Human Gene Therapy Guidances Focus on Efficacy in Early-Stage Trials
A suite of FDA final guidances issued last week suggests that early-phase trials of gene therapies to treat rare diseases and other genetic disorders focus on efficacy as well as safety to maximize results from small patient populations.
The guidance on rare disease gene therapies recommends sponsors consider designing their first-in-human studies to have the potential to provide evidence of effectiveness without progressing through phase 2 and 3 trials.
“Alternative trial designs and statistical techniques that maximize data from a small and potentially heterogeneous group of subjects (including genetic heterogeneity) should be considered,” the guidance adds.
The guidance on therapies for retinal disorders recommends early-phase trials focus on dose-ranging and in a third guidance, the FDA says early-phase trials of gene therapies for hemophilia “should not only evaluate safety and feasibility, but also gauge bioactivity and preliminary efficacy.”
Read the guidance on retinal disorders here: https://bit.ly/31cIbmF.
Read the guidance on rare diseases here: https://bit.ly/37YcxeY.
Read the guidance on hemophilia here: https://bit.ly/37HvUsN.
FDA Drug Center Releases 2020 Clinical Trial Guidance Agenda
Several new FDA clinical trial-related guidances are in the works for 2020, according to the Center for Drug Evaluation and Research (CDER) guidance agenda released last week.
CDER announced plans for new or revised guidances on decentralized clinical trials, using multiple endpoints in clinical trials, use of data monitoring committees in controlled trials, digital health technologies for remote data acquisition and postmarketing studies.
Other topics on the list include adjustment for covariates in randomized clinical trials using nonlinear models, civil monetary penalties for failure to meet accelerated postmarketing requirements and frequently asked questions about FDA’s statement of investigator form.
CDER did not specify when it will issue the guidances.
In addition to the FDA, the International Council on Harmonization (ICH) continues to work on a revision of its ICH E8 — General Considerations in Clinical Trials, which it plans to release in June (CenterWatch Weekly, March 11, 2019). ICH also has begun work on revising its guideline for optimizing safety data collection for publication in 2021, as well as a new guideline on adaptive clinical trials, which has not yet been drafted.
Read CDER’s guidance agenda here: https://bit.ly/2OiAPIZ.
Administration Pledges to Expedite Coronavirus Vaccine Trial but NIH Not Optimistic
The White House said last week that it hopes to have a vaccine for the coronavirus (2019-nCoV) ready for an early-stage clinical trial within the next three months, but it could take a year or more before a product is ready.
NIH is working with biotech company Moderna to develop a vaccine candidate, according to Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases.
Too Few Patients Enrolling in Cancer Trials, Study Shows
A study from Penn State Cancer Institute found that of 12 million patients in the National Cancer Database between 2004 and 2015, only 11,600 patients — or 0.1 percent — enrolled in a clinical trial after their cancer diagnosis.
The study also found that a sizeable proportion of patients who enroll in cancer trials as a first form of therapy are white males with private insurance.
Those who enrolled in clinical trials had a median survival rate of 7.5-months longer than those who did not participate, the study found.
To read the study, click here: https://bit.ly/2S8mW15.
CROs and Sponsors Plan to Ramp Up eConsent Use, Survey Shows
A new survey from Signant Health says 71 percent of sponsors and 76 percent of CROs will implement an eConsent solution for most of their studies within the next three years.
Approximately 65 percent of CRO respondents and 85 percent of sponsor respondents say they will adopt eConsent for at least some studies the next 12 months, according to the survey from Signant, a clinical research technology firm.
To read the survey, click here: https://bit.ly/2RLpNOz.
UK’s MHRA Releases Updated Guidance on Clinical Investigation of Medical Devices
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has released an updated guidance for devicemakers, outlining the necessary clinical data needed to gain CE-mark approval.
In this fifth version of the guidance, the MHRA provides information on when a clinical investigation may be required for medical devices. Also presented in the guidance are seven factors manufacturers should consider when deciding whether to conduct a clinical investigation for a nonCE-marked medical device.
The guidance is included in a group of additional documents from the MHRA that discuss compiling a submission, clinical investigation fees and assessment timelines.
India’s Regulatory Body Increases Oversight of Clinical Trials
Following the implementation of its New Drugs and Clinical Trial Rules 2019, India’s regulatory agency has increased the number of trial approvals to 185 from 30 the prior year.
On the inspection front, the Central Drugs Standard Control Organization (CDSCO) in India has inspected 115 biological product and 30 new drug trial sites the past three years.
In 2017, the number of CDSCO inspections of biological product trials was 16. This number increased to 53 inspections by 2018. Through October 2019, CDSCO had inspected 47 biological product trials.
There were only five CDSCO inspections of new drug trials in 2017, compared with 21 in 2018. Only two inspections of new drug trial sites were reported in 2019.
Researchers Introduce the World of Clinical Trials in New Children’s Book
Elementary and middle school children can learn about clinical trials at their own level with a new book from a team of University of Buffalo professors and researchers.
“Sofia Learns About Research” is an activity book that describes the journey of a child with asthma considering participating in a clinical trial. Teresa Quattrin, professor of pediatrics and senior associate dean of research integration, co-authored the book with another researcher and an undergraduate student at the university to make the topic of clinical research accessible to youth and their parents across cultures, learning and literacy levels.
To learn more about the book, click here: https://bit.ly/2Ueetw3.