Stacey Lee Paper Looks at Access to Trial Drugs


STACEY Lee Paper Proposes Guidelines on Access to Trial Drugs in Terminal Cases

If you’re diagnosed with a terminal illness, your survival could depend on gaining “expanded access” to a drug that’s in clinical trials and far from approval for public use.

The access is difficult but not impossible to get. A frustration for many patients in this situation, however, is that when they reach out to pharmaceutical companies, they often encounter unclear, incomplete, and seemingly capricious guidelines for acquiring the treatment that could save their lives.

A recent study by a Johns Hopkins University expert on the legal aspects of health care proposes a solution. Writing in the Journal of Business Ethics, Assistant Professor Stacey Lee (below right) of JHU’s Carey Business School suggests a framework that would give patients the guidance they need – primarily through clear, comprehensive information on the drug manufacturers’ websites that spells out the procedures for expanded access.

The path to expanded access starts when a physician sends the request on a patient’s behalf to the maker of the desired drug. If the company complies, as some will, then the physician asks the federal Food and Drug Administration to approve the use of the experimental treatment. More than 99 percent of such requests win the FDA’s favor.

But drug companies are under no obligation to comply with a request or even to state a reason for denying it. And when they say no to expanded access, patients become frustrated not just by the denial but also by the lack of communication about how and why the decision was made.

In some cases, patients and their advocates have fought back by demanding expanded drug access via online petitions, social media campaigns, and mainstream media coverage. In a 2014 case, a family obtained expanded access for their seven-year-old son after their story first went out on social media and then was picked up by CNN and other media outlets.

Not all such campaigns result in expanded access for the patients in question. Yet, as noted by Lee and co-author Alexandra Murata, a master of public health candidate at the Johns Hopkins Bloomberg School of Public Health, the publicity has helped to inspire more than 30 states to pass “Right to Try” laws that aim to override FDA limitations on expanded access and thus make it easier for patients to obtain experimental drugs.

The state laws, however, cannot compel drug companies or insurers to provide or pay for treatment, say the authors, who add that no drug company has granted expanded access in response to a state Right to Try law. Moreover, a court test would likely give federal regulations precedence over the state laws because of the Constitution’s Supremacy Clause.

Congress addressed the issue in late 2016 with the passage of the 21st Century Cures Act, which requires manufacturers of life-saving experimental drugs to make public their decision-making policies for expanded access, along with company contact information and links to clinical trial records. To date, only one of the top 10 pharmaceutical companies analyzed by Lee and Murata has taken the steps called for in the Cures Act.

Without “proper buy-in” from the drug makers, the authors argue, patients and their physicians will continue struggling to learn about the companies’ expanded access policies.

The study concludes with a suggested framework that the authors say could prove beneficial across the board, supplying patients with the information they seek while easing the criticism directed at the drug industry. Among the recommendations:

  • Drug manufacturers’ expanded access policies should be easy to find and use on the companies’ websites, starting with a drop-down menu on their home pages.
  • The websites should maintain easily understood databases of all drugs eligible for expanded access.
  • The sites should include lists of contacts at government and private organizations that can provide information and other resources.
  • Videos explaining the policies and procedures for expanded access should be posted on the websites.
  • The drug makers should create standardized informed-consent disclosures, including information about the experimental drug’s current status in clinical trials and the possibility that the treatment could have no benefit or prove harmful.
  • The companies should not condition expanded access on a patient’s waiver of his or her legal rights. (The FDA forbids such waivers, though some state laws have language allowing them.)
  • Companies should provide a written explanation when denying a request. Also, after denials, patients should have the right to appeal to a third party, such as the FDA.
  • Expanded access should not be allowed to compromise the integrity of a drug’s experimental phase – for example, by significantly reducing the supply of a drug undergoing clinical trials. For the sake of transparency and a broad understanding of the balance that must be struck, companies should make it known that a drug trial capable of saving many lives in the long run will not be jeopardized for the sake of possibly saving one individual or even a few in the short term.

The paper by Lee and Murata, titled “The Expanded Access Cure: A Twenty-First Century Framework for Companies,” appeared in May 2017 on the website of the Journal of Business Ethics and is forthcoming in print in that publication. 

Posted on July 31, 2017 In MS in Health Care Management, MBA/Masters of Public Health, Faculty Story, News Item, Press Release, Research Story, Alumni, Current Students, Faculty, International Students, New Students, Partners, Prospective Students, Staff