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ACRO: Right to Try Act compromises clinical trial process

Melissa Fassbender

By Melissa Fassbender

25-May-2017
Last updated on 30-May-2017 at 18:34 GMT2017-05-30T18:34:55Z

The FDA's Expanded Access program provide patients with access to investigational drugs outside of a clinical trial. (Image: iStock/Berezko)
The FDA's Expanded Access program provide patients with access to investigational drugs outside of a clinical trial. (Image: iStock/Berezko)

Enhanced clinical trial design legislation proposes an alternative to the Right to Try Act – both of which address the FDA's current compassionate use program.

The Right to Try Act of 2017 in the House and Senate aims to authorize “the use of unapproved medical products by patients diagnosed with a terminal illness in accordance with State law, and for other purposes,” according to the text.

In response, the Association of Clinical Research Organizations (ACRO) recently published a statement in which it expressed concerns over the act as it gains traction in various states and the US Congress.

According to the organization, the legislation is “deeply flawed” and offers insufficient patient protections, compromises the clinical trial process, and undercuts the FDA’s authority.

'Deeply flawed'

As it stands, a patient must meet certain conditions in order to participate in Expanded Access , or Compassionate Use.

For example, under the Federal Food, Drug, and Cosmetic Act (FD&C Act ) a patient’s physician must determine that there is “no comparable or satisfactory therapy available to diagnose, monitor, or treat the patient’s disease or condition,” according to the US Food and Drug Administration (FDA).

However, as ACRO explained, current Right to Try legislation has no clear requirements around assessing if a patient is eligible for a clinical trial – despite some language around “exhausting available treatment options.”

Without this clarity, “a patient could simply go the Right to Try route even if they would be eligible for a clinical trial,” an ACRO spokesperson told Outsourcing-Pharma.com. “This would ultimately harm trial recruitment and retention rates, which are already a problem.”

Additionally, under Right to Try the FDA would not have access to patient data, including data on adverse events. “This absolutely undermines the FDA’s authority to assess safety,” the spokesperson added.

The FDA’s Expanded Access program also requires the physician or sponsor to provide a clinical protocol in order to determine if the therapy will be made available outside of a clinical trial. This requirement is not outlined in the Right-to-Try legislation, according to ACRO.

Protecting patients

In order to improve the various shortcomings of the Right to Try Act, ACRO proposes several amendments, including a requirement that patient data on adverse events be submitted to the FDA and sponsor company.

Even if the data does not interfere with the market approval process, the agency should have access to all patient data for an investigational therapy,” the spokesperson explained.

Additionally, ACRO said a physician should assess probable risk before a patient takes an investigational therapy.

The spokesperson explained: “It would also help to preserve patient safety if the FDA, like with Compassionate Use, was able to determine whether there is sufficient evidence of safety and effectiveness to warrant circumstantial use.

Enhancing clinical trial design

The Enhanced Clinical Trial Design Act of 2017 (S.1048 ), which was added as an amendment to the Prescription Drug User Fee Act (PDUFA ), was designed to strengthen the FDA’s Expanded Access program.

The provision addresses many of the abovementioned concerns surrounding the Right to Try legislation, and aims to “expand patient access to experimental treatments in clinical trials, and for other purposes,” according to the text.

If enacted, the bill calls on various stakeholders, including patients, health care providers, drug sponsors, and bioethicists, to discuss clinical trial inclusion and exclusion criteria and inform a new guidance.

The guidance documents “shall address methodological approaches that a manufacturer or sponsor of an investigation of a new drug may take” to “broaden eligibility criteria for clinical trials” and “develop eligibility criteria for, and increase trial recruitment to, clinical trials,” according to the proposed bill.

The bill was introduced in the Senate earlier this month.

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