High risk, high capital investment leads to breakthrough in cancer treatment

“Congress recently passed legislation that imposes state price controls on certain innovative drugs. The bill could jeopardize investment in the next generation of breakthrough treatments and delay access to medicines.

Everyone knows someone whose life has been touched by cancer, be it a parent, sibling or friend. But it may be rarer to know a family affected by childhood cancer. Yet cancer is the second leading cause of death in people under the age of 14, affecting nearly 10,500 children annually in the United States.

Fifty years ago, a child diagnosed with cancer had an average five-year survival rate of only 58%. But thanks to biopharmaceutical companies’ investments in discovery science, we have achieved medical breakthroughs that have dramatically improved survival rates, with 85% of childhood cancer patients living five years or more.

In the past decade alone, scientists have discovered dozens of new drugs, offering families hope that one day their child can proudly say, “I’m a cancer survivor.” However, recent events in Washington could jeopardize progress on breakthrough treatments and drugs.

How life-saving treatments are developed

Take, for example, the innovative therapy Kymriah. 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 in pediatric patients with aggressive acute lymphoblastic leukemia. The therapy modifies the patient’s immune T cells to destroy the leukemia cells after they are reinfused into the patient’s body.

The first-of-its-kind therapy had remarkable results: “83% of patients” achieved remission within 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 only had weeks to live – recently celebrated her?10th anniversary?of being cancer-free.

Remarkable clinical trials led to Kymriah becoming the first CAR-T therapy approved by the 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 came into the limelight as an innovative therapy to treat COVID-19, are currently being tested as a new way to defeat childhood cancers.

See Y-mAb Therapeutics treatment, naxitimab. Innovative monoclonal antibody therapy is being studied in patients with aggressive high-risk neuroblastoma, a rare cancer of the adrenal glands. Naxitimab recently received accelerated approval from the FDA following promising clinical trial data.

But these innovations are not inevitable. Breakthrough treatments are the result of decades of high-risk, high-capital investment in cutting-edge research. Even finding new uses for existing drugs requires significant testing and new clinical trials, culminating in years of work plus millions of dollars. To support 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 stems directly from the US free market framework: a robust venture capital network and strong intellectual property (IP) protection. The US innovation ecosystem – and the public policies that support it – have led to the approval of many new pediatric drugs in recent years.

In 1997, the Food and Drug Administration Modernization Act created a “pediatric exclusivity term” that provides an additional six months of market exclusivity for sponsors who study their products in children. Between 1998 and 2012, 108 medicines received a pediatric indication due to the legislation.

Similarly, the Creating Hope Act of 2012 created additional market incentives for innovators to invest in treatments for rare pediatric diseases. Since the law’s enactment, the FDA has approved 34 drugs for rare pediatric diseases, including two pediatric cancers.

This legislation serves as a reminder that good public policy matters, spurs investment in innovation, and offers hope to thousands of families each year who receive a childhood cancer diagnosis. But bad public policy can stop this innovation.

How price controls are negatively impacting breakthrough treatments

Congress recently passed legislation imposing state price controls on certain innovative drugs. The bill could jeopardize investment in the next generation of breakthrough treatments and delay access to medicines.

As a world leader in biopharmaceutical innovation, American patients have “immediate and extensive access” to new life-saving treatments and cures. However, price controls can limit that access, forcing US patients to wait in line for new drugs just like patients in other developed nations that use price controls, including Germany, the United Kingdom and Korea.

What’s more, the legislation will destroy the innovation pipeline. New research from Vital Transformation shows that of 110 currently approved therapies, only six would reach the market if government price controls were in place. This means that patients with 104 different conditions would be left to hope for a cure.

Remember the example of Kymriah, which resulted in 83% remission for many patients who had previously exhausted their options. Kymriah illustrates how a crazy idea—as CAR-T once seemed to many—can become a game changer for patients with devastating diagnoses. In a world without Kymriah, leukemia patients would be left to hope for a cure, but so would patients with lymphoma, glioblastoma and pancreatic cancer.

What should Congress do?

US public policy should allow companies to invest in the impossible. With the right laws in place, innovators can devote time, capital and resources to develop – and continually improve – life-saving drugs. Each discovery not only improves individual medicines, but also enriches our collective knowledge and experience, maintaining a virtuous cycle of transformation and growth. Unfortunately, with the wrong laws in place, this cycle can stop.

We urge policymakers to preserve the existing US 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 turn into a big what-if.

Image Source: Deposit Photos
By YAYImages
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