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FDA Plans to Improve Access to Drugs that Treat DNA-linked Cancers

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The year 2003 was a landmark year for genetic science: it saw the first complete human genome sequencing, a physiological map for how the body manufactures building blocks at a cellular level. And in the nearly decade-and-a-half since that first full genome, scientists have deepened their awareness about how and why cancer spreads. This knowledge has fueled advancements within the pharmaceutical industry, but critics say that the rigid approval protocol of the U.S. Food and Drug Administration (FDA) might hamper timely progress in cancer-fighting treatments.

“…some of the biggest opportunities in medicine…”

Earlier this month new FDA Commissioner Scott Gottlieb appeared before the Senate panel tasked with oversight of the agency’s budget. One of the upcoming FDA proposals Gottlieb previewed for the panel was the Medical Innovation Access Plan, intended to streamline and hasten the approval for a particular class of drugs. Tumor-agnostic therapies categorize and treat genetically-linked cancers at the DNA-level, instead of merely approaching them according to the bodily systems in which they arise.

In his written remarks for his appearance, Gottlieb wrote: “This represents some of the biggest opportunities in medicine to treat and cure debilitating and, yes, very costly diseases.”

Merck and Loxo: drug companies already taking part in the conversation

The first tumor-agnostic drug was approved by the FDA in May. The pharmaceutical Keytruda, manufactured by Merck, is now available to cancer patients who test positive for a particular gene mutation. Most commonly, the mutated gene appears in cases of endometrial and colorectal cancers, although it has also been discovered in other types, such as prostate, bladder, and breast cancers.

President Trump had criticized the FDA this past winter in remarks to Congress when he referred to the agency’s approval process as “slow and burdensome.” Although the FDA’s average rate of green-lighting meds that treat cancer has sped up in the last few years, drug companies are looking for assistance in getting through the testing protocol and landing in the review stage more quickly.

Earlier this month the drug company Loxo Oncology released results of trials for its tumor-agnostic medication. Loxo was able to demonstrate that the drug was effective in reducing the size of tumors for individuals suffering from a rare genetic abnormality. At present, however, it is unclear whether the relatively small trial will meet the FDA’s requirements for approval of the drug.

Medical Innovation Access Plan seeks to clear obstacles for orphan drugs

Gottlieb said that the FDA will issue drug manufacturer guidance in the coming months regarding bringing treatments for rare conditions to market in less onerous ways. And in the written remarks he prepared for the Senate panel, he said: “We’ll clarify when we may be able to give a broad approval to a cancer drug, in multiple different kinds of molecularly similar cancers, that’s not particular to the tumor being in any one specific tissue or organ.”

Another issue Gottlieb is hoping to ameliorate is that of the FDA backlog. At the moment, there are around 200 pending applications from pharmaceutical companies for drugs that fall under the “orphan” category — in other words, they would target medical conditions that impact fewer than 200,000 patients in the country. Gottlieb made some promises to the Senate panel, including the appointment of dedicated reviewers to those applications for the purpose of eradicating the entire backlog within 90 days.

Looking to the future, Gottlieb further promised that each orphan drug designation request made to the FDA would get an answer from the agency within three months and that, once the existing backlog is dispatched, a pileup of accumulated applications will never occur on his watch.

 

This blog post is provided for educational purposes only and is not offered as, and should not be relied on as, legal advice. Any individual or entity reading this information should consult an attorney for their particular situation. For more information/questions regarding any legal matters, please email info@nelsonhardiman.com or call 310.203.2800.