Right to Try vs. Expanded Access: Examining the Regulatory Claims Behind a High-Profile Cancer Survival Story
核心洞察
Natalie Harp, a White House aide, publicly credits the 2018 Right to Try law with saving her life after she failed chemotherapies for a rare Stage 2 bone cancer (搜索) and was denied clinical trial participation.
Medical experts note a key contradiction: Harp says she received an FDA-approved immunotherapy (搜索) drug for an unapproved use, but Right to Try applies only to drugs not approved for any use.
Such off-label use of an approved immunotherapy (搜索) would instead fall under the FDA's expanded access pathway, which has existed since 1987 and predates the Right to Try Act.
A high-profile account of a cancer survivor crediting the federal Right to Try law with saving her life has drawn renewed scrutiny from medical experts, who point to a fundamental regulatory contradiction in the story. Natalie Harp, a 35-year-old aide to U.S. President Donald Trump, has repeatedly stated that she was diagnosed with a rare Stage 2 bone cancer (搜索) and that the Right to Try law, signed by Trump in 2018, allowed her to access an experimental treatment that ultimately saved her life.
Harp first shared her experience in a LinkedIn blog post and a June 2019 Fox News interview, and later in a speech at the Republican National Convention in August 2020. In her accounts, she described failing the chemotherapies available on the market and being denied participation in clinical trials. "When I failed the chemotherapies that were on the market, no one wanted me in their clinical trials," she said. "They didn't give me the right to try experimental treatments, Mr. President. You did, and without you, I'd have died waiting for them to be approved."
The Regulatory Contradiction
The central issue raised by medical experts concerns the nature of the treatment Harp says she received. Harp has stated that she was treated with "an FDA-approved immunotherapy (搜索) drug for an unapproved use." According to the text of the Right to Try law, an eligible patient can consent to the use of an "eligible investigational drug" that has completed a Phase 1 clinical trial and "has not been approved or licensed for any use."
This creates an immediate contradiction. The Right to Try law applies specifically to drugs that have not been approved for any use, yet Harp's claimed lifesaving treatment was an FDA-approved immunotherapy (搜索) drug being used for an off-label, unapproved indication. Such a drug would not fall under the Right to Try framework.
Expanded Access: The Pre-Existing Pathway
Experts note that the use of an approved drug for an unapproved indication would instead be governed by a different, long-standing process: expanded access. Expanded access allows patients who have exhausted all other treatment options for a serious or life-threatening condition to use an investigational drug. Investigational drugs eligible for expanded access have not been approved by the FDA "for their specific use."
Critically, this pathway predates the Right to Try Act by decades. FDA researchers have written that the agency has been facilitating access to investigational drugs since the 1970s, and regulations describing pathways to access these unapproved drugs have existed since 1987.
A comparison by Triage Cancer, a nonprofit focused on practical and legal issues affecting cancer patients, shows that both expanded access and right-to-try require a physician and the drug manufacturer to approve a request to use an unapproved drug. Expanded access additionally requires approval from the Institutional Review Board and the FDA, requirements that right-to-try does not impose.
FDA data indicates that between fiscal years 2019 and 2023, the agency approved between 97.1% and 100% of expanded access requests, depending on the year and type of request.
Plausibility of the Clinical Account
While Harp's story is vague on specifics such as drug names and dates, independent fact-checkers have compared its details against current medical research. Several elements of her account appear plausible.
Bone cancers are rare in general, and there are many different specific kinds. The American Cancer Society even suggests seeking out clinical trials because these cancers are rare and difficult to treat. University College London has written that bone cancers are frequently resistant to chemotherapy, which would be consistent with Harp's claim of failing chemotherapies.
The American Cancer Society notes that "in recent years" researchers have developed non-chemo drugs, including immunotherapy (搜索) drugs, to help treat some bone cancers. The Cancer Research Institute states that while surgery is often the main treatment method for early-stage bone cancers, immunotherapies may be used in treating more advanced or far-reaching forms of the cancer.
The FDA first approved an immune checkpoint inhibitor (搜索), the kind of immunotherapy (搜索) drug often used for treating these cancers, in 2011, according to the Osteosarcoma (搜索) Institute. The institute notes that no new treatment options for osteosarcoma, a type of bone cancer (搜索), have been approved in 40 years.
The rarity of bone cancers can also make it difficult for emerging treatments to be tested with large enough sample sizes in trials to move quickly through the approval process, which could explain why a promising immunotherapy (搜索) might not yet have been approved for a specific bone cancer (搜索) indication.
Unverified Claims
Several aspects of Harp's account remain unverified. Snopes, which examined the claims, stated it "cannot independently verify Harp's account and medical history," and therefore could not rate the claim true or false. The White House did not respond to requests for verifiable details, such as which unapproved drug was used in her treatment.
Harp has also described a medical error preceding her cancer diagnosis, writing that on November 9, 2015, "a nurse mixed up my IV with the deadly substance of sterile water." She has claimed this error is one that "no one has survived." However, a 2008 report from the Pennsylvania Patient Safety Authority documented that bags of sterile water were being mistaken for IV solutions due to similar appearance, and that these mistakes were capable of causing serious harm, sometimes even killing patients. Based on the report's description of a patient who survived the sterile water injection, others have survived the medical error that Harp claimed no one else had survived. The less-extreme version of her claim, describing the error as "notoriously deadly," is accurate.
Notably, Harp's description of events suggests that her physician, and potentially drug manufacturers, would not approve her request for expanded access. Such obstacles would also be an issue under the right-to-try law; based on her story, it appears Harp became able to use an unapproved drug treatment because she obtained a new doctor, not because of the new law.