Editor’s note: This story represents the opinion of a leading bioethicist with regard to the biopharma industry. Views expressed are his own and do not necessarily reflect the views of this publication. PharmaVoice will publish a follow-up article with insight from industry insiders who discuss how they approach ethics in the biopharma realm.
The pharma industry, for all the good it provides in the name of public health and scientific progress, is still an ethical minefield.
When a company like Pfizer develops the first vaccine to address a worldwide health crisis, for example, the conversation quickly turns to massive profits or suspected ulterior motives, regardless of intent and outcome. Reputation and public perception are fleeting wins.
But frank conversations about ethics can better position companies to serve their patients even if the conversations are a little uncomfortable, said Arthur Caplan, professor of bioethics and founding head of the Division of Medical Ethics at NYU’s Grossman School of Medicine.
“I’m not sure we’ve thought about what the term ‘real-world evidence’ really means."
Arthur Caplan
Professor of bioethics, founding head of the Division of Medical Ethics at NYU’s Grossman School of Medicine
Caplan, who has consulted with biopharma companies on the subject of ethics, believes that these discussions shouldn’t exist merely in the academic echo chamber but out in the open between researchers, industry insiders, government officials, patients, media and other stakeholders in the healthcare world.
Here are some of what Caplan — one of the leading voices in bioethics who speaks candidly and sometimes fervidly about such issues — sees as the most pressing concerns for the industry in terms of health, reputation and overall principle.
Ties to patient groups
“The biopharmaceutical industry has developed very close relationships with patient advocacy groups, but they’re not very transparent,” Caplan said.
Patient groups do a great job communicating to the industry that a treatment is needed for even the rarest of diseases, and companies have responded in kind, he said. But that’s not the end of the story.
“Biopharmaceutical companies are starting to use patient-driven demand to shape regulatory policy,” Caplan said. For example, Alzheimer’s disease groups backed the Biogen and Eisai drug Aduhelm for an FDA approval when experts considered evidence of the treatment’s efficacy murky. And patient groups need to justify their existence and funding through results — that means convincing drugmakers to develop a treatment and then backing it to the end, Caplan said.
“Most people have the view that patients are separate from the industry and that patients show up and say ‘I want X,’ but that isn’t quite true,” Caplan said. “They’re kind of connected — if a patient showed up and said, ‘I think your drug is bulls---,’ the sponsorship of the patient group would be coming to a rapid end."
Speed of drug development
Haste is a double edged sword when it comes to drug development. A quickly made drug or vaccine can save lives by providing access at an earlier date, but potentially compromise safety or efficacy in the long run, Caplan said.
“There’s a lot of pressure to do faster development post-COVID,” Caplan said. “Everybody saw emergency use authorizations and using real-world evidence as a substitute for a full clinical trial, and speed is becoming something the industry can push for.”
As with all ethical quandaries, there’s more to faster drug development lurking beneath the surface.
“I’m not sure we’ve thought about what the term ‘real-world evidence’ really means or what the cost is when speed means rolling out things that still may fail,” Caplan said.
Post-market approvals
Conditional approval of a drug falls into a similar category as a speedier development — a regulator will grant the go-ahead to a treatment based on unmet medical need with the requirement that a company conduct a post-market study to confirm the drug’s efficacy.
“I don’t think the industry does a good job on phase 4 post-market approvals,” Caplan said. “We don’t know what the hell’s going on half the time to answer questions about whether something is working or not.”
The post-market approval process is ripe for conflicts of interest, he said.
“Sometimes it’s in the industry’s best interest to get it approved, get paid for a couple years and then withdraw it,” Caplan said. “The FDA is under extreme pressure to speed things up from a Congress that has many members who don’t like regulation — I don’t think it serves the public well.”
Price control
No bioethics conversation is complete without bringing drug pricing to the table. A topic that has been discussed widely and to the benefit of politicians campaigning for office, drug pricing remains a thorn in the industry’s side as new policies threaten profits.
“The industry is trying to fight price control, and it doesn’t quite know what to do because it’s clear that there’s sentiment in the U.S. to bargain prices and cut margins,” Caplan said. “[Companies] are used to living on big margins and big profits from the U.S. to sponsor research.”
“Diversity definitions are almost all racial ... but there’s plenty of other kinds such as disability, age, gender, orientation.”
Arthur Caplan
Professor of bioethics, founding head of the Division of Medical Ethics at NYU’s Grossman School of Medicine
Pricing is one of the biggest detriments to the industry’s wider reputation, and the pandemic response is a case study in how positive sentiment can disappear as quickly as it can manifest, Caplan said.
“Biopharma produced a useful vaccine in the initial outbreak, and they stepped up with the government to get regulation and speed, and that was good,” Caplan said. “But part of the basis for distrusting COVID-related products at this point is that they’re pharma-linked — prices are escalating, and that’s not good because people got used to them being cheap.”
Approach to diversity
Although Caplan supports diversity in clinical trials and access to drugs, he said there are times when diversity is not approached in the right way.
“I think we’re obsessed with diversity and trying to do trials the right way, but we have no idea what that means,” Caplan said. “The FDA is thinking that it means representative of the U.S. population, but that doesn’t overlap with significant biological categories to test drugs.”
For example, requiring small populations to be included in a small phase 1 study can be an unnecessary hurdle to development without addressing the core issue of ensuring scientific rigor, he said.
“Diversity definitions are almost all racial in the racially obsessed U.S., but there’s plenty of other kinds such as disability, age, gender, orientation,” Caplan said. “There are a lot of things you can think about that might shape how you recruit a trial, so it’s become a PC issue with little connection to the science of what you need to do to test drugs and vaccines.”
