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he drug was still experimental, but clinical trials suggested it could be a lifesaver for patients with a lethal form of blood cancer called multiple myeloma.

And those patients were clamoring to get it. They overwhelmed drug maker Janssen Pharmaceuticals with requests for the medication.

Most companies don’t know how to handle such requests. Often, it’s the richest patients, or the best connected, or those who run the most compelling social media campaigns who end up getting the drug. Everyone else is out of luck.

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Janssen’s parent company, Johnson & Johnson, decided on a unique approach: Rather than try to deal with the barrage of requests itself, it asked a leading bioethicist to create an independent committee to determine which desperate patients could get access to the limited supplies of its experimental drug, known as daratumumab.

A year and a half later, the company and patient advocates deem the process a success — and the bioethicist, Arthur Caplan, is looking to replicate it with other drugs, starting perhaps with a psychiatric therapy.

But the novel system has also raised some thorny questions.

One ethicist who strongly supports the concept in general nonetheless asks whether it is truly wise to eliminate all personal lobbying and decide who gets access to drugs based only on anonymous medical records. What if someone like scientist Stephen Hawking is in the mix, he asked: Shouldn’t he get priority access to a lifesaving drug, for the good of humanity?

“Imagine if he wasn’t saved, what a difference in the world there might have been,” said Dr. Charles Debrovner, a cofounder and former president of the Global Bioethics Initiative, a nonprofit that studies and raises awareness about bioethics.

Then there’s the question of clinical trials. Such studies are vital to test the efficacy and safety of experimental drugs — and an essential step in getting them to market. But they generally require control groups — which means that some participants get placebos or older medications instead of the new drug.

Will patients battling a deadly disease be willing to accept the risk of getting a placebo, or will they opt instead to put their fate in the hands of a panel like Caplan’s Compassionate Use Advisory Committee — which ended up disbursing the drug to the vast majority of patients who requested it?

If too many shun clinical trials, the drug maker may find it hard to gather enough data to submit the medication for approval by the Food and Drug Administration.

“You may have helped this one individual, but put off the drug getting into the hands of hundreds of people where it might be beneficial,” said Debrovner, also a clinical professor of obstetrics at New York University, where Caplan also works.

To minimize that risk, Caplan’s committee only accepted patients who were not eligible for clinical trials.

That makes sense, said Aaron Kesselheim, an expert in bioethics and drug development at Harvard Medical School and Brigham and Women’s Hospital. “This is an important challenge in the expanded access system, and one that programs like J&J’s are going to have to consider as they hopefully move forward,” he wrote in an email.

Weighty decisions based on anonymous records

The 10-member panel Caplan heads is a virtual group, a joint project of J&J and the New York University Langone Medical Center. Patient advocates, doctors, and ethicists read medical records on their own and then a randomly selected group of three of them — chosen just a few days before — get on a conference call to discuss their recommendations.

All they know about each patient is his or her age, dependents, and medical records. Not their name, gender, race, profession, friends, income, or even country of origin.

“No more social media campaigns. No more calling up the senator who calls the CEO who then gives you the drug,” Caplan said. And no disadvantage to “somebody who’s not photogenic or isn’t articulate.” That’s how it should be, he said: He doesn’t agree that a Stephen Hawking deserves a lifeline more than anyone else.

To minimize lobbying from prospective patients, most of Caplan’s fellow panelists have not been publicly identified.

At first, picking who should get the experimental multiple myeloma drug daratumumab was tricky. Janssen didn’t have enough supply to go around. The committee had to deny the promising drug to some and approve it for others.

They decided based on how much the drug was likely to help. It takes several weeks for the body to metabolize. Some patients were simply too close to death to benefit; others still had options left to them. When the supply was tight, committee members favored younger patients, those with more dependents, and those who had previously participated in a clinical trial. They also factored in the length of time the patient had suffered with the disease, Caplan said.

Once the FDA approved daratumumab (brand name Darzalex) last November, supplies increased. Now, nearly anyone who lives in a country where it hasn’t yet been approved can get compassionate use access through the panel.

The Multiple Myeloma Research Foundation, a patient-founded advocacy group, is very happy with how the process turned out, said MMRF Vice President Anne Quinn Young.

She said the committee provided daratumumab to dozens more people than would have gotten it otherwise. The committee reviewed 76 patient requests and approved 60 of them in its first half-year. “The fact that in six months, an additional 60 patients were treated with the drug before approval is not insignificant,” Quinn Young said.

In Europe, she noted, some patients had been denied access to daratumumab even after they’d participated in a clinical trial designed to win its approval. The committee made the drug available to them.

Janssen “is doing everything they can to get patients fair access to the drug, which truly is an incredibly important therapy for myeloma,” Quinn Young said. “I can’t think of anything that they should have done differently.”

A land mine for companies

The federal government allows companies to provide “expanded access” to drugs before they are approved, if they have already been shown to be safe, the patient is aware of the risk, and the company agrees to make the medicine available. The FDA must also approve the request, which it has done 99 percent of the time since 2010, press officer Sandy Walsh said in an email.

“The FDA is proud of its track record,” she wrote.

But compassionate use has become a land mine for some companies.

In 2014, Kenneth Moch, CEO of Chimerix, received death threats and then was replaced after he initially turned down a request to provide an investigational antiviral drug to an 8-year-old Virginia boy named Joshua Hardy.

Following an initial rush of bad publicity and a #savejosh social media campaign, Moch arranged for the boy to become part of a hastily arranged clinical trial, so he could get the drug.

But two years later, after failing several late-stage clinical trials, the drug brincidofovir still has not won FDA approval. Josh died in September at age 10.

Companies have valid reasons for hesitating to give away their experimental drugs. They often have just a small stockpile on hand and need to save much of it for clinical trials. They also fear, justifiably, that their chances to get the drug approved could falter if dying patients take it outside of clinical trials and suffer bad side effects — or simply don’t improve.

One recent survey found that only 19 percent of 100 drug companies publicly posted their compassionate use policies.

Johnson & Johnson sought a more transparent, objective, and equitable plan for disbursing daratumumab, said Dr. Joanne Waldstreicher, the company’s chief medical officer. It also wanted to respond to requests within days: “We wanted to be sure we weren’t just leaving patients and their families hanging.”

To minimize conflicts of interest, Caplan has declined payment, though Johnson & Johnson does pay some other members and covers infrastructure costs such as setting up a website to take requests. The company doesn’t have to take the panel’s recommendations, but so far, it has.

Caplan hopes the ethics review model that started with a single cancer drug will soon spread to other medications and diseases; he’s particularly interested in urgent cases when a child’s life hangs in the balance.

He’s already consulted informally with other drug companies and is hoping to persuade the World Health Organization to set up a similar system.

A panel like CompAC, he said, would have been useful during the 2014 Ebola outbreak, for instance, when a very limited supply of potentially effective treatments were available, and much of it went to sick foreign aid workers rather than local Africans.

Debrovner agrees. “I really look forward to seeing this adopted universally,” he said. “It’s hard to believe it’s taken this long for someone to embark on this, because it just seems so obvious.”

Correction: A previous version of this story incorrectly stated J&J’s compensation arrangement with members of the panel.

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  • A better solution would be to change the way the FDA approves drugs. Instead of the Go/No-Go method they currently use why not implement the rolling approval process that has been suggested for well over 2 decades?
    With rolling approvals the PharmaCos are allowed to put the drug in the field under very tight yet available restrictions and the doctors who are prescribing the drugs must consistently provide data to the FDA and the drug company and any deviation from either expected outcome or compliance is a trigger to stop allowing new admissions. By using the rolling process the better the results the more are allowed to join the testing process. In addition instead of a deadline of approval which could shift the balance of stock and profits the drug simply slides into the society and becomes integrated into the formulary as it gains success and acceptance. Instead of selected high profile (read EXPENSIVE) university only type facilities each licensed doctor who has a patient in need can participate with the rolling process if he/she the patient and the PharmaCo agrees. In terms of costs it is FAR less costly to give some drugs to patients then it is to pay a university team a few million dollars to supervise “a clinical trial”
    Dr D

  • The so – called ” Compassionate ” Use system for pre – approval access to drugs in this country is broken . The situation in Europe ( Bigger Gov.t Bureaucracy) and around the world is even worse. My wife is a Stage IV breast cancer survivor. Which is normally a death sentence , but new drugs are changing the situation . However the System is not keeping up . My wife is alive today because of a new 21st century drug , Kadcyla. We could not get compassionate use in 2010. We could not wait for the Slow FDA and the up-tight , Profit Driven Pharma industry in 2010. The FDA shockingly denied accelerated approval for the drug and it was not available in Boston . So we went to Wash. , DC 16 times at our own expense to get the drug . Her cancer disappeared by 2011. We stopped the drug in 2014. She is cancer – free.
    We applaud both Janssen and the NYU team for starting to change the System . The FDA is trying to get quicker – we need more pharma co.’s to follow Janssens’ lead . This drug is just one of many , every yr. , that still take 7 – 10 yrs to get through the System . Even then they often have conditional approval from the FDA limiting access for many yrs. after . We pt. activists do have some differences with Dr. Caplan and his NYU team – he gives the FDA a free pass, and does not factor in the FDA’s bureaucratic slowness ; their Regulatory Power contributing to co.’s reluctance to grant compassionate use. His words about the ” the rich ( not factually proven ) and ” connected ” , pt.’s who are ” savvy with social media ” tend to cast blame on the pts., the victims of this broken system . We do appreciate his initiative. But this is just a drop in the bucket . Much more reform needs to come .

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