Diary

Free to Choose Medicine Offers a Pathway for Rare Diseases

AP Photo/Gerald Herbert

Rare Disease Day, which recognizes the 7,000 identified rare diseases that currently have no known cure, occurs on the last day of February every year. Even though a rare disease is defined by affecting 200,000 Americans or less, this is no small matter. These 7,000 diseases affect 30 million Americans.

As we recognize the millions of families suffering from these diseases, it is important to utilize this day for advocacy as well. We all want the safest and most effective drugs on the market for these patients, but the current drug approval process is outmoded and should be reformed to encourage innovation and treatment development for those suffering with rare diseases.

There needs to be a discussion about reforming the Food and Drug Administration’s (FDA) antiquated drug approval process, which currently takes, on average, 12 years and costs $2.9 billion.

Perhaps FDA should consider what has worked well in the past. For example, in the 1980s, the AIDS community worked around the clock to push for expedited approval of treatment options for those suffering with HIV/AIDS. It worked, saving more than 75,000 lives.

More recently, FDA’s streamlined process helped create COVID-19 vaccines that were developed in less than a year and have been administered in all 50 states. Why can’t rare diseases get the same consideration in terms of expedited approval?

RNA technology is being studied and used to treat rare diseases around the world. Individuals with rare diseases face many trials and tribulations, including getting access to treatments. It is difficult to conduct clinical trials when there is only a small number of patients with said disease. That is why RNA research is so crucial and can be extremely beneficial for the rare disease community.

One example of this is the treatment development for Mila, a 10-year-old in Colorado who was diagnosed with Batten disease, a rare genetic condition. At the time of diagnosis, Mila had lost her sight, and ability to walk and talk. With no known cure, the disease progressed.

Mila’s mother, Julia Vitarello, had not yet given up hope and started Mila’s Miracle Foundation (MMF). She raised $5 million from a grassroots campaign of more than 6,000 supporters. These efforts lead to the development of a genetically targeted treatment for Mila’s specific condition.

Gene therapy allows for development in rare genetic mutations like this. Miraculously, the drug for Mila’s condition was created in just over a year.

The treatment worked, initially. Mila was able to see again. Her seizures subsided.

Unfortunately, Mila’s disease had progressed too far. Sadly, she is no longer with us. However, her legacy still lives.

Mila’s harrowing story is what Rare Disease Day is all about.

Because of brave Americans like Mila, progress in drug development is being utilized for future cures. Specialized gene development for Mila was the first of its kind. Milasen, which was developed at Boston Children’s hospital and named in Mila’s honor, offers hope to all who suffer from the rare disease that took Mila.

The Heartland Institute has a similar proposal that would expedite approval of safe and effective drugs. Free to Choose Medicine (FTCM) offers an alternate pathway for unapproved therapies, similar to the drug created for Mila.

Under a FTCM track, unapproved therapies would be required to pass initial FDA safety testing and at least one efficacy test before becoming available for patients. Of course, there would need to be doctor approval and the requirement that no alternative options exist, which is the case for rare disease patients.

Medicine is transformative. Innovations like gene therapy represent the next frontier in drug development. It is time for FDA to reform its convoluted drug approval process so that potentially life-saving drugs are more accessible for all patients, especially those with rare diseases, before it is too late.

Christina Herrin ([email protected]) is the government relations manager at The Heartland Institute, a nonpartisan, free-market think tank headquartered in Arlington Heights, Ill.