Wednesday May 22, 2013 – Congressmen Morgan Griffith (R-VA), Scott Peters (D-CA), and Michael McCaul (R-TX) introduced H.R. 2090, the Patient Choice Act of 2013, which aims to speed up the Food and Drug Administration’s (FDA) approval of drugs for patients. Among other reforms, the Patient Choice Act would create a fast track provisional approval process for innovative drugs and treatments, while giving patients with terminal diseases the option to purchase these new therapies at their own expense.
Currently, it takes approximately 15 years and $1 billion to bring a new drug to market in the United States. Meanwhile, over 500,000 patients die each year of cancer alone. One in two men and one in three women will be diagnosed with cancer in their lifetimes. For diseases like melanoma, Lou Gehrig’s disease and Parkinson’s disease, either very few drugs are available or those in the pipeline cannot make it through FDA’s delays and regulations. The Patient Choice Act, first introduced by former Congressman Brian Bilbray (R-CA), aims to address these issues.
“Patients and their physicians need and deserve options as they battle deadly diseases,” Congressman Griffith said. “For patients whose doctors have exhausted current medical options and have been told that the end of life is nearing, why should the federal government interfere if the patient wishes to spend their own money on an experimental treatment plan? The Patient Choice Act would empower a patient faced with that dilemma to help move the ball down the field in the area of medical science and medical knowledge. In rare cases, the individual may be cured or the patient’s life lengthened. But even when that doesn’t occur, the individual will have the satisfaction of knowing that they helped save someone in the future.”
“There are research facilities in San Diego developing potentially life-saving medicines and therapies, which have been proven safe after rigorous testing. Unfortunately, because of the lengthy FDA approval process, patients with the most urgent needs aren’t able to access them,” Congressman Peters said. “We should be doing more to promote research and innovation here in the United States, and this bill is a way to incentivize researchers to stay here while getting patients remedies that they need.”
“The Patient Choice Act offers hope to patients by giving them more treatment options and control over their own health decisions,” said Congressman McCaul, Chairman of the Childhood Cancer Caucus. “I am particularly proud to support legislation which will spur the development of much needed treatments for children with cancer and other rare disease for which there are few treatment options available.”
The United States boasts an array of companies working hard to develop new treatments for terminal and life-threatening diseases. However, many times their work languishes on the shelf in labs as federal regulations and long review times impede their efforts to bring comfort to American citizens. Further, companies are leaving the United States and heading overseas to address this issue, citing FDA delays, unpredictability and an ever-expanding and costly regulatory process.
The Patient Choice Act would create a fast track provisional approval process, in which the Secretary of Health and Human Services would have 90 days to grant provisional approval status to the sponsor of the drug after the drug has been determined to be a fast track product and the sponsor has submitted the appropriate safety data and requested provisional approval. The sponsor must continue to follow the normal FDA approval process, ultimately proving the full safety and effectiveness of a drug.
The legislation would also provide patients with terminal diseases with the option to purchase investigational drugs that have been given provisional approval at the patient’s own expense.
If a drug has been approved in Europe and other FDA “listed countries” for at least four years, a company can submit an application with existing safety support data from their “listed country” approval, and the Secretary must make a decision on granting the drug provisional approval.
Finally, the FDA can withdraw approval if any data shows it is not safe. This provisional approval operates similarly to what is currently allowed in off-label use of approved drugs for new unapproved indications. Provisional approval enables this use with full disclosure and informed consent at the outset of safety in anticipation of the first indication for use. This will bring this access to patients much sooner, and does so responsibly and with common sense to provide patients the freedom to battle deadly diseases.