Today, it takes approximately 10 years and $1 billion to develop a new medicine, including new cancer treatments. The time and cost of development have been on the rise for the past 40 years.
It takes a great deal of resources and persistence to complete the lengthy trek from exploratory process to FDA-approved treatment.
During the past 20 years, the overall investment in research and development of new cancer therapies has been increasing. Sounds like we're on the right track... or are we?
Despite increased time and money invested, the probability of getting a new cancer medicine to patients has improved only slightly since the 1990s. Today, new cancer medicines take longer to bring to market than do therapies for other complex illnesses, including HIV. Without changes in the clinical trial and approval processes, that trend is likely to continue.
There are many reasons for longer development times. According to former FDA Deputy Commissioner Scott Gottlieb, one of the most important factors may be "an increasingly unreasonable hunger for statistical certainty on the part of the FDA."
We know more about the genetic mutations that trigger cancer. We're able to detect who has these genetic mutations. As a result, we can quickly learn who would benefit from a treatment targeting such triggers.
In recent years, however, the FDA has been demanding bigger studies that measure the average survival rate in trials of cancer patients instead of confirming that a specific targeted treatment has stopped a tumor from growing. The FDA claims that the larger studies protect patients from unsafe medicines. But as the chart above shows, such requirements result in an inordinate amount of time and expense needed to develop a medicine. And that deprives patients who are critically in need of new treatments.
Ironically, the FDA is taking the opposite approach it pursued in responding to pressure from HIV/AIDS activists. The agency established an accelerated approval process that delivered new medicines to market in 2 years instead of 10. Rather than denying access to treatment by conducting randomized trials with placebos, the FDA agreed to 3 things: First, it based approval on how well an HIV drug increased the number of healthy immune cells. Second, it allowed HIV patients and doctors to test new medicines in the "real world" and use that information to support approval. Third, it agreed to speed up the review of all that information through a fast-track approval pathway.
Unfortunately, the accelerated approval approach has not been applied to the development of new cancer medicines. Despite the fact that companies are increasingly developing cancer medications that are more likely to work, the availability of these new therapies continues to lag.
Legislation passed in 1981 that provides tax breaks and longer patent protection for medicines that treat rare, often fatal, diseases known as "orphans" because they were overlooked by researchers.
Many new cancer medicines are for groups of people who have a specific cancer-causing genetic mutation. These medicines are classified as orphan drugs because they are intended to treat so-called orphan diseases, conditions that affect fewer than 200,000 persons in the United States.
The FDA is allowed to speed up the development and approval of medicines for orphan diseases. In particular, the FDA has the authority to allow drugs to be studied in these smaller patient groups without requiring the need to also give an inactive placebo, as done with other major illnesses. However, few cancer medicines have been granted fast-track or accelerated approval.
Cancer drugs going through a "special status, faster path" didn't go much faster at all. Between 2007 and 2011, patients got those much-needed medicines less than 1 month faster than those the FDA did not regard as special.
Orphan drug classification does not necessarily speed up the approval process. In fact, orphan therapies have actually taken longer than non-orphan therapies to get to patients.
While the number of new medicines for rare diseases increased from 10 in 1970 to more than 200 in 2010, it actually takes more time to get approval for orphan drugs than for other drugs. That means new cancer treatments actually could take longer to get FDA approval.
In 2010, the FDA refused to approve the only medicine that treats a rare and fatal lung disease called idiopathic pulmonary fibrosis. The medicine, pirfenidone, has been available in Japan since 2008 and in Europe since 2011. The FDA rejected the drug because it only showed efficacy in a single big trial, not the 2 large studies the FDA wanted but does not have to require for orphan medicines.
While new orphan designations have increased, the actual number of orphan drug approvals has declined between 1985 and 2010. In many cases, assigning a new medicine to a fast-track or accelerated orphan pathway doesn't count for much, even when researchers have a very good understanding about who will benefit from new treatments.
Our understanding of cancer today exceeds what we knew about HIV when new drugs for that disease were speeding through the FDA. New technologies, such as genomic and molecular testing, help researchers, doctors, and patients develop more personalized and predictive cancer treatments.
There are more than 700 targeted cancer treatments in development, each of them with the potential to impact cancer the way HIV therapies impacted AIDS. So why are we taking more time, not less?
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