The Value of Life-Saving Drugs

Feb 24, 2016

Guest post by Candace Lerman, Rare Disease Advocate

I am alive today thanks to off-label use of Rituxan to treat my rare disease, Immune Thrombocytopenia. Without the treatment, I could have suffered a fatal bleeding episode and I certainly would not be enjoying the quality of life I have right now. Every day is a reminder that victories in medical research can have a lasting, positive impact for patients. Some may argue I beat the odds, I see it as utilization of data from studies and patients. Now I hope to see more stories like mine thanks to advancements in biotech. There is no reason why we cannot create a movement that combines patient experience with research and development of new drugs and devices. If we are serious about getting anything done, combining forces will create the most rapid changes.

 It is important to recognize the strain in the relationship between pharmaceutical and biotech companies with patients. This is not beneficial to either side because we both have the same goals; find cures and treatments for different diseases and disorders. The best way to extend the olive branch is through patient success stories. So often we hear about the failure of drugs, the death of patients. We seem to forget that every day doctors, researchers and patients are setting new milestones in medical innovation. This is what we strive for and everyone can play a part in making a difference from treating patients, creating drugs, participating in clinical trials and furthering conversation. Healthcare providers, industry and patients can all come together to work towards our shared goal.

There are a lot of important pieces of legislation working through Congress at the moment. In a few weeks, both patient advocates and healthcare industry leaders will be in Washington to discuss ways to improve funding for medical research and the drug approval process. It pains me to think how many potential treatments are available for rare disease patients, but cannot be utilized because there have not been enough studies to determine effectiveness. This is something that Congress hopes to change through the Orphan Product Extensions Now Accelerating Cures and Treatments (OPEN ACT), which is currently in the Senate. The bill will allow FDA approved drugs to be repurposed for rare disease patients. With over 30 million Americans struggling with a rare disease, this is a step in the right direction.

 I appreciate that there are people out there who I have never met that are working towards finding a cure for my rare disease, and the more than 7,000 others that our population is plagued with. Many times the industries developing drugs and devices are under appreciated and go unnoticed. With scrutiny facing drug companies in recent months, the focus has turned away from the amazing people fighting to improve the lives of patients. We have to turn the attention back to our goals.

This Rare Disease Day appropriately falls on February 29th. I look forward to encouraging fellow patients to participate in the ongoing dialogue and join the mission for cures and treatments. Together, we can accomplish great things in medical innovation.

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