Gut Check: What Are Small, Simple Ways You Could Be Helping People in the Fight for Their Lives?

In my family, the presence of chronic rare disease has placed us in a constant fight. For generations we’ve watched the family members battle Alpha-1 Antitrypsin Deficiency. And, on any given day, we have had to support one another in the battles with health care too. Not with individuals, but a system not designed for rare conditions.

As my family and I met the milestones of treating my father’s Alpha-1 including an eventual lung transplant, he took on advocacy with gratitude, knowing he could use his intimate knowledge to help others. I followed in his footsteps. Today, the focus of my life’s work with Optime Care is on advocating for people with rare diseases so they can find the best care possible and stick with effective care plans.

I would not trade a moment spent with my father — even the ones that were hard, the ones when we had to do real battle. And I love the work I do today as a result.

At the same time, I know that if more people on the pharma manufacturing side of things knew just how much of a family’s time, energy, and sanity is collapsed into a term like “be your own advocate,” we would be seeing real changes in the small ways we care for patients.

The fight for people with a rare condition begins before diagnosis. It can take seven years and three to 5 different doctors. In the case of Alpha-1, even if you’re having breathing problems, unless you’re a 40-year-old with 40% lung capacity, most doctors won’t think of it. Maybe they will think early-stage emphysema. And the older you get, the less likely you are to be tested for Alpha-1. Once you hit 60, it’s rare that they would test for it. Most likely, the doctor will just say, “Take this inhaler, and I’ll see you in a year.”

What if you don’t know this reality? And, let’s be honest, most people don’t unless they have lived it.

What we do know is that successful patients have advocated for themselves. But this also means, as health care professionals, we’re asking these people who are suffering the condition to find the strength, time, and energy to fight.

Essentially, here is the expectation on people with the rare conditions and their families: Navigate the science of disease, the communications with physicians, and negotiate with insurance.

Given that reality, here is my question for you: What are you, as a leader in health care, with the knowledge and expertise in treatment, doing to meet these patients and families in the fight?

Chances are that you could be doing more. The good news is that it has been my experience, both as a patient advocate and with the work we do at Optime Care, that small gestures go a long way.

One way is doing something each day to shorten that length of time to diagnosis. Ask yourself, how are you raising awareness, detection, and education on this disease rather than just promoting a product? If you are on the manufacturing side, you likely have access to the latest information to equip and persuade physicians to test for your disease state. Find a way to inspire them. Get that into their hands.

Once a patient receives diagnosis and is on your therapy, it has, again, been my experience that small gestures go a long way. These people are in a fight, so any relief makes a huge difference in their mental health state and their overall wellness. Something as simple as providing a ride to the hospital or arranging for a night in a hotel can inspire a patient to follow through on the care plan.

Small gestures make a big difference to people in a fight.

Too often, I have seen fear from pharma manufacturers. They do not want to stick their neck out to get involved with advocacy in impactful ways, and they will use regulatory language as cover. That language has trained them to turn a blind eye and keep distance from patients. While it might feel like risky proposition, and while it can open you to more criticism, it is why you got into health care in the first place: to help patients. So do it.

I call on you: Focus on doing what’s right, meeting patients in the middle and being a voice of clarity in the midst of uncertainty. Then, and only then, you can claim to be patient first.