New Treatments – How Much Longer?


After months - maybe even years - of feeling like a hamster on its exercise wheel chasing a solution to your child’s health issues, you are devastated to finally learn that your loved one has a rare disease that is so novel, a cure has yet to be found. To manage the disease and provide your child with a shred of quality of life, you have your child cycle through several different medications, some of which may alleviate a few of their symptoms, but almost none that alleviate all of the symptoms or even the major burden of the disease. Why is that the case? How difficult can it be to find that one medication that could be the ultimate remedy? What does it take to find a new medication to help these children? Why does it take so long for a drug to get approved?

I’m sure these are some of the questions that have been occupying your mind as you navigate the rare disease journey. I am hoping, through this blog, to answer these questions and many others you may have pertaining to the clinical trial process.

How difficult can it be to find new medications?

The clinical trial research path is long, expensive and arduous for drug developers, clinicians and patients. The estimated cost to develop a new medicine that gains marketing approval is $2.6 billion according to a study published by the Tufts Center for the Study of Drug Development (Journal of Health Economics, May 2019, https://doi.org/10.1016/j.jhealeco.2016.01.012). Where does a drug development company get this type of funding to develop a new drug? If the company doesn’t have a product on the market (commonly referred to as a research and development company or R&D company), it is not generating revenues and therefore, has very limited financial resources to sustain its research. It is necessary for these R&D companies to rely on the investment community for funding to support their research. Such funding typically comes in waves and is usually aligned with achievement of milestones. Obtaining adequate and timely funding is essential for an R&D company to conduct clinical trials in an efficient manner. Without appropriate funding many R&D companies need to halt or delay their clinical trials.

Funding is only piece of the puzzle. The regimented process to advance an investigational drug through clinical development is highly regulated. As noted in Figure 1 below, it can take 12-15 years to move an investigational drug through clinical trial research to approval (the drug development and approval process).

With limited funding and multiple years necessary to execute trials, the process to find new medications is very difficult.