In May of 2017 the Board of Directors of the Association of Clinical Research Organizations, which represents the world’s leading clinical research organizations, unanimously approved a statement explaining ACRO’s deep concerns about proposed federal Right-To-Try legislation. While promising hope to patients and parents desperate for treatment options, instead, Right-To-Try laws passed by a number of states have at least three critical failings:
- They do not adequately inform or protect the safety and basic rights of patients.
- Compromising the clinical trial process that is the gold standard for determining whether an experimental drug or treatment actually works.
- They undermine the FDA’s authority to protect the public health and to promote new medical products that are both safe and effective.
We noted that the FDA’s Expanded Access (also known as “Compassionate Use”) program does what Right-To-Try bills do not – offer access to experimental therapies with scientific and ethical oversight – and that the FDA approves over 99 percent of compassionate use requests. ACRO and its Board of Directors continue to believe that any program to make unproven therapies available to patients outside of the clinical trial framework must include proper patient protections and avoid compromising FDA review of safety and effectiveness. The Senate’s version of Right-To-Try fails to meet that important threshold.
When S. 204 passed the Senate without a recorded vote in August of 2017, we were disappointed that that body advanced a piece of legislation that, from the definitions of eligible patients and eligible investigational drugs to the exclusion of oversight by the FDA, was deeply flawed.
ACRO was pleased that in October 2017 the House Energy and Commerce Committee, under Chairman Walden and Health subcommittee Chairman Burgess, held a hearing on Right-To-Try, taking testimony from supporters of Right-To-Try, as well as from patient groups, an ethicist and FDA Commissioner, Dr. Scott Gottlieb.
The House thoughtfully considered a range of views on Right-To-Try, taking feedback from ACRO and others regarding the dangers of any Right-To-Try legislation that did not explicitly include key safety provisions, including a requirement for an Informed Consent by the patient, notification to the FDA about such use of experimental and unapproved drugs, and – crucially – the reporting of adverse events to the drug manufacturer and the FDA by a physician who prescribes such treatments. If deaths and other serious adverse events that occur because of the use of an experimental drug are not captured and reported, then not only is the individual who received the unapproved drug injured, but so is every subsequent person who might be given the Right-To-Try the same drug. This is not merely bad science, it is incredibly bad ethics.
Even as we continued to believe that the push for Right-To-Try legislation was unnecessary and misleading, ACRO asserted that these basic ethical and scientific protections must be included in any bill because in public health, in science and in legislation the devil is indeed in the details. We acknowledged that the bill (H.R. 5247) that passed the House on its second attempt in March of this year made efforts to address some of the concerns that had been raised, providing for better protection of desperate patients and raising the scientific and ethical requirements for the (we hope rare) physician who might otherwise use Right-To-Try in less than responsible ways.
ACRO remains opposed to federal Right-To-Try legislation, but given the thoughtful work by the House only a few months ago, we are especially disturbed to see that that body is now scheduled to take up the fatally flawed Senate bill next week. In our opinion, Chairman Walden and the leadership of the House should remain committed to making absolutely essential improvements to the Senate bill, as they did with H.R. 5247. Not only for the House, but for Vice President Pence and President Trump, the goal should not be to get a bill to sign, but to produce legislation that aims to help and protect patients and that supports the critical importance of the Food and Drug Administration’s oversight of the public health.