March 25, 2019
WIRB-Copernicus Group (WCG) is inviting clinical researchers to apply for membership in its Global Research Network (GRN), a free service that offers networking opportunities, trial insights and sponsor connections.
Members of the network will have access to WCG’s broad collection of clinical trial metrics and analytical data to help them compare their sites’ performance to the rest of the industry. GRN also allows members to participate in WCG webinars, thought leadership events and roundtable discussions.
The network also supplies sponsors and CROs with information on its members’ capabilities and clinical research interests, giving members access to more industry-sponsored trials. “We aim to make sites more attractive and effective in study conduct,” says Jonathan Zung, WCG executive vice president.
WCG also recently announced its SiteReady suite, an end-to-end solution to help institutions and independent sites improve research efficiency and profitability.
Sign up for GRN here: https://bit.ly/2HytbbT.
FDA Issues Guidances on Pediatric HIV Treatments, Prevention
The FDA last week issued two final guidance documents that the agency hopes will help drug sponsors develop more effective HIV prevention medicines and HIV treatments for children.
The agency urges drug sponsors to include adolescents — defined as children between 12 and 17 years old — in Phase III adult trials. Sponsors should begin pediatric formulas as soon as Phase II trials have helped to zero in on the adult dose.
In a separate guidance on HIV prevention, the FDA recommends that trial sponsors include healthy, infection-free adults who are “at substantial risk” of becoming infected.
Generally, the agency looks for “two adequate and well-controlled trials” to demonstrate a treatment’s effectiveness. But a single Phase III trial might prove useful “if the results are robust with internal consistency, clinical and statistically persuasive and supported by additional evidence.”
Read the pediatric HIV infection guidance here: https://bit.ly/2TY3gQr.
Read the HIV pre-exposure prevention guidance here: https://bit.ly/2WcCXUk.
Natural Histories Can Help in Designing Rare Disease Trials, FDA Says
A new FDA draft guidance encourages sponsors of rare disease trials to use natural history studies — observational studies that track the course of a disease — when developing rare disease trials.
The agency has promised to make more room for such real-world evidence studies in the hopes of speeding up drug development for rare diseases.
According to the draft guidance released last week, natural histories can help zero in on patient populations, figure out ways to measure clinical outcomes, identify or develop biomarkers and even design externally controlled studies.
“Information obtained from a natural history study can play an important role at every stage of drug development,” said FDA Commissioner Scott Gottlieb in a statement, “from drug discovery to the design of clinical studies intended to support a drug’s marketing approval.”
Natural histories “should have well-defined, carefully documented protocols” that include:
- Study duration, including date of inception and date of cutoff;
- Descriptions for how data will be collected;
- Definitions of diseases and diagnostic criteria that will be included in the study;
- A list of demographic information to be collected;
- Disease-related information, such as signs and symptoms, age at onset, diagnoses, the development of “important morbidities” and death; and
- Any regional treatment guidelines or algorithms.
Read the draft guidance here: https://bit.ly/2FpeILz.
FDA Reaffirms Flexibility on GCP Waiver Requests for Device Trials
The FDA last week amplified its commitment to exercising discretion when devicemakers seek waivers for new GCP requirements for device trials.
A final rule on human subject protection, which took effect Feb. 21, requires that data from medical device trials, including foreign trials, be gathered in accordance with GCPs. GCPs call for review and approval from an independent ethics committee and well-documented informed consent.
In a March 19 webinar on implementation of the final rule, the agency said it may grant waivers in cases where informed consent is difficult or impossible to obtain, such as for research on leftover human specimens and where sharing subject medical data is prohibited by local laws.
In the case of foreign clinical trials, the FDA will consider waiver requests that include:
- A statement that the trial was or will be conducted in accordance with GCP; and
- A description of the actions the sponsor or applicant took to ensure credible, accurate data and to ensure human subjects are adequately protected.
The agency issued a guidance along with the final rule outlining how to meet the agency’s requirements, request waivers and provide the required information to support clinical data submissions.
Read the guidance here: https://bit.ly/2TgsXaJ.
Read the final rule here: https://bit.ly/2Fpvlbq.
FDA Orders AbbVie to Stop Recruiting in Myeloma Trial After Deaths Spike
The FDA has ordered AbbVie to stop recruiting multiple myeloma patients for a Phase III trial of its anti-cancer drug Venclexta/Venclyxto, the company announced last week.
Forty-one out of 194 patients taking AbbVie’s Venclexta (venetoclax) in the company’s trial have died, the FDA found, nearly twice the percent of the patients in the trial’s placebo arm.
Venclexta already is approved for chronic lymphocytic leukemia and acute myeloid leukemia but the halt doesn’t affect any patients outside the multiple myeloma experiments, and AbbVie said it “remains confident in the benefit/risk profile” of the drug.
Patients in the Venclexta arm have met the primary endpoint with a progressive-free survival rate of 22.4 months, on average, AbbVie says. Patients in the trial who are benefiting from the Venclexta treatments have the option to stay on the medicine, AbbVie says, while the company works with regulators to address the problems.
Biogen Halts Study of Alzheimer’s Drug
Biogen and its pharmaceutical partner Eisai have announced they are halting two Phase III clinical trials of aducanumab, a drug designed to slow Alzheimer’s disease by targeting the brain-destroying protein fragments known as beta-amyloids.
Interim analysis conducted by an independent monitoring committee found that aducanumab was unlikely to benefit Alzheimer’s patients when compared to placebo. Biogen and Eisai enrolled patients with very early signs of Alzheimer’s disease to test whether aducanumab would slow the progression of brain deterioration, but results were not positive.
The failure of aducanumab is particularly discouraging as the beta-amyloid hypothesis was once the most well-accepted theory on how Alzheimer’s disease destroys the brain.
Microsoft “Bot” Matches Patients with Trials
Tech giant Microsoft is getting into the clinical trials industry, marketing a “bot” that uses AI to improve trial recruitment.
The Clinical Trials Bot — part of a suite of new healthcare-related services the company announced in February — has the capability to search for clinical trial studies as requested by either patients or clinicians for a specific disease in the hope that it will ease the process for patients to find trials that could provide otherwise unavailable medicines and therapies. Machine reading, a form of AI, is used to read and upload selection criteria for each clinical trial. Data mined is then used to determine which patients are a suitable match for the disease trial requested.
Once studies have been identified, patients are required to answer text questions to refine the list of suitable trials. The bot then suggests links to those trials that best meet patients’ needs. The bot can also be used for drugmakers to connect to test subjects.
Microsoft will not be releasing or commercializing the bot at this time, although the company is in talks with pharmaceutical companies interested in adopting the technology.
Investor-University Partnership Focuses on Novel Therapies
Healthcare investment fund Deerfield Management has committed $100 million to a new R&D partnership with Harvard University, the New York-based company announced.
The new company, called Lab1636, will be based in Cambridge, Mass., and will be headed up by Harvard’s Office of Technology Development. Organizers say they will focus on the early-stage pipeline for novel therapies in both pre-clinical and potentially even clinical trials.
The projects that will receive funding through Lab1636 will be selected by a joint advisory committee and projects “will generally focus on the development of novel therapeutics, ideally advancing many to a stage that would enable the filing of an IND application and, if successful, the commencement of clinical trials in patients,” Deerfield officials said in a news release.
AstraZeneca to Share New Cancer Research at AACR Annual Meeting
AstraZeneca will showcase the R&D progress it has made on its oncology pipeline and portfolio at the annual American Association for Cancer Research (AACR) meeting this week, the drugmaker announced.
Sharing 84 data presentations in total, the company will present headway it has made on its oncology pipeline, including 28 abstracts on new immuno-oncology data. AZ said that 33 of the presentations will focus on complementary biological pathways exploring the DNA damage response (DDR) mechanism, while 20 others will detail the company’s progress on tumor drivers and resistance mechanisms.
José Baselga, AZ’s executive vice president of research and development, oncology, said that the company also will unveil 28 new molecular entities and six combinations.