The ethical minefield surrounding the FDA’s compassionate use application process was front page news in 2014. The debate over who gets access to new and experimental treatments when all other options have run out for people with terminal illnesses or debilitating diseases spawned a social media campaign that led to a media firestorm. This led state and federal lawmakers to propose and pass compassionate use legislation, which spurred the development of the New York University Langone Medical Center Working Group on Compassionate Use and Expanded Access to work with lawmakers, regulators and companies on the issue.
The social media campaign launched by the family of 8 year-old Josh Hardy earlier this year led to the demonizing of Chimerix, the biotechnology company that turned down a compassionate use application by St Jude physicians on behalf of Hardy. Chimerix’s former CEO Kenneth Moch received death threats, which prompted the FBI to provide armed guards for Moch. The application sought to use the biotechnology’s drug, brincidofovir, to treat Hardy for adenovirus following a bone marrow transplant, although its lead indication was for cytomegalovirus.
A Biocentury article noted that prior to beginning pivotal trials, Chimerix had provided brincidofovir to compassionate use applicants. Following dramatic results for some patients, the demand for compassionate access to the compound far outstripped the company’s ability to administer an access program.
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Ultimately, the family won. Chimerix initiated a clinical trial of the drug for adenovirus in immunocompromised patients, Hardy got the drug and the treatment worked. Some might point to this case as a fine example of the power of social media. Others, like the Working Group, see it as tragically unfair for anyone not fortunate enough to have the advantages that Hardy’s family did. The BioCentury article reported on the consequences of the campaign and others like it:
“As the Hardy case illustrates, patients or their relatives can generate hundreds of thousands of supporters virtually overnight, along with a wave of attention on television. In the heat of a media feeding frenzy, it is impossible for a CEO to communicate the complexities of drug development and why the integrity of the regulatory approval pathway must be protected to get a drug to as many patients as possible.”
Dr. Arthur Caplan, a bioethicist in the population health department at NYU Langone Medical Center and a professor at the medical school, is part of the 13-member working group.He acknowledged the impact of social media and the ethical issues it raises.
“Social media is a wildcard in compassionate use,” Caplan said. It can be a huge factor but it’s clearly unfair. “When you are a photogenic kid it works a lot better than if you were say a 60 year-old alcoholic.” It can matter a great deal who you are, who you know, such as someone who is savvy with social media or folks with deep pockets. Another problem with social media, Caplan noted, is that it wears out. When you get to the 20th person appealing for a drug, the media firestorm has burned out. Hardy may have gotten plenty of attention, but no one after him did.
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He pointed out a couple of things that tend to get overlooked in the compassionate use debate. For one thing, there is expanded access — a term used to refer to requests from groups of patients for access to unapproved drugs and medical devices. Another is that many of the requests aren’t by patients with terminal illness. Think treatments to prevent blindness, restore vision or to regain the ability to walk.
States have been rushing out feel good Compassionate Use laws this year with Colorado claiming bragging rights as the first state to do it. As Congressmen propose their own legislation, a working group assembled at New York University Langone Medical Center sees room for improving the current process for dealing with these requests.
If state lawmakers view their Right to Try laws as evening out the playing field for access to experimental drugs, Caplan pointed out they are nothing more than feel good laws that are ineffective.
“We are very critical of these laws. They create no obligation of companies to offer drugs. They can turn down requests. The laws are not set up to track what happens to these applications. You can’t create a right without creating an obligation. These are right to beg laws. We already have a right to beg.”
He points out that the laws make no mention of what should be charged for the drugs. No insurers wants to pick up the tab for an experimental drug and neither do the states. The laws don’t help applicants with funding to travel to the company or hospital to obtain the drug. They also only focus on people with terminal illnesses.
Caplan believes they will probably be defeated in a court challenge. “You can’t just throw the FDA out of the way.” The working group sees increasing access to unapproved therapies as potentially detrimental to longstanding and effective research. It also regards these efforts as undermining the clinical trial systems tat are designed to prove the safety and effectiveness of drugs, so they can be greenlighted by the FDA.
On its website the working group clarifies a couple of myths about compassionate use and expanded use, based on its preliminary research on the issue. Among them:
- Biotechnology companies have no legal or regulatory obligation to provide access to unapproved treatments on the grounds of compassionate use. Some companies allow access under the guidance of well thought out policies; some companies decline to allow access; some companies grant access but have no set guidelines; and some companies change their practices midstream as a result of public pressure. This lack of uniform policy is confusing to those seeking unapproved treatments.
- Contrary to widespread perception, the U.S. Food and Drug Administration is not an obstacle to those seeking compassionate use. In fact, the FDA almost always defers to the company that is developing the unapproved treatment to decide whether to grant compassionate use access.
Members of the working group formed at NYU Langone Medical Center include people with ethical, medical, biotech, pharmaceutical and legal backgrounds. Moch is among the members. It has met with patient groups and next year plans to meet with lawmakers and the FDA. Next year, the working group hopes to have a conference looking at these ethical issues in more detail from a variety of perspectives.
Among the two biggest problems with the compassionate use application process the working group wants to rectify are lack of awareness and inefficiency, according to Caplan. A lot of physicians don’t even know it’s an option. Doctors need to be better educated about the application process. It’s not likely that patients will know about it either. But patients are probably relying on the expertise of physicians to tell them about options. Another challenge is the FDA paperwork for applications is too time-consuming. It can take four to five hours to complete, Caplan said. The paperwork could be reduced. It’s also unclear which phase of drug or device development the FDA favors for granting compassionate use. So far, state laws passed this year require that treatments have advanced past Phase 1.
The group will be keeping a close eye on how states with compassionate use laws on the books deal with applications. The debate on access to experimental drugs is set to get more heated as legislation moves through Congress and other states adopt Right to Try laws.
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