In a recent support group meetup, one participant asked, “Why is it so hard to find a cure?” There are countless researchers, clinicians, and nonprofit administrators who are dedicated to finding a solution, but the road to a cure is fraught with obstacles that are common to all rare disease researchers, as well as unique challenges specific to PF. In this blog post, we will explore why PF poses such a significant challenge to researchers and how you, as readers, can play a crucial role in speeding up the process. Together, we can fight PF right now.
Here are some of the top reasons curing a rare disease can be so difficult:
- By definition, rare diseases like PF affect only a small portion of the global population. This scarcity makes it challenging to garner adequate attention and funding compared to more prevalent health conditions. As a result, finding a cure becomes even more daunting.
- With a small number of patients available for clinical trials, it becomes difficult to recruit sufficiently sized cohorts. This makes it hard to design studies that will yield statistically significant data.
- Rare diseases are often poorly understood due to the lack of available data and research. This limited knowledge makes it challenging to identify the underlying causes of disease onset and to develop targeted treatment strategies. In many cases, the causes are unknown and considered ‘idiopathic,’ as is the case with IPF.
Beyond the common obstacles faced by all rare disease researchers, PF presents its own set of unique challenges:
- PF is notorious for being difficult to diagnose. Some patients experience a subtle and gradual onset of symptoms over the course of many years, leading to delayed diagnosis. Even when symptoms begin more rapidly, it can still be time-consuming and challenging to receive the appropriate diagnosis. As frustrating as this is for patients, it is also a challenge for researchers and clinicians, because identifying the disease early is crucial for understanding how PF progresses – and how to treat it.
- Not all patients with PF qualify for PF-related clinical trial participation, or are able to participate due to accessibility barriers. The inclusion criteria in clinical trials can be quite strict, and disqualify many patients based on factors beyond their control, such as age or pre-existing medical conditions. Even for those who do qualify, it might not be feasible to participate due to the area they live, the time it’d take to travel to the research/medical center, or the difficulty of traveling with supplemental oxygen.
- PF is not a singular condition; it encompasses various subtypes with different underlying mechanisms. It also affects people of all backgrounds, from all around the world. This quality of being diversely characterized is known as ‘heterogeneity’ and makes it challenging to develop a one-size-fits-all treatment approach.
Even though finding a cure is difficult, it is possible when patients, caregivers, relatives, clinicians, researchers, and all other stakeholders collaborate. Here are a few ways you can help us fight PF right now:
- Raise awareness. Share information about PF with friends, family, and your social network. Raising awareness about the disease can lead to new collaboration opportunities and other forms of support.
- Advocate for funding and legislation. Write to your elected representatives, urging them to support funding for rare disease research and policies that facilitate research and drug development for patients with PF.
- Spread the word about reputable clinical trials, and participate if you can. Clinical research is crucial for testing potential treatments and advancing medical knowledge – but beware of predatory companies trying to get your personal information.
- Support nonprofits and research organizations: Consider volunteering for and/or donating to nonprofit organizations dedicated to PF research. Your perspective, skills, and contributions can significantly impact the progress of ongoing studies.
Rare disease research demands our attention and action. By joining hands and contributing in any way possible, we can be instrumental in transforming the lives of those affected by PF. Together, we can bring hope to those facing the uncertainty of this debilitating condition. The journey to finding a cure for PF is undoubtedly challenging, but you might be surprised by how fast scientific breakthroughs can happen!
Stay In Touch
PF NOW! hosts virtual support groups on Teams multiple times each month. Not only will you strengthen your network of connections but you’ll learn firsthand how those with PF best look after themselves. PF NOW! also has a Facebook group whose active community shares their PF journey and their tips with others. New to online support groups? Download our free step-by-step guide for online advocacy here.
Never disregard professional medical advice or delay in seeking it because of any information received from us.