FDA’s Recent Approval of Radioactive Drug Reveals Criteria for Market Entry

Pharmaceutical Drugs Displayed at a Health Care Facility. Photo of products regulated by the FDA on display at a health care facility. Photo by Olivia P. Walker

On 26 January, the U.S. Food and Drug Administration (FDA) announced its approval of the first radiopharmaceutical — a drug that contains a radioactive substance — for the treatment of GEP-NETS, a rare group of digestive tract cancers.

According to an  FDA News Release, “This approval demonstrates how the FDA may consider data from therapies that are used in an expanded access program to support approval for a new treatment.” The name of the drug is Lutathera. Lutathera was approved through an accelerated approval application process.


Expanded Access programs are regulatory mechanisms by which patients are granted access to investigational drugs — to include biologics and medical devices. Investigational drugs are not FDA approved. Meaning, their safety and efficacy have yet to be determined via clinical trials. Notwithstanding, the aforementioned provide treatment options to individuals suffering from detrimental cancer diagnoses with unmet medical needs.


Section 901 of the Food and Drug Administration Safety Innovations Act (FDASIA) enabled the FDA to approve Lutathera under an accelerated approval application. When Congress passed the FDASIA in 2012, the Federal Food, Drug, and Cosmetic Act (FD&C Act) was amended to allow the FDA to approve certain drugs provided a “drug has an effect on a surrogate or an intermediate clinical endpoint […] The FDA bases its decision on whether to accept the proposed surrogate or intermediate clinical endpoint on the scientific support for that endpoint. Studies that demonstrate a drug’s effect on a surrogate or intermediate clinical endpoint must be ‘adequate and well controlled’ as required by the FD&C Act.”


For Patients – One out of 27,000 people are diagnosed with GEP-NETs per year. The FDA’s approval of Lutathera expands patient access to quality health care by providing treatment in the absence of clinically tested drugs.

For Pharmaceutical Manufacturers & Industry Stakeholders – The approval of Lutathera reveals the criteria for market entry. The following two paragraphs entail language extracted directly from the FDA’s press release:

  • The approval of Lutathera was supported by two studies. The first was a randomized clinical trial in 229 patients with a certain type of advanced somatostatin receptor-positive GEP-NET. Patients in the trial either received Lutathera in combination with the drug octreotide or octreotide alone. The study measured the length of time the tumors did not grow after treatment (progression-free survival). Progression-free survival was longer for patients taking Lutathera with octreotide compared to patients who received octreotide alone. This means the risk of tumor growth or patient death was lower for patients who received Lutathera with octreotide compared to that of patients who received only octreotide.
  • The second study was based on data from 1,214 patients with somatostatin receptor-positive tumors, including GEP-NETS, who received Lutathera at a single site in the Netherlands. Complete or partial tumor shrinkage was reported in 16 percent of a subset of 360 patients with GEP-NETs who were evaluated for response by the FDA. Patients initially enrolled in the study received Lutathera as part of an expanded access program.


Click here for FDA—Expanded Access to Investigational Drugs—guidance updated October 2017.