Regulation in Regenerative Medicine: Patient Safety Has to Come First
Posted on October 1st, 2012 in Aastrom Clinical Trials
Dear Friends of Aastrom,
In July 2012, after several companies had been treating patients with unapproved stem cell therapies, the U.S. District Court in Washington affirmed the right of the FDA to regulate therapies made from a patient’s own stem cells. I believe this is a good decision that will protect patients, strengthen our industry and ensure the safety and therapeutic value of approved products.
The move to introduce appropriate oversight and regulation of cell therapy comes as no real surprise – the number of authorized cell therapy clinical trials has increased steadily in the U.S. over the past 10 years, from 12 trials initiated in 2001 to more than 50 trials initiated in 2011. While certain aspects of the U.S. regulatory process might be considered burdensome, I believe it is imperative that stem cell therapies be tested rigorously and to the highest standards. The fact that cell therapies are derived from cells found in the body does not eliminate this responsibility. Regenerative medicine has transformational potential, but that does not necessarily mean that each cell therapy technology or product will result in a safe and effective therapy.
Clinical trials often identify unexpected and unanticipated safety issues. Rigorous clinical trials also often find that some proposed products are not as efficacious as they need to be to balance their risks. For example, only one in four biologic treatments has ultimately proven to be both safe and effective, according to a BioMedtracker study. Marketing any therapy – for cancer, heart disease or any other disease – that has not been proven to be both safe and effective in rigorous clinical trials puts patients at risk and ultimately hurts all research organizations working to develop new therapies.
Some recent changes in the law include welcome regulatory reforms. For example, the Federal Drug and Safety Innovation Act (FDASIA), which President Obama signed in July 2012 to speed up drug development, helps Aastrom in two important ways. First, FDASIA has granted more therapies “Accelerated Approval” status, which reduces the number of regulatory steps necessary before a therapy is submitted to the FDA. Second, it also created a “Breakthrough Therapies” category to expedite the review of therapies intended for serious medical conditions, such as CLI and ischemic DCM. A streamlined approval process can help to reduce the cost and complexity of clinical trials, and will potentially make it possible for companies such as Aastrom to advance promising new therapies more efficiently in the years ahead.
Clinical investigators must also work with industry regulators to develop better regulation. The insights and recommendations of clinicians are key to the success of many clinical research programs in regenerative medicine, and we should encourage their participation in this process. At Aastrom, we have worked extensively with respected and well-known physicians in the vascular community to develop clinical protocols that will yield the best information in support of product marketing applications.
Currently, our cell therapy ixmyelocel-T is being evaluated in a Phase 3 clinical trial for critical limb ischemia and in a Phase 2 clinical trial for ischemic dilated cardiomyopathy. We are working hard to maintain the highest standards for our clinical trials, engage the most knowledgeable and experienced investigators, and give our cell therapy product the best chance of clinical, regulatory, and commercial success. We are also committed to presenting and publishing our data and other research findings in peer-reviewed settings to researchers, clinicians, patients and others involved in developing and using new treatments for patients with serious cardiovascular diseases.
There is already substantial evidence to indicate that regenerative medicine has the potential to improve medical outcomes, enhance quality of life and reduce overall healthcare costs for many patients. But none of these goals can be achieved without first ensuring that cell therapies are both safe and effective for patients. A rigorous clinical trial system and regulatory oversight are key to protecting patients, improving care and bringing much-needed therapies to patients and the physicians that care for them.