“Congress recently passed legislation that imposes government price controls on certain innovative medicines. The bill could jeopardize investment in the next generation of breakthrough treatments and delay drug access.”
Everyone knows someone whose life has been impacted by cancer, be it a parent, a sibling, or a friend. But it is rarer, perhaps, to know a family touched by pediatric cancer. Yet, cancer is the second leading cause of death in individuals under 14, impacting nearly 10,500 children annually in the United States.
Fifty years ago, a child diagnosed with cancer had a median five-year survival rate of only 58%. But, thanks to biopharmaceutical companies’ investments in discovery science, we’ve achieved medical breakthroughs that drastically improved the survival rate, with 85% of childhood cancer patients living five years or more.
In the last decade alone, scientists have discovered dozens of new medicines, offering hope to families that one day their child might proudly say, “I am a cancer survivor.” However, recent developments in Washington could jeopardize the march of progress for breakthrough treatments and medications.
How Life-Saving Treatments Are Developed
Take the innovative therapy, Kymriah, for example. Developed in partnership between the University of Pennsylvania, the Children’s Hospital of Philadelphia, and Novartis, Kymriah is an innovative CAR T-cell therapy first studied for pediatric patients with aggressive acute lymphoblastic leukemia. The therapy modifies a patient’s immune T cells to destroy leukemia cells once re-infused into the patient’s body.
The first-of-its-kind therapy had remarkable results: 83% of patients achieved remission in 3 months, while 66% were alive two years after the infusion. The first patient ever treated with Kymriah – who at one point was told she had only weeks to live – recently celebrated her 10th anniversary of being cancer-free.
The remarkable clinical trials resulted in Kymriah becoming the first CAR-T therapy approved by FDA in 2017. Since then, Kymriah has been approved to treat certain types of lymphoma and is currently being studied for cancers with limited effective treatments, including glioblastoma and pancreatic cancer.
And there are many more promising treatments in the pipeline. For example, monoclonal antibodies, which entered the public spotlight as an innovative therapy to treat COVID-19, are currently being tested as a new way to conquer childhood cancers.
Look at Y-mAb Thearpeutics’ treatment, naxitimab. The innovative monoclonal antibody therapy is being studied in patients with aggressive high-risk neuroblastoma, a rare cancer of the adrenal glands. Naxitimab was recently granted accelerated approval from the FDA following promising clinical trial data.
But these innovations are not inevitable. Breakthrough treatments result from decades of high-risk, high-capital investment in cutting-edge research. Even finding new uses for existing drugs requires considerable testing and new clinical trials, culminating in years of work plus millions of dollars. To sustain this investment in innovation, it is critical to have an effective legal and regulatory ecosystem.
The United States Leads the World in Biopharmaceutical Innovation
The United States’ leadership in biopharmaceutical innovation has resulted directly from the U.S.’ free market framework: robust venture capital network and strong intellectual property (IP) protection. The U.S. innovative ecosystem – and the public policy that supports it – has resulted in the approval of many new pediatric medicines in recent years.
In 1997, the Food and Drug Administration Modernization Act created a pediatric exclusivity term which provided an additional six months of market exclusivity for sponsors who study their products in children. Between 1998 and 2012, 108 drugs received a pediatric indication due to the legislation.
Likewise, the Creating Hope Act of 2012 established additional market incentives for innovators to invest in treatments for rare pediatric conditions. Since the Act’s enactment, the FDA has approved 34 medicines for rare pediatric diseases, including two pediatric cancers.
This legislation serves as a reminder that good public policy matters, driving investment in innovation and offering hope to thousands of families each year who receive a childhood cancer diagnosis. But bad public policy can stop that innovation in its tracks.
How Price Controls Negatively Impact Breakthrough Treatments
Congress recently passed legislation that imposes government price controls on certain innovative medicines. The bill could jeopardize investment in the next generation of breakthrough treatments and delay drug access.
As the world leader in biopharmaceutical innovation, American patients have immediate and extensive access to new lifesaving treatments and cures. However, price controls could limit this access, forcing U.S. patients to wait in line for new medicines just like patients in other developed nations that utilize price controls, including Germany, the UK, and Korea.
What’s more, the legislation would devastate the innovation pipeline. New research from Vital Transformation shows that, of 110 currently approved therapies, only six would have made it to market if government price controls had been in place. That means patients with 104 different conditions would have been left hoping for a cure.
Think back to the example of Kymriah, which led to 83% remission for many patients who had previously run out of options. Kymriah illustrates how a crazy idea – like CAR-T once seemed to many – can become a game changer for patients with devasting diagnoses. In a world without Kymriah, leukemia patients would be left hoping for a cure, but so would lymphoma, glioblastoma, and pancreatic cancer patients.
What Congress Should Do
U.S. public policy must enable companies to invest in the impossible. With the right laws in place, innovators can take time, capital, and resources to develop – and continually improve – lifesaving medicines. Not only does each discovery improve individual medicines, but it also enriches our collective knowledge and expertise, sustaining a virtuous cycle of transformation and growth. Unfortunately, with the wrong laws in place, this cycle could halt.
We urge policymakers to preserve the existing U.S. framework—the fruits of which have offered hope and healing to thousands of families with daunting medical diagnoses. We can’t let the next big idea become a big what-if.
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