Smart Patients Get Smart Care™

The World’s Leading Authority for Chronic Lymphocytic Leukemia Patients

Opinion: The FDA is Making it More Difficult for New Drugs to be Approved. Why CLL / SLL Patients Should Care.

In science and medicine, information is constantly changing and may become out-of-date as new data emerge. All articles and interviews are informational only, should never be considered medical advice, and should never be acted on without review with your health care team.

It is not news that chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL / SLL) patients have greatly benefited from breakthroughs in understanding the disease’s biology and the resulting new targeted therapies that have been developed. These include three BTK inhibitors (ibrutinib, acalabrutinib, and zanubrutinib), one BCL-2 inhibitor (venetoclax), and new monoclonal antibodies-especially obinutuzumab. These medications have revolutionized and radically improved outcomes. Those with CLL / SLL are the grateful beneficiaries of this recent “Golden Age.”

Many of the indications for these novel therapies also reached patients sooner than they usually would have through a special FDA process known as the Accelerated Approval Program.

The financial rewards and risks for the companies involved in drug development can be enormous, as is well outlined in Nathan Vardi’s book, For Blood and Money: Billionaires, Biotech, and the Quest for a Blockbuster Drug that gives a close-up review of the approval journey of ibrutinib and acalabrutinib. (It is not often I get to reference a book where I am one of the characters).

The Accelerated Approval Program, as the name implies, makes new treatments available sooner so that more patients might benefit, especially those who cannot wait and urgently need something now to control their cancer’s progression. At the same time, it begins the positive return on investment for the drug developer.

But has that Golden Age that encouraged the explosion of new cancer treatments ended? If so, then, as patients, we should be concerned, not because we are focused on the bottom line for biotech and pharmaceutical companies, but because we want to see many more therapies cross over the approval finish line? Unfortunately, CLL / SLL is not a solved disease. As patients who are now living longer, we need even more treatment options available to us over our lifespans-especially when CLL / SLL relapses. And sadly, it nearly always relapses sooner or later.

The Accelerated Approval Program and related vital issues in the Inflation Reduction Act (IRA), which was passed in late 2022, is how the government plans to control high drug costs. But there are many complicated issues within it that have multiple facets, and there are several valid competing values. Some of these include better access to affordable medications for all, while at the same time encouraging drug innovation. 

CLL Society is fighting for our community’s interests in these and other regulatory and legislative issues through our newly founded CLL Society Policy Institute.

For one perspective on these changing times, Nathan Vardi wrote this opinion piece that provides some background on the financial, investment, and regulatory aspects, but more importantly for us, how these changes may have real clinical implications for all cancer patients: Is the Golden Age of Biotech Stocks Over? 

Take a look. And please consider supporting the ongoing work of CLL Society’s Policy Institute efforts by donating to our efforts.

Stay strong. We are all in this together.

Brian Koffman, MDCM (retired), MS Ed
Co-Founder, Executive VP, and Chief Medical Officer
CLL Society, Inc.