The long development process of lab screening and animal tests is expensive and weeds out most compounds. Only about 0.1% of potential medicines eventually make it to clinical trials in human subjects. Nevertheless, the pharmaceutical industry continues to produce new oncology products. Hundreds are in the drug development pipeline.  New drugs approved by the FDA are sometimes combinations of old drugs or incorporate new delivery methods to get the active medicine into the body.  On this page we cover New Molecular Entities, which are truly new at the molecular level, and include both small molecule drugs and biologics.

In recent years the FDA has approved on average 13 new molecular entities (NME) per year for cancer treatment. Additional new therapies include combinations of existing drugs and new delivery methods for existing drugs.  About a quarter of the new drugs approved by the FDA in the past decade are for cancer treatment.

How New are the New Drugs?

Most new drugs approved by the FDA aren’t totally new. They are variations or combinations of old drugs – new methods of delivery, etc. A minority are NMEs; the rest are combinations with other drugs or differ from previously approved medicines in method of administration or dosage. The 1984 Hatch-Waxman Act allows the FDA to give market exclusivity to new medicines which are not NMEs and therefore cannot be patented.

When a drug does prove effective enough to be approved, it motivates developers to pursue “me-too” drugs that work in a similar way.  New medicines often focus on the same biochemical targets as medicines approved in the past few years.  Although the number of new oncology drugs has been increasing in recent years, the percentage that work by a novel mechanism declined from 38 percent in 2009 to 12 percent in 2020 according to an analysis published in the Journal of the American Medical Association.  Further the number of new biochemical targets drugs attack is considerably lower than the number of new drugs.  In an average year new cancer medicines are associated with only 4 new biological targets.

New Immunotherapy

In recent years, development of new immunotherapy agents has revolutionized cancer treatment. These drugs induce or potentiate the anti-tumor activity of the immune system.

The immune system identifies tumors and triggers an anti-tumor response. Such response, however, is modulated by stimulating and inhibiting factors. The new immunomodulating agents work by slowing down the action of natural immune system inhibitors and by speeding up natural stimulatory factors. The novelty of this approach stems from the fact that the drug targets the host immune cells and not the tumor itself. Antibody-drug conjugates combine an antibody and a toxin to target malignant cells. Following therapeutic and commercial successes, a lot of efforts are being put in the development of new ADCs.

More on immnotherapy.

See more on notch inhibitors.

Near-term future of chemotherapy

Gene Therapy

Gene therapy has been talked about by futurists for decades and only in the past few years have appeared in the clinic. The idea is that by changing the DNA in the body’s cells, the genetic cause of illness can be addressed. Strategies include inactivating a bad gene, changing a mutated gene to a healthy gene, and putting a new gene in the body to fight disease. Researchers are making progress in employing gene therapy against sickle cell anemia, connective tissue disorders, and retinal disease. Some gene therapy for cancer is have entered clinical use. The most advanced area in anti-cancer gene therapy is CAR-T therapies, chimeric antigen receptor T-cell, which involves genetic modification of T-cells removed from the patient and then re-introduced to the body. In 2017 the FDA approved tisagenlecleucel and axicabtagene ciloleucel which are CAR-T processes, and in 2020 brexucabtagene autoleucel was approved.

Recently Approved Oncology Drugs

Oncology drugs (chemotherapy and immunotherapy agent) approved in recent years:

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Drugs currently approved by the FDA for cancer by year of approval: