Drug Development: Our Wondrous, But Challenging, Future

Our ongoing national health care crisis has been going on for so many years largely because remarkable new therapies continue to transform how medical care is provided.

There has been more medical progress since World War II than in all previous human history. The progress in the last decade has been especially exciting. The Food and Drug Administration is setting records in the number of new drugs approved, with a super surge in drugs to treat various cancers.

But this unprecedented and remarkable progress has not come without consequences — in this case, higher health care costs. Virtually all the new therapies cost more than the drugs they replaced, but as a result, people are living longer and need more health care services as they age, thereby placing a higher demand on the need for more health care practitioners and medical facilities.

In terms of medical progress, as the expression goes, “you ain’t seen nothing yet.” While the past decades have been called the “golden age” of drug development, we are now entering the “platinum age.”

In this platinum age, we will be able to administer one-time therapies that save lives or enable people to avoid debilitating diseases. We will see sick infants today, who in the past would not survive infancy, lead a normal lifespan. We will see people who have a chronic disease — who now must visit their doctors frequently and take multitudes of drugs — lead healthy lives.

These new therapies will continue to affect how medicine is practiced, how patients are treated and how health care is financed. For example, earlier this year, one company announced that its gene therapy for a rare disorder called spinal muscular atrophy would be priced at $2.1 million.

Hospitals that administer CAR-T stem cell transplants may charge as much as $1.5 million, with the majority of the cost being for hospital and medical care. As additional therapies become available, the cost for the therapy itself plus the associated hospital and medical care will be comparable to one another.

These new treatments require extraordinary expertise to develop and administer. Each therapy is developed specifically for the individual recipient. It is a very costly process, unlike anything we have seen in the past.

At the same time, of course, the value of these new treatments for patients and their loved ones is incalculable — how do you calculate enabling an infant who faced certain death to live a normal life, or enabling people with disabling chronic diseases to be cured? Even though the new therapies appear expensive, some of them will lead to savings in aggregate health care costs by enabling people with chronic illnesses to avoid a lifetime of special medical attention and costly drugs.

Parts of our health care system already have acknowledged and embraced the coming changes. Genetic testing programs and newborn screenings enable us to diagnose diseases more quickly and efficiently. Advanced medical training is creating a new cadre of health care professionals who can diagnose and treat/prevent diseases that until now could not be treated effectively.

Insurers are developing new insurance and reimbursement models to facilitate cost-sharing. The FDA is adopting new policies to help companies research and test new therapies and secure rapid approvals.

Lagging, however, are legislative and political policies — at both the federal and state levels — that would establish a framework and uniform standards for integrating these new treatments into the rapidly evolving medical education, hospital, regulatory and reimbursement systems. The political debate today focuses on how we currently provide health care services. We seldom hear from political leaders or candidates about how we will accommodate and adjust to the fundamental changes now getting underway.

It is critical that our policymakers and politicians understand how these new treatments work. Gene therapies alter genes that cause diseases in an individual person. By altering the disease gene, these therapies enable people to avoid a disease entirely or be cured of one they already have.

Unlike the life-extending drugs that we are familiar with, which are taken over a lifetime, gene therapies are given just once. A single treatment lasts a lifetime.

Hundreds of gene therapies are in the research and development pipelines of biotech companies. They are being developed for common diseases such as hemophilia, Parkinson’s disease and HIV. But most are being developed for rare genetic diseases that are detected at birth or childhood. As a consequence of the clinical testing to date, many children who would have died soon after birth or during childhood are alive and thriving.

Stem cell therapy is different but equally promising. It helps repair diseased tissues by transferring healthy cells into a patient with a disease. For example, bone marrow transplants already are being used to treat certain conditions such as blood cancers.

It is unlikely that the presidential candidates will mention gene or stem cell therapies in their campaigns, but any solutions they propose for today’s health care crisis must take note that these treatments will add a new dimension to health care in the future. The upcoming platinum age of drug development will change the health care landscape forever, and it’s just beginning. Our public and health care policies must keep pace.


Wayne Pines is president of healthcare at APCO Worldwide in Washington, D.C., and a former associate commissioner at the FDA.

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