Richard Garr, Beacon of Hope CRO, founder, told us the company was created to establish the infrastructure to facilitate Right to Try (RTT) treatment programs, “for companies and patients to expand access, at scale, to investigational drugs and significantly accelerate the drug development timeline for life threatening diseases.”
The Boca Raton, FL-based company is currently partnering with other contract research organizations (CROs), providing advice about how to treat screen fails in ongoing randomized clinical trials. It also is designing and recruiting patients for ‘pure’ RTT treatment programs and working with customers to determine if and where RTT can be used.
“Right to Try is an FDA-approved expanded access program,” said Garr. “It’s an alternative expanded access pathway – It’s just cheaper, quicker, and a better way to get to real world evidence, real world data.”
The CRO, which launched last month at the ALS Association Florida Chapter Hope and Help Symposium, is the only CRO exclusively dedicated to RTT programs, Garr said, and is working directly with sponsor companies and in parallel with traditional clinical CROs.
“CROs are typically followers, not leaders; whatever the clients wants is what they want. But clients are telling them now 'we need a way to get more data cheaper and quicker,'” he said. “And this is going to end up, I think, being the answer for how they are going to do it.”
The mandate
Signed into law last year – after much debate – the RTT bill amended the Federal Food, Drug, and Cosmetic Act to enable terminally ill patients access to ‘certain unapproved, investigational drugs.’ To be eligible, patients must have ‘exhausted approved treatment options’ and be unable to participate in a clinical trial involving the drugs.
Speaking to initial concerns from industry stakeholders, among which included the Association of Clinical Research Organizations (ACRO), Garr said the law has been “vastly misunderstood as a result of the political fight engendered by the early history of the law.”
According to Garr, opponents to the bill weren’t attacking the heart of the legislation, which he said was to create incentives for companies to participate in RTT programs.
“There was a perception when this was being run through the states – that this was an ‘anti-FDA’ proposal … I think that’s why, and it’s not an unreasonable reaction, that all of those groups came out against it,” he explained.
Garr said the bill was amended to address these concerns and incorporate further safety measures. At both a federal and state level, the law also requires that patients be, in some way, ‘trial challenged.’ “This can be either by inclusion/exclusion criteria, or by the reality of not having meaningful access to a trial for the relevant drug for their indication,” he said.
“The final product has created an incredibly cost- and time-efficient method for safe, ethically-sourced patient data that can greatly enhance the development process, while fulfilling the law’s mandate to provide a meaningful increase in access to investigational drugs for patients with life-threatening diseases.”
As Garr sees it, the benefits to all involved are ‘so great’ that he believes such treatment programs will ultimately become a standard component of the development process.
'Time is money'
There are several reasons to include RTT programs in the drug development process – and more will become apparent as the market matures, according to Garr.
Among these are the ability to treat screen fails, he said, noting that many inclusion/exclusion criteria are not safety-related, but instead an attempt “to optimize the trial group to eliminate as many variables as possible, even though these patients will ultimately be the ‘intend to treat’ population.”
In one example, Garr said the company is working with a client which is losing 60% of its clinical trial recruits. While these patients are otherwise eligible, they have been using various CBD products to self-treat, making them ineligible to participate in the study.
“Consequently, the recruiting is going to take literally twice as long as it otherwise would and be twice as expensive – time is money in a drug development budget,” he said.
However, by treating these screen fails under RTT, sponsors are able to determine the differences, if any, between the patients who met the criteria and those who did not.
Said Garr, “The sponsor can then decide to let those people, or not, into the trial going forward, potentially saving a great deal of time and money in the next trial, or even the current one.”
“Even if the sponsor doesn’t change the inclusion/exclusion criteria, it can use this data in reimbursement discussions and possibly eliminate the need for a Phase IV trial to broaden the label,” he added, “again saving a significant amount of time and money.”
‘Unmatched’ flexibility
Sponsors also would look to an RTT treatment program for the ‘unmatched flexibility,’ said Garr, who has seen companies developing cancer therapeutics suggest using RTT for a basket trial of sorts. This helps determine which combination trials would be best, he said.
“The relative ease, and significant cost- and time-savings of doing those adaptive/observatory trials under the RTT umbrella, is significant,” added Garr.
Additionally, sponsors retain control of the data, which is only shared with the FDA at the request of the sponsor.
“If the same group of trials were done under the FDA’s various observatory control arms, the FDA could use any data, from any group, to stop the sponsor’s core trial,” explained Garr. “This, plus the additional cost, would clearly dampen the enthusiasm of a sponsor to expand the possible treatment populations they are investigating.”
A new class of milestones
Dubbed the ‘valley of death’ by investors, the period during which a company is waiting for clinical evidence that a drug is working can be years long.
As Garr explained, the sponsor controls the data during a RTT program and can share with the FDA and publish it if they so choose – enabling them to create ‘interim clinical milestones,’ which he said can be used to help fund companies through this ‘valley of death.’
“Might investors ‘discount’ RTT data,” Garr asked rhetorically, noting that it’s possible, though the value of data is always dependent on quality. “In any event,” he said, “it creates an entirely new class of milestones for an industry in sore need of them.”
Added Garr, “Companies want to know how their drugs are working in patients sooner, without compromising their randomized clinical trials. RTT allows for that. Investors want to know the same things. RTT allows for that.”
