Journey to Chronic Rare Disease Treatment

Living with a rare disease is difficult. Accessing treatment can be hard for many. Many rare disease drugs are expensive. Some are even subject to price gouging. The FDA provides incentives under the Orphan Drug Act of 1983. These help create drugs for rare diseases. This includes 7 years of market exclusivity. This increases prices by preventing competition.¹

Your treatment plan may need to change over time. The body can build a natural tolerance against certain substances.

My journey finding a narcolepsy diagnosis and treatment plan

I would give my past self some advice. I would say it is a marathon, not a race. It is easy to hope symptoms will vanish with 1 treatment. For most, this is not the case.

Here are 3 things I wish I'd known when starting.

1. Finding the right narcolepsy treatment plan takes time

Finding the right plan for your rare disease is not always easy. We face many disadvantages. These include issues with accessing specialty drugs. We also have fewer treatment options.

When I was first diagnosed with narcolepsy, I was frustrated to find that it took months to get access to treatment. There were so many insurance and medical hoops that I had to jump through. It made the experience of being diagnosed with a rare disease feel that much more vulnerable. I wish I could go back in time and tell myself to keep holding on—that things would get better, but it would take a lot more time than I'd initially thought.

2. Listen to your body when trying new narcolepsy drugs

Side effects are real. Their effects can range from mild to deadly. It is important to know all the side effects of a drug when starting it. Listen to your body. If something doesn't feel right, bring it up with your doctor. And if they dismiss your concerns, it might be a sign to get a second opinion! On that note...

3. Do not be afraid to switch narcolepsy doctors

People with rare diseases have less access to experts. I was scared of losing treatment. I stayed with 1 doctor for too long. This doctor ignored my concerns about drug side effects. They said it was 1 of the only options for narcolepsy. That was not true.

Your doctor might put profit or ego ahead of your needs. If so, get a second opinion. This helps you find more treatment options. People employ their doctors. You can "fire" them for any reason!

What do you wish you knew before starting narcolepsy treatment?

Finding an adequate plan for my rare disease took years. Do not blame yourself for the long process.

What do you wish you knew before starting your rare disease treatment journey? Share in the comments below.