Executive Leadership | From the Editor-in-Chief

June 2019
The End of Chronic Therapies?
Alberto Grignolo

Alberto Grignolo, PhD
Global Forum
Fellow of DIA


ell, maybe not in the immediate future, but the end may indeed come.

The recent FDA approval of a gene therapy for Spinal Muscular Atrophy (SMA) raises fundamental questions about the future relevance of traditional medicines.

FDA’s press release announcing this approval states: “A one-time intravenous administration of Zolgensma results in expression of the SMN protein in a child’s motor neurons, which improves muscle movement and function, and survival of a child with SMA.”

A single dose apparently delivers a cure. This is transformative.

Technology and molecular biology are progressing and converging at such speed and to such an extent that within a decade it may be possible to predict, prevent, or cure myriad diseases at the fetal or neonatal stage and later in life; gene editing and highly specific targeted interventions applied even once at the cellular or subcellular level, perhaps before the disease is even expressed, could make many drugs as we know them today (particularly chronic treatments) unnecessary and irrelevant. This would be a major disruption.

The underlying precondition is a far more profound understanding of the molecular biology of many diseases than we have today, allowing highly targeted one-time interventions to deliver rapid and complete cures, often by activating (or reactivating) the body’s own defense mechanisms. Precision medicine in action. It’s already happening today.

What does this mean for drug development stakeholders?

  • Patients will increasingly be invited to participate in ground-breaking clinical trials and to contribute real world data in unprecedented numbers and ways in order to inform the clinical research of the future, specifically in cell and gene therapy. This is already happening.
  • Regulators must be ready to review the most advanced science and cutting-edge interventions with an open mind and with the patient at the center. This is clearly already happening.
  • Industry must be ready to abandon traditional drug development norms and act “outside the box” to speed breakthrough innovations to patients, clinicians, regulators, and payers. The race is already on.
  • Reliance on revenues from chronic therapies with medium-to-high prices will transition to reliance on revenues from one-time therapies at “astronomical” prices that will nonetheless be cheaper than the total cost of chronic patient care. This is starting to happen.
  • Sponsors, HTAs, and payers will need to think differently about the value of medicines and devise novel outcome and payment models that society can afford. This is already recognized and is starting to happen.

We have entered a “golden age” of science, medicine, regulation, and therapy. The question of patient access to all of this innovation looms large and it must be answered.

As patients, we may find that our medicines cabinet will become rather empty, replaced by one-time therapy solutions; after that, we may not need to take drugs every day, or several times a day, or at all.

Clinicians may rationally argue that diabetes, obesity, congestive heart failure, renal failure, rheumatoid arthritis, Alzheimer’s disease, schizophrenia, stroke, and many other pathologies will not be amenable to one-time curative interventions. Might we expect disruptive innovation there, too? If we can imagine it, it could happen.

Is this fiction? For centuries, so was air travel.

See you at the airport.

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