There has been a great deal of criticism of pharmaceutical and biotechnology companies in recent months. Most of the anger surrounds drug pricing, and many in the rare disease community have participated. While affordable healthcare is a priority and we need access, an even more alarming need is actually treatments for orphan disorders. Many rare disease patients are struggling day to day, without anything to help them manage their symptoms and conditions. Drugs for small populations cost money, the FDA is particularly critical of rare disease drugs making investments risky and the cost of treatments sky rocket.
The “industry” is our friend and should be viewed that way. Without pharma and biotech companies, we would not have most of the amazing drugs we use today. If we continue to attack our partners in business, we will see less and less willingness to work with our community. I have been observing rare disease advocates discussing their experiences at various conferences in the past few months. Many have had conversations with groups that are all over the spectrum. Some people are very open and aware of the need for orphan drug development, while others don’t seem to have a clue what is going on.
In a way, every patient is an ambassador for rare diseases and the critical need for industry participation in finding cures and ultimately treatments for our disorders. We need to encourage innovation, investment and teamwork for the future.