Flag Real-World Evidence in IND Applications, Guidance Says

May 13, 2019

Sponsors that plan to use real-world evidence in drug development must identify the sources of that evidence when requesting an IND from the FDA.

IND cover letters should indicate whether a sponsor hopes to use real-world evidence to support the safety or efficacy of a drug, to change a drug’s label, or to comply with a postmarketing requirement, the agency says in a draft guidance released last week.

The guidance draws a line between real-world data — information about patients’ health status or healthcare delivery “routinely collected from a variety of sources,” including electronic health records, medical claims or billing data — and clinical evidence for a drug from analysis of real-world data.

Regulators only want to track real-world evidence that relate to specific products or regulatory decisions, so sponsors don’t have to flag natural histories used to establish a clinical outcome test or biomarker, feasibility studies involving real-world evidence or studies that use real-world data “to perform exploratory analyses and generate hypotheses,” the draft guidance states.

Read the draft guidance here:

FDA Releases Draft Guidance on Clinical Lactation
The FDA has published recommendations for drug sponsors planning trials that potentially will enroll breastfeeding women. The aim of the draft guidance issued last week is to protect the health and safety of research subjects and their babies.

The guidance specifies the circumstances that should lead sponsors to study the effects of a new investigational drug on lactation before enrolling women of reproductive age in a clinical trial. It also addresses approved drugs seeking a new indication that may be used by breastfeeding women.

Read the draft guidance here:

Nonprofit Healthcare System Plans Its First Gene Editing Trial
Christiana Care Health System’s Gene Editing Institute has announced plans for its first investigator-initiated trial, which will use genome editing to address late-stage lung cancer.

The nonprofit community medical system plans to use the CRISPR genome editing platform to develop a treatment for K-ras-positive non-small cell lung cancer patients in stages 3 and 4 of the disease.

Christiana’s Gene Editing Institute is collaborating with its Graham Cancer Center and Research Institute on the trial protocol and development of an IND submission to the FDA.

Elligo to Spend $20 Million on Patient-Trial Connection Database
Elligo Health Research last week announced it had raised $20 million to expand its program to connect physicians and their patients with clinical trials.

The Texas-based integrated research organization plans to extend its reach into more states and further develop its IntElligo Research Stack technology, which it uses to search electronic health records for study criteria like therapeutic specialty, patient pool demographics and office location and connect practices with upcoming clinical trial opportunities.

Sensei, AstraZeneca to Collaborate on Study of Combination Therapy
Sensei Biotherapeutics has announced a clinical trial collaboration with AstraZeneca to evaluate the safety, tolerability and preliminary activity of AstraZeneca’s IMFINZI (durvalumab) in combination with Sensei’s first-in-class immunotherapy candidate, SNS-301.

The companies will study the effect of the combination on patients with locally advanced head and neck cancer as well as patients with various locally advanced unresectable or metastatic/recurrent solid tumors.

AstraZeneca will supply IMFINZI and Sensei will sponsor and fund the Phase II studies. AstraZeneca and Sensei each will retain full worldwide rights to their respective molecules.

WuXi AppTec Adds Pharmapace to Clinical Portfolio
Shanghai-based WuXi AppTec will bolster its CRO arm, WuXi Clinical, with the acquisition of biometrics service provider Pharmapace, the company announced last week.

Taking advantage of China’s recent regulatory reforms allowing overseas trial data, WuXi plans to use Pharmapace’s services to expand its global reach and provide drug development solutions across the board.

C3J Therapeutics and AmpliPhi Biosciences form Armata Pharmaceuticals
Armata Pharmaceuticals, Inc., a new clinical-stage biotechnology company, has been created with the close of a merger between C3J Therapeutics, Inc., and AmpliPhi Biosciences Corporation.

The newly created company will concentrate on the development and advancement of phage-based therapeutic candidates to treat antibiotic-resistant infections.