Gottlieb, FDA Execs Urge Drug Sponsors to Expand Access to Treatment After Trials

April 1, 2019

FDA Commissioner Scott Gottlieb and two of his top aides are urging drug sponsors to continue treatment for especially needy clinical trial patients after their studies end and before the new treatment is officially approved.

Gottlieb, CDER Director Janet Woodcock and CBER Director Peter Marks say the agency’s expanded access program has offered a lifeline to more than 13,000 patients who otherwise might be without hope.

“Although we often focus on EA as providing access to patients who cannot enter clinical trials, EA can also be considered as a mechanism for those who have participated in a trial in order to allow them to continue receiving a drug that may have provided benefit,” the trio says. “We would generally support such efforts,” including in the case of trials that have shown benefit for only a few individual patients.

Gottlieb, Woodcock and Marks point out that offering patients expanded access “rarely impacts development timelines” and it’s “very rare” to see expanded access cause problems for a drug’s development. A review of more than 300 FDA regulatory decisions between 2010 and 2016 showed no negative impact of offering expanded access on any application.

In addition, providing expanded access beyond a trial also may provide additional data to support an application, they say.

“We support efforts by sponsors to offer promising medicines to patients through EA. This includes continued access after the completion of a clinical trial, sometimes through an extension study,” the trio say. “While EA may not be feasible in every situation, concerns about regulatory risks are often overstated. At the same time, information from EA has sometimes proven valuable, contributing to the overall data available about a drug that is included in the marketing application.”

Trials Industry Living in Conditional Tense
Clinical trial sites and sponsors in the UK are stuck in a kind of regulatory purgatory as the British parliament tries to come up with a plan to break with the European Union.

The uncertainty has resulted in a 25 percent decline in the number of trials since UK voters narrowly approved the split in 2016.

To provide guidance in the absence of clear rules, the Medicines and Healthcare products Regulatory Agency (MHRA) has issued what it calls a “conditional plan” for continuing clinical research and development activities should the UK leave the EU without a new deal in place.

The MHRA plan would extend the use of current EU criteria for the approval of drugs or other treatments for unmet needs. Such conditional approvals would be good for a year but could be renewed under new UK rules after that, MHRA says.

UK regulators also would extend the EU’s rules for exceptional approvals but are “likely to impose specific obligations on the holder of a marketing authorization that is approved under exceptional circumstances,” MHRA says.

Agios Wins Breakthrough Status for Rare Blood Cancer Treatment
Cambridge, Mass.-based biotech firm Agios Pharmaceuticals was granted a breakthrough designation by the FDA for its rare blood cancer treatment Tibsovo (ivosidenib).

The FDA awarded the designation for Tibsovo in combination with azacytidine to treat patients 75 years or older who have been newly diagnosed with acute myeloid leukemia.

Agios submitted data from a Phase I/II study that showed 13 out of the 23 patients in the trial had a complete response to the combination treatment.

FDA Denies Recro Pharma’s Intravenous Meloxicam a Second Time
The FDA has once again put the brakes on Recro Pharma’s efforts to bring its painkiller, meloxicam, to market, questioning its ability to meet patient and prescriber needs after analyzing clinical trial data.

In a complete response letter, the agency took issue with the non-opioid drug’s delayed onset, which they said failed to meet expectations for IV drugs. The agency also had concerns regarding the drug’s role as a monotherapy in acute pain.

The FDA issued Recro a CRL last year, noting that the drug’s analgesic effect did not meet the agency’s expectations. The drugmaker purchased the global rights for intravenous meloxicam from Alkermes in 2015 for $170 million.

Javara Uses Uber Technology to Drive Trial Enrollment
Integrated clinical research organization Javara has partnered with Uber Health, the ride-share company’s HIPAA-compliant technology solution, to transport patients to clinical trial sites.

Uber Health’s web-based dashboard allows hospitals and other healthcare professionals to request, manage and pay for rides for their clinical trial participants.

Patients participating in clinical trials with Javara’s healthcare partners will be able to receive prepaid transportation to study visits coordinated by Javara’s Clinical Trial Navigators. They will have the ability to schedule a ride up to 30 days in advance without the need for a smart phone or the Uber app. The goal is to encourage enrollment, decrease the number of missed appointments and increase the number of on-time visits.

ALS Biomarker Study to Begin This Spring
In an effort to identify biomarkers for ALS therapy trials, a drugmaker-hospital partnership will initiate a nationwide study this spring to assess the effect of the antioxidant drug edaravone on people with early-stage amyotrophic lateral sclerosis.

Mitsubishi Tanabe Pharma America (MTPA) will collaborate with the Massachusetts General Hospital Neurological Clinical Research Institute on a 200-patient, 40-site study they hope will identify quantifiable biological effects of the treatment that can be used as indicators of efficacy in future ALS clinical trials.

Sold under the brand names Radicava and Radicut, edaravone was approved by the FDA for ALS treatment in 2017.

The first biomarker study subject is slated to be enrolled in late spring, with early interim analyses planned for later in the year.

AI Platforms Provide Boost to Clinical Trials from Development to Enrollment
Two new efforts to use artificial intelligence in clinical trials show the range of applications the technology can have.

Bristol-Myers Squibb will use Concerto HealthAI to infuse real-world evidence from medical practices across the U.S. into Bristol’s research, while Health Quest, a four-hospital health system, has partnered with IBM to use its Watson technology to match patients to clinical trials throughout the region its hospitals serve.

Concerto uses data from CancerLinQ, a national repository of information on cancer patients, to search for patterns that may inform drug development, including identifying patients who are not well-served by existing drugs and helping design studies to develop novel therapeutics approaches.

Health Quest will use Watson to compare enrollment criteria with medical data on its patients in the Hudson River Valley and northern Connecticut and match patients with trials. The AI platform has proven successful for other healthcare organizations, including the Mayo Clinic, which in 2018 experienced an 80 percent increase in clinical trial enrollment.

Biotech Startup Raises a New Moon Over Oncology Research
A new immunotherapy startup has grown from the ashes of the formerly independent biotech firm Potenza Therapeutics.

Potenza was acquired last year by Japanese drugmaker Astellas, but much of its former leadership has stayed behind to launch a new venture, inexplicably named Werewolf Therapeutics.

Werewolf, which was founded in 2017 by healthcare investment firm MPM Capital, will take over Potenza’s former lab space in Cambridge, Mass.