American Society of Clinical Oncologists opposes state "right-to-try" laws

American Society of Clinical Oncologists opposes state "right-to-try" laws

The American Society of Clinical Oncologists (ASCO) released a statement this past week opposing state "right-to-try" (RTT) laws. While the ASCO remains committed to expanded access to investigational drugs and to improving current processes for obtaining those drugs, the organization thinks state RTT laws will be both ineffective in improving access and may cause unintended harms.

RTT laws, which have passed in 33 states as of last month, purport to allow terminal patients access to drugs that have passed Phase I of the FDA's drug approval process directly from the drug's manufacturer. Terms vary, but generally the patient must have considered, although not necessarily tried FDA-approved drugs and must have a physician's prescription, although there is no requirement that the physician be a specialist with education and training in the patient's condition. (E.g., an oncologist in the case of a cancer patient.) Some laws protect physicians from discipline by a state medical board for using the RTT process and absolve insurance companies from any responsibility for covering the drug or its side effects. No state law mandates that the manufacturer provide an investigational drug.

SFSBM Board chair David Gorksi, MD, has covered RTT laws extensively over on SBM. I've written about RTT laws too, as well as federal bills which would remove one of the main barriers to implementation of RTT laws: the fact that providing access to investigational drugs outside the FDA's own expanded access program would be a violation of federal law. (You can find that post, as well as a collection of SBM posts on the subject here.)

The ACSO recognizes criticism of the FDA's current access program, but points out that the process has been streamlined and that about 99.5% of applications for investigational drugs have been approved. It also notes that the time lag between desire for access and decision isn't necessarily the FDA's fault. Applicants must determine the manufacturer's willingness to provide the drug, but

"Providers and patients report difficulty in locating information about drug manufacturer contacts for such requests, significant delays in response and even denial of the request altogether."

As the ASCO also points out, the recently passed 21st Century Cures Act will help address this issue by requiring drug manufacturers to make their expanded access policies and contact information available to the public, along with describing the process for making requests, the criteria they will use in determining whether to grant it and the length of time estimated to respond. These measures are supported by the ASCO.

While the ASCO finds RTT laws "well-intentioned," the organization does not think they are

"an effective mechanism for improving access to investigational drugs for terminally ill patients and may cause unintended harms."

This is because RTT laws

  • Don't require drug companies to provide investigational products, therefore they "do not remove a frequent barrier to access."
  • Don't require insurance companies to pay for routine costs associated with treatment delivery, unlike coverage requirements for patients who participate in FDA clinical trials.
  • Lack independent review of the potential safety and efficacy of investigational drugs, unlike the FDA's expanded access program.
  • Jeopardize insurance coverage for costs of care, particularly in case of complications.
  • Circumvent the government's responsibility to monitor and protect patients seeking access to investigational drugs.
  • Don't require transparency, partially due to lack of reporting requirements, on how patients respond to these drugs.
  • Don't do anything to mitigate the delays caused by drug company response times or lack of information about the process.

In summary, the ASCO, believes that

"existing and proposed RTT laws do not adequately protect patients, do little to facilitate patient access to such therapies, and potentially interfere with recent reforms that are already streamlining patients' access to investigational agents."

The ASCO is correct. In fact, in the nearly two years since the first RTT law was passed, there isn't a single verified instance of a patient being helped by these laws. Unfortunately, if patients are being harmed, lack of reporting requirements means we may never hear about it. 

Points of Interest 04/10/2017
Points of Interest 04/08/2017