Right to try

In May, Colorado became the first state to pass a “Right to Try” bill, giving terminally ill patients access to experimental drugs without needing approval from the Food and Drug Administration (FDA). Missouri and Louisiana have also passed similar bills, and Arizona has scheduled a referendum on “Right to Try” in November. While these bills easily passed with bipartisan support, it is important to note that the FDA already grants “compassionate use” exemptions for use of unapproved drugs. Why then, were “Right to Try” bills needed, and what impact will they have?

What is the goal of “Right to Try”?

One success story highlighting the promise of “Right to Try” is that of Josh Hardy, age 7, who was diagnosed with myelodysplastic syndrome – a bone marrow cancer [1]. During a bone marrow transplant, Josh suffered an adenovirus infection, and existing therapies were ineffective and caused kidney toxicity. Josh’s doctor recommended an experimental antiviral called brincidofovir, from Chimerix, which had been used in clinical trials to treat adenovirus infections. Josh’s parents lobbied for compassionate use of the drug from the company and after intense social media pressure and publicity, Chimerix agreed to open a small clinical trial that included Josh. Two weeks after Josh received the drug, the adenovirus was cleared and the boy is currently in recovery .
The push for “Right to Try”

However, the patient originally behind “Right to Try” was less fortunate. After his daughter, Abigail, lost her fight against cancer, Frank Burroughs created the Abigail Alliance for Better Access to Experimental Drugs and has been one of the driving forces behind “Right to Try”. While the FDA approves virtually all applications for compassionate use, Burroughs claims that the process is too complicated and lengthy [2]. Therefore, the new state laws only require patients to have a prescription or recommendation from a doctor and for a drug company to agree to provide their drug. Rep. Joann Ginal (D) co-sponsored the bill in Colorado and said, “For people who are facing death and have one last hope, they should have a choice to try every possible drug.” [2]
The other side of the coin

It is easy to frame the FDA and pharmaceutical companies as callous organizations in this debate, especially when patient’s lives are on the line. After all, the argument goes, if the drug works, then everybody wins; and if it doesn’t work, then at least the patient (and their family) has the comfort that everything was tried. As Burrough says, “The risk-benefit is much different than someone who’s waiting for a new allergy medication or a new toe fungus cream.” [2] However, this viewpoint overlooks some very real concerns about these new laws. “Right to Try” only extends to drugs that have passed Phase I clinical trials, but it is far from assured that these drugs are safe for use or will actually work. In fact, only 16% of drugs that pass Phase I are eventually approved [3]. While patients are still willing to risk these odds, these drugs could also exacerbate their condition and leave them worse off than before. In this scenario, the prescribing doctor, and/or the drug company could potentially be held at fault.
Furthermore, drugs are often designed for very specific disease indications, which is why companies are often hesitant to provide them for cases that do not fall under these requirements. Indeed, Chimerix initially refused to provide bricidofovir because Josh’s infection was more advanced than any of their previous patients. Also, if a patient died while on the experimental drug, this could delay or prevent FDA approval since the company would have to prove that their drug did not contribute to their death. This concern was echoed by Sascha Haverfield, vice president of scientific and regulatory affairs for the Pharmaceutical Research and Manufacturers of America. “We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the Food and Drug Administration and clinical trial process, which is not in the best interest of patients and public health,” Haverfield said [4]. Arthur Caplan, a bioethicist at New York University, points out that small pharmaceutical companies also may not have the resources (either money or drug supply) to allow for compassionate use trials. Caplan says, “We’re treating it as if it’s a standoff between a desperate family and a little company. It isn’t.” [1]
Impact of “Right to Try”

While the process of connecting patients with drug companies should be streamlined under these new regulations, it is critical to note that these laws do not compel companies to offer their drug. A payment scheme for treatment expenses is also not laid out under these laws, meaning that patients, drug companies, and health insurance will have to address these steep costs on an individual basis. Last and most important, these laws may violate existing federal laws. As Jann Bellamy notes at Science-Based Medicine, “Once the FDA-drug approval process is underway, the manufacturer is by definition under FDA jurisdiction.” [5] Therefore, the FDA has constitutional authority over all such drugs, including their use. While the new “Right to Try” laws have not yet been legally challenged, the FDA released a statement that these laws affect their “congressionally-mandated authority and agency mission to protect the public from therapies that are not safe and effective.” [2]
What are the alternatives?

If “Right to Try” laws end up being overturned, there are other viable options. One is to facilitate compassionate use requests at the FDA, either through increased staffing or reduced paperwork. Another option is to make the clinical trial and approval process faster. Toward this goal, the FDA has developed “Breakthrough therapy”  designations for novel drugs that will provide substantial improvement over existing therapies. Imbruvica (Pharmacyclics, Johnson & Johnson) was one of the first drugs to receive this designation and went from clinical trials to approval in less than 5 years, compared to the industry average of 10 years. Disease foundations such as the Multiple Myeloma Research Foundation are also playing a more proactive role in drug development by working with companies to develop clinical trials for rare diseases and to increase patient enrollment in these trials.
Curing diseases is one of the greatest challenges faced by our society, and patients, drug companies, and the government must all work together in this fight. Whether it is through “Right to Try” laws, changes in the drug development process, or another option, all of these parties have the same goal: the approval of lifesaving drugs to cure patients.



[1] Sydney Lupkin (2014) ‘Dying Boy to Get Unapproved Drug After Family’s Plea’ ABC News 12th March, Retrieved [4th July 2014]

[2] Brady Dennis and Ariana Eunjung Cha (2014) ‘‘Right to Try’ laws spur debate over dying patients’ access to experimental drugs’ Washington Post 16th May, Retrieved [4th July 2014]

[3] Hay M et al., Clinical development success rates for investigational drugs. Nature Biotech, 2014, 32: p. 40-51

[4] Michael Ollove (2014) ‘Right-to-try for the terminally ill’ USA Today 19th June, Retrieved [4th July 2014]

[5] Jann Bellamy (2014) ‘The illusions of “right-to-try” laws’ Science-based Medicine 6th March, Retrieved [4th July 2014]