News & Comment

Clinical trial data transparency, an IPO surge and more made the news last year.

Regulators and health technology assessment bodies discussed early experiences with pilot programmes in which they work together to provide drug developers with joint advice on the design of clinical trials.

The expedited US regulatory pathway for 'breakthrough therapies' has already yielded its first two approvals and more than 26 designations, for 30 candidates in 22 indications.

Japan's biggest pharmaceutical companies, the Bill and Melinda Gates Foundation, and the Japanese government have awarded their first round of global health research grants.

If Duchenne muscular dystrophy drugs that modulate gene splicing can overcome key hurdles, they could boost enthusiasm for related antisense oligonucleotides in other indications.

A Phase III trial for a first-in-class psoriasis drug provides encouraging data, but the emerging agents still have to prove their worth in a crowded autoimmune market.

A series of setbacks almost caused the demise of PARP inhibitors, but four companies are now beginning pivotal trials of these agents in breast and ovarian cancer.

First meetings see regulators and patient advocates start working to incorporate the perspective of patients into benefit–risk assessments.

The approval of Boehringer Ingelheim's anticancer drug afatinib highlights the growing enthusiasm for once-shunned irreversible kinase inhibitors.

Stem cell technology and computational modelling offer the promise of reducing the current burden of cardiotoxicity assessment.

Merck's dual orexin receptor antagonist suvorexant is approaching the regulatory finish line, 15 years after orexins were discovered.

Immunotherapies that target the PD1–PDL1 axis continue to generate solid clinical data, and fresh combination trial results offer tantalizing hints of greater effectiveness to come.

Takeda has filed for European approval of vedolizumab in inflammatory bowel disease, potentially providing new hope for spurned integrin inhibitors.

Gilead has filed the first interferon-free hepatitis C virus regimen for approval in the United States, but drug developers are making strides with better combinations.

Driven by recent clinical breakthroughs and technological progress, 30 antibody–drug conjugates against over 24 targets are now in trials for blood cancers and solid tumours.

Disappointing developments with Amgen's ganitumab and Bristol-Myers Squibb's BMS-754807 highlight the need for rational combinations and predictive biomarkers to rescue IGF1R pathway antagonists.

CGRP makes a comeback as antibodies advance into Phase II trials.

Molecular imaging is already engrained in early-stage trials for central nervous system disorders, but used infrequently in other therapeutic areas. What will it take to make it standard practice across the pipeline?