Johnson & Johnson is forming a first-of-its-kind panel to review requests by desperately ill patients for experimental drugs. The effort comes as a growing number of states adopt "right-to-try" laws intended to improve access to last-ditch treatments.
The company announced Thursday that it would collaborate with the NYU School of Medicine to form an advisory committee composed of 10 medical experts, bioethicists and patient advocates to make recommendations to J&J on patient requests for access to drugs that haven't been approved by the Food and Drug Administration.
The FDA allows patients to request access to experimental drugs based on compassionate use for patients who are terminally ill and have no other treatment options. While the vast majority of requests are approved, the decision to make the medicine available remains with the pharmaceutical company.
Drugmakers have been reluctant to allow access to unapproved treatments. In some cases that's because the requested drug is in limited supply. The companies may also be concerned about attracting negative publicity if a medication doesn't work or harms the patient.
Under J&J's pilot program, patients who don't qualify for an investigational drug through a clinical trial or under the company's expanded-access program will have their request sent to the advisory committee for review. The company will make the final decision on whether to make the drug available.
“We want to establish a model that will create a structured policy of allocation based on equality, need and efficacy to ensure that the utility of our scarce resources are maximized,” said Art Caplan, director of the division of Medical Ethics at the NYU School of Medicine. “If successful, CompAC will serve as a model for others in industry and in government to follow.”
The debate over whether dying patients with no other options should be allowed access to experimental drugs has received much attention over the past year. Sixteen states have adopted right-to-try laws, which allow physicians to prescribe unapproved drugs to terminally ill patients without seeking FDA compassionate-use approval. But even then, the manufacturers are not obligated to provide the drugs, and the industry has been critical of the laws.
“Compassionate-use decisions are incredibly challenging, and we readily acknowledge that the current allocation system remains a work in progress," Caplan, who serves as a chair of the new committee, said in a news release.
Caplan has been among the critics of the right-to-try laws who argue that the laws offer patients no practical support to make it easier or faster for them to obtain an investigational drug.
In addition to offering no mechanism to actually obtain the drugs from pharmaceutical companies, the laws don't compel insurers to cover the cost of the treatment or any related healthcare services.
