Trump Right to Try Legislation Could Boost Orphan Drug Markets

Press Release
Jun 10, 2018
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Rockville (MD), June 10, 2018 — Market Research firm Kalorama Information said that recently signed legislation to give terminally ill patients the right to use experimental medications could boost the market for orphan drugs – pharmaceutical treatments for diseases which there are no known therapies. The legislation authorizes doctors to prescribe drugs that have cleared first phase but have not been through human clinical trials and thus not yet been approved by the Food and Drug Administration. The New York City-based healthcare research estimated the market for orphan drugs to be over 90 billion dollars in a research study on the topic. The firm has also studied monoclonal antibody markets, which are a drug category possibly affected by the change.

"Right to try is a powerful concept and we envision a long-term effect on FDA regulation and more patient focus, but this particular legislation would serve more to codify existing programs," said Kalorama Publisher Bruce Carlson. "There's already provisions for a kind of right to try in the FDA framework and there’s no compellation in the legislation that adversely affects pharmacos. Still, we think it is one more step along with the FDA program to encourage the industry to develop treatments that may not financially survive a clinical trial process at this time.”

Kalorama notes that the FDA "compassionate use" program, performs essentially the same function as the right to try legislation, allowing patients to try experimental drugs after approval from a review board and panel of experts. News sources indicate the FDA has approved 5,816 of 5,849 requests -- a 99.4 percent approval rate. Still the firm thinks the legislation addresses patient demand for attempts at experimental treatments. And although the trial candidate pool is smaller than a clinical trial, such uses could always lead to discovery of successful treatments.

“We’ve considered high patient demand in all our studies of the market,” said Carlson. “We’ve observed the trend of patients traveling abroad to obtain experimental treatments. While in an orphan drug setting, there is a very small pool of trial candidates available, a successful treatment could emerge from these uses.”

The legislation has come under fire for possibly increasingly litigation over negative outcomes from treatments. Kalorama did not think the legislation was as risky as some critics suggested.

“Our analysts are seeing the same risk for litigation as clinical trials and in some ways, could see developers be more upfront and transparent about the risks of the experimental drug which is something that is among the leading reasons for litigation in clinical trials,” Carlson said.

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Kalorama Information, a division of, supplies the latest in independent medical market research in diagnostics, biotech, pharmaceuticals, medical devices and healthcare; as well as a full range of custom research services. Reports can be purchased through Kalorama’s website and are also available on and

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