First patient-focused drug development guidance released

First patient-focused drug development guidance released | FasterCures, Children's Tumor Foundation, and CureSearch collaborate to accelerate available treatments for children with cancer | Gene therapy for SCD showing real-world promise

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June 23, 2020

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First patient-focused drug development guidance released

The FDA released its first patient-focused drug-development guidance that offers information on what constitutes patient experience data as well as general considerations when collecting such data. The guidance is the first in a series of four that aims "to address, in a stepwise manner, how stakeholders (patients, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making."

Full Story: Regulatory Focus (6/18)

News from FasterCures

FasterCures, Children's Tumor Foundation, and CureSearch collaborate to accelerate available treatments for children with cancer

FasterCures is proud to announce its partnership with the Children's Tumor Foundation and CureSearch for its pilot BRIDGE project. FasterCures developed the BRIDGE initiative in 2019, intending to facilitate a more dynamic marketplace for biomedical innovation. BRIDGE's first project will focus on the process of matchmaking discontinued drug assets with partners that have the expertise and capital needed to develop those medicines. FasterCures, the Children's Tumor Foundation, and CureSearch will take the essential steps to build partnerships to develop discontinued drugs to treat pediatric tumors. Read more about the BRIDGE pilot project.

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Patient Engagement

Gene therapy for SCD showing real-world promise

The first person in the US to be treated for sickle cell disease with a CRISPR-based gene therapy is approaching the one-year anniversary of her treatment, which appears to be effective and durable. The treatment also appears effective in two patients with beta thalassemia, researchers say.

Full Story: National Public Radio (6/23)

Commentary: No need for coronavirus challenge trial

More than 25,000 people have volunteered with an online recruiter for SARS-CoV-2 challenge trials meant to accelerate approval of a vaccine, but too little is known about the virus to fully inform volunteers of the risks posed and no cure is available, writes Michael Rosenblatt, chief medical officer at Flagship Pioneering and an advisor to Moderna. Any missteps in a human challenge trial could significantly undermine public trust and delay development of an effective vaccine, and a conventional study may be able to be conducted just as quickly, Rosenblatt writes.

Full Story: STAT (tiered subscription model) (6/23)

Science and Technology

All of Us study collecting COVID-19 data

Researchers working with the All of Us precision medicine study will test the most recently collected blood samples for SARS-CoV-2 antibodies, starting with samples collected in March and working through previously collected samples until none test positive. Participants are being asked to answer an online questionnaire regarding the pandemic's effect on their physical and mental health, and researchers will also collect and study data from participants' EHRs to find patterns and determine the effects of various treatments.

Full Story: Health IT Analytics (6/17)

Companies rush to develop COVID-19 vaccines, antibodies

Scientists are developing vaccines and treatments for COVID-19 at unprecedented speed, but FasterCures Executive Director Esther Krofah warns that optimism must be tempered with caution. Some groups are working on monoclonal antibodies specifically for the novel coronavirus, and they could serve as a bridge until a vaccine is approved, Krofah said.

Full Story: Chemical & Engineering News (tiered subscription model) (6/19)

Online network diversifies COVID-19 research

Computational epidemiologist Maimuna Majumder had little trouble adjusting to working from home, but she noticed other scientists weren't as lucky, so on March 18 she sent a tweet seeking qualified volunteers to help answer research questions at the intersection of computing and COVID-19 epidemiology. Thus was born the COVID-19 Dispersed Volunteer Research Network, and Majumder says the group's "cross-institutional, almost cross-cultural, work is very much at odds with academia's usual way of doing things."

Full Story: Wired (tiered subscription model) (6/18)

NIH establishes national repository for COVID-19 data

The NIH's National Center for Advancing Translational Sciences unveiled the National COVID Cohort Collaborative to aggregate and standardize clinical, diagnostic and laboratory data to advance research and treatment decisions. NCATS Director Christopher Austin expects the platform to "serve as the foundation for addressing future public health emergencies."

Full Story: Nextgov (6/16)

Finance and Economics

NCI to seek supplemental funding from Congress

The National Cancer Institute will be hard-pressed to fund new clinical trials after studies idled during the coronavirus pandemic resume, unless Congress appropriates additional funds, says NCI Director Ned Sharpless. The NCI's budget for training is also strained, as researchers whose projects have been sidelined will need extra time to complete them, and Sharpless says NCI staff are generating estimates for Congress.

Full Story: Bloomberg Law (free content) (6/22)

Lack of coordination hobbles coronavirus antibody testing

Only a small percentage of the $35 billion allocated by the federal government for COVID-19 testing has been designated for antibody testing, scientists in the US working antibody tests have had to rely on luck and ingenuity. "It would be better to have a unified information sharing system, a unified testing strategy, and the resources to execute it everywhere in the country," said Joe DeRisi, co-president of the Chan Zuckerberg Biohub.

Full Story: Reuters (6/16)

Policy and Regulation

FDA collaborates with EMA to spur COVID-19 research

The FDA, European Medicines Association and European Commission officials agreed on priorities for collaboration and discussed challenges to developing and distributing a COVID-19 vaccine. Agency officials have been in regular contact with one another under the umbrella of the International Coalition of Medicines Regulatory Authorities to reduce regulatory barriers and facilitate collaboration on COVID-19 research, including using observational data to build real-world evidence.

Full Story: Regulatory Focus (6/22)

FasterCures, a center of the Milken Institute, is driven by a singular goal: to save lives by speeding up and improving the medical research system. The Milken Institute is a nonprofit, non-partisan think tank determined to increase global prosperity by advancing collaborative solutions that widen access to capital, create jobs, and improve health.