Treatment of Rare Diseases Must Be Improved
This article appeared in the Buffalo News on Saturday June 13, 2015.
Millions of Americans woke up this morning battling health problems from diseases like lupus, spinal muscular atrophy and Tardive dyskinesia. Illnesses like these are just a few of the thousands of rare diseases afflicting individuals across the country.
Costly medical bills, painful side effects or treatments and the psychological struggle of facing currently incurable diseases combine to take a tremendous toll on the lives of those suffering and the people closest to them.
This should not be the case. The United States is the world leader in medical innovations, but improvements can still be made in how we treat and cure rare diseases.
Of the over 7,000 known diseases, we have cures or treatments for only 500 of them. To change this startling statistic, I have joined with my congressional colleagues on both sides of the aisle to help craft the 21st Century Cures Act.
This legislation will modernize and advance health care innovation in America. It increases funding for National Institutes of Health grants used by scientists at the world-class universities in Buffalo and Rochester and restructures the drug approval process at the Food and Drug Administration in order to get new cures and treatments to patients more quickly.
I am proud to have authored several provisions included in the legislation, which will help incentivize innovation and defeat disease.
As someone who has worked in the biotechnology industry, I understand firsthand the burdensome and antiquated regulations that delay lifesaving drugs from getting to patients quickly.
Biomedical innovators must strike a balance between getting treatments to patients as fast as possible and making sure those treatments are safe and effective. It is the FDA’s job to ensure that occurs, but in order to expedite the development of lifesaving drugs, the FDA approval process must be modernized.
For this reason, I introduced a provision that enhances and accelerates effective clinical trials. It allows drug developers to customize trials to an individual’s DNA by utilizing what we know about the human genome. Coupled with modern statistical modeling, researchers are able to zero in on how a drug trial is affecting its participants, keeping patients with genetic makeups that the drug is successfully treating and removing patients with genetic makeups that are not responding to treatment.
These small policy changes will unleash new, groundbreaking therapies to cure and treat a variety of rare diseases.
Patients are demanding a fresh approach to drug approval and biomedical research. The 21st Century Cures Act will provide America’s medical innovators the guidance they need to lead a new age of medical innovations.