First Right-to-Try CRO Launches

Florida-based Beacon of Hope is the first CRO to focus solely on right-to-try activities.

Launched this month, Beacon of Hope will help companies provide safe, ethical and legal patient access to their investigational treatments outside of traditional clinical trials. The CRO also will work with patients, support advocacy groups and educate the community on right-to-try issues.

The Beacon of Hope Patient Database will help match patients with sponsors conducting clinical trials on ALS and other life-threatening diseases.

The privately held CRO is headed by CEO Richard Garr.

Biogen Halts Another Trial Over Safety Concerns

Biogen continued its run of bad luck this week, halting another drug trial over safety concerns.

The termination of its human monoclonal antibody trial of BG00011 comes just days after the company closed three of its Alzheimer’s disease trials last week.

The trial of the drug to treat idiopathic pulmonary fibrosis was in phase 2 and was halted because it did not outperform the placebo arm.

New COA Development Projects in the Works

Three universities and one technology partnership are working on separate projects to increase the availability of clinical outcome assessments (COA).

The FDA has awarded a $4 million grant to the Albert Einstein College of Medicine, Duke University and Northwestern University to conduct projects on using patient input to inform selection of clinical outcomes.

The Einstein College of Medicine and Vector Pyschometric Group will use their $1.29 million to standardize a set of endpoints and related COAs for migraine clinical trials.

Duke University will use their $1.4 million grant to identify COAs and endpoints for use in developing acute pain therapeutics in infants and children under two years old.

Northwestern will use $1.4 million to develop COAs across various chronic conditions that assess physical function severity.

In the corporate sector, tech company Medidata is partnering with Icon’s Mapi Research Trust (MRT) to add MRT’s library of COAs to a new collection of electronic questionnaires that will save trials the time required to create their own COAs.

The company estimates its Medidata Rave eCOA will help trials cut startup timelines by 50 to 60 percent by providing ready-made COAs in multiple languages.

The nonprofit MRT provides patient-reported outcomes data and has assembled a library of more than 470 eCOA forms and 7,000 translations representing 190 instrument developers.

Developer agreements in the database will be pre-approved so sponsors don’t have to seek approval to use a COA for each individual study.

Public Trust in Pharma at an All-Time Low, Says Gallup Poll

Americans feel that pharma is the least trustworthy industry, according to a new poll.

Pharma came in dead last on the 2019 Gallup Work and Education poll, which asks respondents to rate the trustworthiness of 25 business sectors. Even government, the traditional holder of last place, scored higher.

According to the report, Americans are more than twice as likely to rank pharma negatively than positively, with 58 percent expressing negative opinions and only 27 percent viewing the industry in a positive light. Fifteen percent of respondents had no opinion.

The new low accompanies public criticism of an industry that generates the highest-cost drugs in the world and has spent large amounts of money on lobbying in the wake of the U.S. opioid crisis.

Few industries have ever received as low a score in the poll, including the federal government, which spent the first month of 2019 shut down.

Gottlieb Joins Aetion Board to Advance RWE Platform

Former FDA Commissioner Scott Gottlieb will advise healthcare startup Aetion on developing its tool to assess treatment safety, effectiveness and value of using real-world data.

The Aetion Evidence Platform is part of the RCT Duplicate project, a partnership with the FDA and Brigham and Women’s Hospital that will use real-world data to replicate the results of 30 completed randomized clinical trials and predict the results of seven ongoing Phase 4 trials (CenterWatch Weekly, April 15, 2019).

Sindh Healthcare Starts IRB in Pakistan

The Sindh Healthcare Commission of Pakistan announced the formation of its first IRB to review clinical research and promote responsible ethics in life sciences.

The new IRB is welcoming research projects and investigators willing to initiate research in the region and is setting up five district offices and a divisional office.

The move is in response to the commission’s closure of 2,148 non-compliant clinics, including 28 in one week.