Editorial: In drug development, science isn’t enough

If you ask the commissioner of the US Food and Drug Administration about the greatest hurdles to treating disease in the US, he doesn’t point to any of the scientific problems his agency is working on. Nor does he lift up any one drug as a breakthrough, a solution to society’s health woes.

Instead, when Commissioner Robert Califf was presented with this question at a conference of the Biotechnology Innovation Organization in June, he spoke about social problems: access to primary care, nutritious food, and high-quality information.

“As simple as it sounds, I think the most important thing we could do in the United States is institute a primary care system,” Califf said onstage.

The life sciences industry is riddled with stories of scientific success followed by market failure. The first and only human vaccine against Lyme disease, LYMErix, was shelved only 3 years after it hit the US market in 1999, because of a perfect storm of growing antivaccine sentiment, concerns over a side effect (which turned out not to be linked to the shot), and a sense among the public that Lyme disease wasn’t all that prevalent—ticks had yet to march across the US the way they have in recent years.

Times change. Tick-borne illnesses are on the rise across the US and Europe, as is the understanding that Lyme disease can be quite serious, leading to chronic conditions for about 5–20% of people according to Columbia University. Pfizer and the vaccine maker Valneva are testing a new Lyme vaccine in Phase 3 trials that works much the same way LYMErix did. If it succeeds, there is still the issue of vaccine hesitancy amongst the public. And it remains to be seen whether the US Centers for Disease Control and Prevention would make a widespread recommendation, which would guide insurance coverage.

Even where demand seems straightforward, issues of access are a problem. When the FDA approved the sickle cell disease treatment Casgevy in December, the medical community hailed it as a victory for science—the first CRISPR-based gene editor to cross the regulatory finish line and possibly a full-on cure for a painful hereditary illness that affects more than 100,000 people in the US and 20 million people worldwide. Less in focus was the enormous burden of taking Casgevy. The medicine is made from a patient’s own cells, which are extracted and isolated in the hospital, sent to be genetically modified in a manufacturer’s labs, then infused back into the body. In the meantime, the patient has to go through chemotherapy to clear their bone marrow of diseased cells and make room for new ones. A monthslong hospital stay is a tall order for any person, if it’s even possible.

In business terms, when medicine is inefficiently distributed, for instance undermining rational incentives for its use, it’s a market failure. In more human terms, it means sick people aren’t getting the treatment they need. No medicine is effective if the people it’s meant for cannot reach health-care providers, have had experiences that undermined their faith in medicine, or do not have the time or money it takes to visit a doctor. Meanwhile, the industry wheel rolls along; companies stop making unprofitable drugs, and R&D’s focus turns elsewhere.

The World Health Organization describes these types of barriers as social determinants of health, and addressing them has been an organizational priority since 2005.

It’s up to the life sciences enterprise to help improve global patient access. This means expanding how the industry conceives of drug development to include both innovating scientifically and building up some of the medical infrastructure needed to get the right drugs to the right patients.

For instance, drug developers could engage regulatory and medical affairs specialists before development begins. Hire executive-level patient liaisons to establish relationships with advocacy groups, and set up patient steering committees that can contribute to decision-making around clinical trial design. Invest in communities that the medical establishment has marginalized. Make clinical trials more accessible—and, consequently, inclusive of people with diverse backgrounds—by including remote options and working with community health centers. And make existing medicines easier to take, thus bridging the gap for people who can’t find the time or money to sit in an infusion chair every other week.

As Califf says, “It’s not rocket science.” It’s health care.

This editorial is the result of collective deliberation in C&EN. For this week’s editorial, the lead contributor is Rowan Walrath

Views expressed on this page are not necessarily those of ACS.