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Regulatory News | 04 August 2017 | By Zachary Brennan
What do a former prosecutor living in Arizona and unexpectedly diagnosed with neuroendocrine tumors by a specialist at the Mayo Clinic and referred for treatment in Switzerland, a Coloradoan who was also diagnosed and referred to Switzerland for treatment, and a Los Angeles-based lawyer who tried to get into immunotherapy trials but was denied have in common? They all say they obtained treatment from a Houston-based oncology center that has extended their lives thanks to a Texas "Right-to-Try" law.
The six patient testimonies provided to Focus by the libertarian Goldwater Institute, which is pressing for the passage of such laws across the country, all focus on obtaining this same investigational cancer therapy outside of a clinical trial from the former director of therapeutic nuclear medicine at M.D. Anderson Cancer Center in Houston, Texas, named Ebrahim Delpassand. And they offer a brief glimpse into why the institute says it’s pushing for states and the federal government to adopt these Right-to-Try laws.
But the examples and Delpassand’s explanation (see more below on his perspective) of using the Texas law raise questions about how a more widespread uptake of these laws, which skirt around FDA's expanded access program to provide terminally ill patients with sometimes last-minute treatments, will play out and be tracked, especially with the US Senate’s passage of its own Right-to-Try bill Thursday.
Rachel Sachs, an assistant professor of law at Washington University in St. Louis, told Focus: "It’s telling that although 37 states have adopted these laws, when asked to provide examples of success stories, one of the primary groups pushing for their adoption can only provide the testimonies of six patients who received access to experimental medicines through a single physician in a single state."
In a phone interview Friday, Delpassand, founder and medical director of Houston-based Excel Diagnostics & Nuclear Oncology Center, who is the physician at the center of the Goldwater Institute’s push to show how the Right-to-Try laws are taking effect, explained to Focus that he had been treating patients under an FDA-approved IND that began in June 2010, with a treatment that he said has been used in Europe and saw "promising publications related to safety and effectiveness."
In February 2015, after initially enrolling 60 patients under his IND, Delpassand said he requested that FDA increase the number of patients he could enroll to 150, and then later to 250 as more patients sought treatment.
About a month after he requested the first increase, he said FDA requested a telephone conference call "and I said that’s fine and we talked and the group at FDA said that we cannot approve your request and I said why, you know the drug is not yet available," and FDA offered a few reasons, including that his clinical trial was "delaying commercialization," which he said he was surprised to hear.
He said FDA confirmed they had not received any complaints from patients enrolled in his trial and that he did not want to have to tell these patients that they had to go to overseas to be treated.
"I told FDA I don’t want to break any laws, [but this is] very unfair to patients, and now they will have to go to Europe for this treatment," he told Focus. However, in June 2015, the Right-to-Try law passed in Texas and Gov. Greg Abbott immediately made it effective. Delpassand said he resumed treating patients that same month.
He said he continued to use the same protocols under the IND approved by FDA and continued to obtain supplies of the treatment from a Houston-based firm known as Radio Medix, of which Delpassand is chairman and CEO, and a European company.
FDA, meanwhile, told Focus that it's "unable
to disclose proprietary information about investigational drug products" and is "unable to confirm whether or not a sponsor provided an
investigational peptide receptor radionuclide therapy (PRRT) treatment to
oncology patients outside of a clinical trial."
He also sent articles to Focus on the treatment and his trials. One of the articles, of which he is co-author, was received for publication in Clinical Nuclear Medicine in December 2016 (with a revision accepted in January 2017).
The treatment, known as Peptide receptor radionuclide therapy (PRRT) with radiolabeled somatostatin analogues, was evaluated for patients with inoperable, well to moderately differentiated metastatic neuroendocrine tumors (NETs).
The trial enrolled 144 patients (85 men and 59 women; age range, 11–87 years old) and 143 patients received at least one cycle of treatment.
Among them, 132 were deemed evaluable by having at least one cycle of treatment and according to the results section, "Of 28 patients who have completed Lu DOTATATE therapy (completion of 4 or more cycles of treatment and all designated followups), no patient showed complete response (CR), 8 patients (28.57%) showed partial response (PR), 16 patients (57.14%) showed stable disease (SD), and progressive disease (PD) was observed in 4 patients (14.28%)."
In terms of what Delpassand charges patients to be a part of his study under the Right-to-Try law, he said he covers the cost of purchasing the components of the treatment.
He also said he expects the PRRT treatment to be approved in the US for use by the end of this year or the first quarter of next year, noting, "In this kind of situation Right-to-Try comes in handy," though he said he understands FDA "needs to go through its due diligence but this is a cancer therapy and they need the medication."
Delpassand noted that all the patients he treats have exhausted other treatment options and he receives referrals from neuroendocrine tumor specialists from across the US, including from the University of Iowa, Cedars Sinai, Memorial Sloan Kettering Cancer Center and MD Anderson.
In terms of whether he thinks other companies might try to abuse Right-to-Try laws across the US and sell investigational drugs outside clinical trials and FDA’s purview, he said, "I think abuse is possible, but overall we need to respect the physician and patient relationship - no one knows better than the treating physician."
And with the newly passed Right-to-Try bill in the Senate calling for more transparency on how companies and physicians are using these new laws, some say the additional transparency on successes will be welcomed.
Alison Bateman-House of the Division of Medical Ethics at New York University told Focus that she hopes there will be more data and examples of using Right-to-Try laws. She also questioned whether there will be penalties for companies failing to report their use of Right-to-Try laws.
Washington University’s Sachs added: "Until now, these state-level bills have operated in the shadow of more restrictive federal laws, and I suspect these laws have been relatively infrequently used as a result. That may change going forward, if the House passes the Senate’s bill. But if the Senate bill becomes law and these drugs are still infrequently provided, backers of these laws may want to consider whether their ‘right to try’ advocacy has created false hope. These bills provide a right to ask – nothing more."
Tags: Right to Try, Goldwater Institute, Texas Right-to-Try, Delpassand