In 2018, my daughter, Mila, became the first person in the world to receive a medicine designed for one person, an antisense oligonucleotide (ASO) called “milasen.”

Last week, the world received the breaking news that a baby named KJ had received the first personalized CRISPR treatment — one that may save his life. Just as technology is revealing thousands of genetic conditions behind previously unexplained symptoms, Mila and KJ’s stories are proving an entirely new way of treating those who suffer from genetic disease. The new era of individualized medicines is here.

For nearly 80 years, drugs have been developed almost entirely for large populations due to profitability. This model utilizes extensive, costly clinical trials with hundreds of patients, measuring success by average response, often helping only a subset of patients. The traditional approach may work for a few dozen genetic disorders that affect large numbers of people, but certainly doesn’t for 10,000 other small genetic diseases.

We need a new model, one I like to think of as “genetic surgery” or “interventional genetics” — a marriage of drug development and surgical intervention. This new model would benefit both patients and the FDA, as the U.S. figures out how to lead the world in scientific and medical innovation while at the same time cutting costs and updating the way our government works. This model might usher in an FDA 2.0, one in which the agency’s mandate to protect public health is inclusive of ensuring patients rapid and safe access to lifesaving technologies, no matter for how few people are affected.

The system of drug development and access has not kept pace with the revolution that has come for many other aspects of our lives. Recent scientific breakthroughs allow us to identify the root cause of diseases and create hyper-targeted treatments that are significantly more effective. But this level of diagnostic precision inevitably breaks up diseases into smaller and smaller groups, eventually requiring thousands of medicines each for just a few people.

This new paradigm started with my daughter, Mila. Growing up in Colorado, she had been running around, skiing and climbing, laughing and singing her favorite songs. But between 4 and 6 years old, she started losing her vision, her words, her ability to walk and eat, and had up to 30 seizures per day. My previously healthy, outgoing little girl rapidly degenerated before my eyes. After years of countless doctor and therapy appointments in search of an answer, genetic tests showed that Mila had a form of Batten disease, a fatal condition affecting just 25 children in the world. I was told she would lose each of her abilities in the next few years, then die. And just like that, my life as I had known it ended.

Mila was still smiling and laughing despite losing so much, so I said to myself, “If she can fight, so can I.” I immediately started a foundation, raised millions of dollars selling lemonade and cookies with the help of generous people around the world, put scientists together, and raced against time alongside physician Tim Yu and his team at the Division of Genetics and Genomics at Boston Children’s Hospital. Despite being told we were 10 years away from any hope, just one year later we moved to Boston and Mila made history, receiving the first ever truly personalized medicine.

Initially, her seizures stopped, she started eating by mouth again, sat up straight, and lifted her legs higher. Most importantly, her laughter and smiles, which had begun to fade, returned. But her medicine came too late and a few years later, Mila died at just 10 years old.

Until recently there was nothing we could do for children like Mila. We could barely find them, let alone treat them. But all that has changed.

In his book, “Blind Spots,” new FDA Commissioner Marty Makary wrote that challenging the norm in medicine is difficult but necessary. We are clinging to the traditional ways patients have accessed medicines, but the status quo is neither working nor acceptable. History has shown us that a big break from the past is possible when there’s a moral imperative. Today, that’s exactly what we face: Tens of millions of children around the world are dying of genetic diseases. Yet in the seven years since milasen, only a few dozen children have been treated with individualized medicines, many of whom saw dramatic reduction in seizures and improved mobility, and in some cases, with earlier intervention, little to no further disease progression.

Kiran Musunuru and Rebecca Ahrens-Nicklas have pushed us even further by diagnosing baby KJ at birth and treating him at just 6 months old, potentially in time to stop his disease. But like Mila, KJ required a medicine unique to just him.

Now that the new reality of individualized medicines is coming to fruition, we need a system that can keep up.

What should that look like? Some inspiration may come from the highly personalized fields of surgery and transplants, which require doctors to assess each patient individually, carefully weighing the risks of intervention against the risks of not acting. Surgeons can’t wait years for multiyear clinical trials and approvals when treating a single dying child; they must act swiftly. Within weeks, in many cases, they consult with the family and their colleagues, discussing potential benefits and risks. Ultimately, if the decision is to move forward, they pull out the scalpel and operate.

While innovative interventions carry risks, often quite high, the risk of inaction in many cases is inevitable death. Clear guardrails allow surgeons just enough leeway to do what’s best for each patient, while also trying better approaches that often become the new standard, helping patients around the world. All of this falls under an accreditation system, in which safety is ensured through rigorous standards and processes, allowing for a necessary level of individualization.

To create a genetic surgery system, the FDA should work toward proportionate regulation, moving away from approving one medicine at a time to approving a process. This system would prioritize the sharing of data and information, allowing for regular improvements in standards and innovation. Medicines would be developed and administered by following standardized methods and guidelines, allowing for individualized, safe, and rapid intervention. In a critical shift, tying reimbursement to medicines that meet the approved process in the new system would incentivize companies to join hospitals in developing individualized medicines at scale, allowing exponentially more patients amenable to today’s technologies access to potentially life-changing treatments. Families would no longer carry the burden of raising millions of dollars and driving drug development, but would instead be referred by their child’s doctor to a treatment center where a medicine might already be available or could be commissioned.

If we’re successful in putting in place a new system, we can begin to solve the global health crisis by connecting exponentially more children to targeted treatments early in life so that eventually they and their families never know life with disease. We could see more productive members of society, fewer in-patient hospital stays and medications, doctors offering their patients treatments instead of palliative care and an entirely new marketplace of companies producing thousands of individualized medicines. And all patients will feel they have been given the right to safely access the technologies available today, no matter how few. If medical technology continues to develop at this pace and the regulatory system does not, we abandon hundreds of thousands of dying children in the U.S. — a reality that is not acceptable.

Julia Vitarello is Mila’s mom and founder/co-founder of Mila’s Miracle Foundation, the N=1 Collaborative, and EveryONE Medicines, the first and only company solely focused on individualized medicines.

This piece first appeared in STAT.