Not only don’t these laws accomplish anything as a legal matter, as a policy matter, to the extent that they confuse patients or manufacturers about the process, they could actually be quite harmful.
—Patricia J. Zettler, JD
One thing that the new laws in development show is the level of engagement of lawmakers in representing the public on an issue of importance to patients. It is good for the public to see dialogue that involves all of the stakeholders, including lawmakers, patients, industry, and academic groups.
—Amrit Ray, MD
Since 2014, “Right to Try” legislation has been sweeping the nation. Created to enable terminally ill patients to gain access to experimental drugs, biologics, and devices by sidestepping the approval process of the U.S. Food and Drug Administration (FDA), at press time, 22 states have enacted Right to Try laws, and 19 states have legislation pending.
Although provisions in the law vary from state to state, they generally permit terminally ill patients access to an investigational treatment if a physician recommends use of the treatment; the patient gives informed consent; and the treatment has completed a phase I clinical trial. In some states, the laws exempt drug manufacturers and physicians from liability against claims arising from adverse side effects of the treatment. However, Right to Try legislation does not mandate that pharmaceutical companies provide their drugs or devices to patients, nor are health insurance companies required to pay for the treatment.
Critics say these laws have created a false expectation that desperate patients will be able to gain quick access to potentially lifesaving treatments because they can bypass the FDA’s expanded-access application process and go directly to the manufacturer for the drug. However, according to Patricia J. Zettler, JD, Associate Professor at Georgia State University College of Law and former Associate Chief Counsel for Drugs at the FDA, the U.S. Constitution guarantees that federal law supersedes state law and that state laws cannot permit manufacturers to provide patients with access to unapproved drugs under the Food, Drug, and Cosmetic Act,1 which prohibits the sale or distribution of any drug or device into interstate commerce until it is proven to be safe and effective.
“Not only don’t these laws accomplish anything as a legal matter, as a policy matter to the extent that they confuse patients or manufacturers about the process, they could actually be quite harmful,” said Professor Zettler.
Active Engagement and Dialogue
Although Amrit Ray, MD, Chief Medical Officer for Janssen Pharmaceutical Companies of Johnson & Johnson, states that no steps should be taken that would create false hope for patients, overall, he said, it is good to see greater engagement and dialogue on the issue of compassionate drug use (See here for more on the Johnson & Johnson Independent Bioethics Panel).
“One thing that these new laws and other legislation in development show is the level of engagement of lawmakers in representing the public on an issue of importance to patients,” said Dr. Ray. “I think that active engagement has more potential to be productive than leaving things to chance. It is good for the public to see dialogue that involves all of the stakeholders, including lawmakers, patients, industry, and academic groups.”
Professor Zettler said that although Right to Try legislation may not accomplish its goals, it is understandable why the statute is being enacted across the country. “People want to help terminally ill patients, and I think that for a lot of politicians, there are probably good motives behind wanting to pass these laws,” said Professor Zettler. “But what terminally ill patients really need are effective treatments for their particular condition, and these laws don’t do anything to create those treatments. So, to the extent that they are taking away resources from efforts to achieve the result that patients really need and add confusion about the FDA’s approval process, I don’t see them as a positive thing.”
To date, there are no reports that patients have actually acquired investigational drugs through Right to Try laws.
Expediting Medical Innovation Nationally
On May 21, 2015, the U.S. House of Representatives’ Committee on Energy and Commerce approved the 21st Century Cures Act.2 It proposes to accelerate the development and approval of new drugs and devices on a national level and adds about $1.5 billion per year for 3 years to the National Institutes of Health’s (NIH) current $30.3 billion budget. The law also provides an additional $2 billion per year for 5 years to create the NIH Innovation Fund, which would support precision medicine initiatives and the work of young scientists’, as well as $550 million over 5 years in additional funding for the FDA to implement drug-approval changes called for in the law.
Although a number of nonprofit organizations representing patients, family caregivers, health-care providers, and researchers support the bill, in a recent article in The New England Journal of Medicine,3 Jerry Avorn, MD, Division Chief, Pharmacoepidemiology and Pharmacoeconomics and Professor of Medicine at Harvard Medical School, and Aaron S. Kesselheim, MD, JD, MPH, Associate Professor of Medicine at Harvard Medical School and Associate Physician at Brigham and Women’s Hospital, voiced concern that the Cures Act could actually reduce the effectiveness of the current FDA approval process.
“Over the past 80 years, this country’s regulatory approach has embraced steadily improving criteria for accurately assessing therapeutic efficacy and risk. Patients and physicians would not benefit from legislation that instead of catapulting us into the future, could actually bring back some of the problems we thought we had left behind in the 20th century,” wrote the authors.
A vote by the full House of Representatives on the 21st Century Cures Act is expected this summer. If it passes, it would then go to the Senate for a vote and could be before President Obama to be signed into law in early 2016. ■
Disclosure: Dr. Ray is an employee of Janssen Research & Development, LLC.
1. U.S. Food and Drug Administration: Key legal concepts: Interstate commerce, adulterated, and misbranded. Available at http://www.fda.gov/Cosmetics/GuidanceRegulation/LawsRegulations/ucm074248.htm. Accessed July 28, 2015.
2. Johnson JA, Thaul S, Bagalman E: H.R. 6: The 21st Century Cures Act. Available at https://www.fas.org/sgp/crs/misc/R44071.pdf. Accessed July 28, 2015.
3. Avorn J, Kesselheim AS: The 21st Century Cures Act: Will it take us back in time? N Engl J Med 372:2473-2475, 2015.