelerate the development of treatments and a cure.

The difference is significant. The PFF is not creating a drug, but working with scientific and pharmaceutical communities to support the researchers who are seeking answers. Following are several examples of how the foundation provides that support.

The PFF Scholars program accepts applications from researchers who are studying pulmonary fibrosis. Proposals are accepted when a project shows promising potential of improving our understanding of PF, and selected researchers are granted $100,000 to support their investigations. I encourage you to read more about the research projects that have received support from this program.

The PFF also helped launch PROLIFIC, or the Prognostic Lung Fibrosis Consortium, which aims to help scientists develop more targeted PF treatments. PROLIFIC is developing tests to detect certain blood protein biomarkers, which would hopefully predict the treatments that offer the most benefit to individual patients.

In addition, PFF advocates — including patients, caregivers, and family members — seek support annually from Congress. The Department of Defense’s Peer Reviewed Medical Research Program (PRMRP) provides funding for PF research, so advocates work to ensure that pulmonary fibrosis remains an eligible topic in the PRMRP each year.

The PFF also nominates patients to speak at PRMRP panels reviewing research applications. Patient voices provide critical commentary on how research projects may affect our community. I’ve served as a consumer peer reviewer, which was a rewarding experience.

Understanding the role of advocacy organizations is important for everyone in the PF community. It helps us to set expectations and understand how we can contribute to the pursuit of new therapies and a cure. Working together, we can all make every breath count.

Note: Pulmonary Fibrosis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pulmonary Fibrosis News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to pulmonary fibrosis.


Jason avatar


Indeed, I am so angry that the recipients of U.S. Government-sponsored research funds do not investigate the real mechanism of the disease. Rather they try to develop a drug for the disease for their own benefit. We have to make a change NOW!!!

Samuel Kirton avatar

Samuel Kirton


Thanks for reading my column and for your perspective. I would like to better understand your comment. Short of a public health crisis, such as COVID-19, the government does not typically directly fund drug development in the private sector.
The Congressionally Directed Medical Research Program (CDMRP) is an example of government sponsored research funds being spent for the purpose of developing a better understanding of pulmonary fibrosis. It has been my experience as a consumer reviewer for CDMRP grants that the funding was not to any drug development company. It did go to investigators and researchers seeking a better understanding of this disease. Ultimately, that research is a building block for further research. Every scientist can benefit from that research whether they are in academia, medical, or the pharma sectors. Precluding pharma from using the results of that research seems counter to the goal of finding a more effective therapy and a cure. Does pharma benefit from that government funded research? Absolutely. Should they? Again, my answer is absolutely. Perhaps it is a matter of perspective but my desire is to see the mystery solved. Drug development cost money and pharma does attempt to recover the cost of that development over the life of the drug.

Sam ...


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