The Genetic Revolution 2.0: Why Paying for Cures Is the Next Great Challenge

We are now in the second great wave of the genetic revolution, not defined by reading the human code of life, but by rewriting it. In recent years, the U.S. Food and Drug Administration has approved more than a dozen gene-editing and cell-based therapies. Over a thousand clinical trials worldwide are testing similar technologies, some designed to intervene even before birth. For the first time, we can treat disease at its source, repairing or replacing the faulty genes that cause illness.

Yet as transformative as this moment may be, the question ahead is not just how far genetic medicine can go, but how we can afford to go there. Each past revolution—from protein therapeutics to genome sequencing to CRISPR gene editing—expanded our ability to intervene in biology. The next must redefine how we pay for the cures we create.

Reading and Rewriting the Code of Life

Modern medicine changed with the rise of molecular biology. The first big change was using human and viral proteins as medicines. Insulin, growth hormone and vaccines for diseases like hepatitis B showed that biology could be used to treat illness. When monoclonal antibodies were developed, protein-based drugs made up almost half of all pharmaceuticals. This started the global biotech industry.

After scientists learned how to make therapeutic proteins, they began to focus on understanding and fixing the genes that control them. Sequencing the human genome was the next major step. Twenty years ago, sequencing a genome cost millions of dollars and took months. Now, it can be done in hours for about $100. This progress has made it possible to treat diseases that were once thought to be untreatable.

New sequencing platforms, like Ultima’s wafer-based systems, can process billions of genetic fragments at once. The main challenge now is not reading DNA, but interpreting it quickly and at a reasonable cost. Sequencing can be done in hours, but analyzing and confirming the results still takes days. This slows down diagnostics and delays important newborn screenings.

Intelligence and Innovation in Genetic Medicine

Technology is helping to solve these problems. Automation, computational biology and artificial intelligence can now analyze genomic data in minutes instead of days. These tools can find sequencing errors, predict clinical outcomes and help design new therapies. Genome sequencing is becoming routine, like a blood test. Hospitals use real-time sequence analysis to find early cancers, adjust treatments and track infections in hours instead of weeks.

Gene therapy has advanced along with precision drugs and AI-driven innovation. HIV treatment showed that understanding genetics can turn deadly diseases into chronic ones. Now, similar methods are being used to treat inherited metabolic disorders and rare genetic diseases. The Metabolic Treatabolome Project has found more than 275 treatable disorders. Some are managed with enzyme replacement or drugs that change metabolic pathways, while others are treated by editing DNA directly.

CRISPR and other gene-editing tools are now being used to fix the mutations that cause disease. Studies like GUARDIAN and BeginNGS show that sequencing newborn genomes can find hundreds of treatable disorders at birth, which is much more than traditional tests. AI and genetic engineering are helping medicine move toward curing diseases at the molecular level instead of just managing symptoms.

Genetic Screening Goes Global

These advances are now being used in public health systems. In 2025, Florida’s Sunshine Genetics Act created the first statewide newborn genome screening program in the United States. The goal is to find and treat genetic conditions before symptoms appear. This is part of a larger global trend.

In the United Kingdom, the Newborn Genomes Programme, run by Genomics England, aims to sequence 100,000 babies over two years. Early data suggest that genome screening could double detection rates compared with conventional blood-spot tests. Australia’s BabyScreen+ study and Singapore’s National Precision Medicine Programme are following similar paths. Across Europe, the Nordics and the Netherlands are integrating full-genome sequencing into routine neonatal care.

Together, these programs show a global change in preventive medicine and bring a new challenge. If gene editing can change the health of families and populations, policies and payment systems need to adapt to make sure people have access. The main barrier now is economic, not scientific.

Paying for a Genetic Revolution

The promise of gene therapy comes at an extraordinary price. Some treatments cost more than $3 million per patient. Zolgensma, for spinal muscular atrophy, costs $2.1 million; Hemgenix, for hemophilia B, $3.5 million; and CAR-T cell therapies such as Kymriah and Yescarta often cost more than $475,000. While these prices reflect decades of research and production complexity, they also reveal deep flaws in how health systems value innovation.

Traditional pricing models do not work well for one-time cures. The U.S. fee-for-service system pays for each dose or procedure, which does not fit treatments that can eliminate disease. Even bundled payment systems like Medicaid’s DRG model can make it hard for hospitals to offer expensive therapies. As a result, some life-saving cures are too expensive for many people.

To close this gap, the Centers for Medicare & Medicaid Services introduced the Cell and Gene Therapy Access Model in 2025—an outcomes-based reimbursement system. Under this model, states negotiate contracts with drug makers, tying payments to results: if a therapy works, it is fully reimbursed; if not, payments are reduced or withheld. This approach shares risk and rewards performance, redefining “value” as measurable health improvement over time.

Private insurers are also testing shared-risk pools and amortized payment systems that spread costs over several years. The principle is straightforward: a one-time genetic cure that prevents decades of hospitalization, disability and chronic treatment should be considered an investment rather than an expense. In this framework, curative therapies may ultimately save rather than strain health systems.

However, success depends on having the right scale, data and political support. The systems needed to track patient outcomes over time and across different payers are still being built. As more gene therapies are approved—20 to 30 each year by the end of the decade—there will be more pressure to create fair and clear ways to pay for them. The main question is whether our economic systems can keep up with scientific progress.

Fulfilling the Promise

From proteins to genomes, from sequencing to editing, each generation of biotechnology has become increasingly intertwined with technology—and with economics. The next breakthrough will not be molecular but structural: a new financing framework that allows the cures we discover to reach those who need them most. Genetic medicine has given humanity tools to rewrite fate itself. The decisive question is whether we can also rewrite the economics of health to match

Read Dr. Haseltine's latest piece with

Forbes

© William A. Haseltine, PhD. All Rights Reserved.