Biogen Resurrects Alzheimer’s Drug for FDA Approval

Biogen has decided to renew its bid for FDA approval in early 2020 of its investigational anti-amyloid Alzheimer’s drug aducanumab after halting clinical trials earlier this year.

Following discussions with the FDA about using data gathered after the trial ended, the company has reassessed its phase 3 trials of aducanumab, concluding that the longer-term exposure to the drug in one trial succeeded in reducing decline in Alzheimer’s patients.

Biogen’s EMERGE and ENGAGE trials were initiated in 2015 with the same dosage, but data from the ENGAGE trial assessed after only 18 months indicated negative effect compared to placebo and led Biogen to halt the study (CenterWatch Weekly, April 29, 2019).

The new, larger dataset provided longer-term results and showed a statistically significant reduction of clinical decline in EMERGE, Biogen says, and a small subset of ENGAGE results would have followed suit.

For more information on Biogen’s decision, click here: https://bit.ly/2Jc0y36.

FDA Clarifies Its Approach to Postmarket Studies and Clinical Trials

In a draft revision of an April 2011 guidance, the FDA has laid out its current thinking on when the agency may require postmarket studies or clinical trials for approved prescription drugs.

The draft adds new information on when the FDA’s Adverse Event Reporting System (FAERS) and Vaccine Adverse Event Reporting System (VAERS) may be sufficient for identifying and assessing new serious adverse drug events that “occur rarely and are closely linked in time to the initiation of the drug and for which the background rate of events is low,” the draft says.

Certain questions related to serious risk “may only be answerable through specific types of studies or clinical trials,” the agency says. For example, animal studies or clinical pharmacokinetic and pharmacodynamic trials may be the only way to determine if a drug is carcinogenic or could potentially interact with other treatments.

The agency uses the term postmarketing requirement (PMR) to cover all required postmarket studies or clinical trials. PMR’s aim to “more fully characterize a serious risk, if one exists.”

The agency will review data and information from a PMR to “assess its effect on the benefit-risk profile of the drug,” which may result in labeling changes.

The Substance Use-Disorder Prevention that Promotes Opioid Recovery and Treatment for Patients and Communities Act (SUPPORT Act) of 2018 required the agency to issue new guidance on when it may call for postmarket studies or trials.

Read the revised draft guidance here: https://bit.ly/345TYDD.

Innovation Needed to Cure Rare Diseases, Says Sharpless

Sponsors need to be innovative in their approach to developing therapies for rare diseases, Acting FDA Commissioner Ned Sharpless said last week at the 2019 National Organization for Rare Diseases (NORD) meeting.

The FDA recently has provided support for several initiatives on the study of rare diseases, including $4.1 million for two research grants to fund a natural history study of rare diseases such as medullar thyroid cancer and Duchenne Muscular Dystrophy.

The agency’s Center for Drug Evaluation and Research also provided funding through an agreement with NORD and the Critical Path Institute (C-Path) to develop a data platform to manage information on rare diseases. The Rare Disease Cures Accelerator — Data Analytics Platform (RDCA-DAP) will contain patient-level data from clinical trials, observational studies, patient registries, and real-world data such as electronic health records across a multitude of rare diseases.

NIH Makes Rare Disease Grants to Three Universities

NIH has awarded $7.8 million in grants to three universities conducting research on myasthenia gravis.

In addition to the funding, Yale University, George Washington University and Duke University will join the rare disease network for Myasthenia Gravis (MGNet).

MGNet, established under the NIH’s National Center for Advancing Translational Sciences, will add seven study sites to the 200 established at NIH rare disease research centers across the U.S.

UK Trials Still Underreported Despite Lawmaker Attempts

Clinical trial transparency problems continue in the UK despite warning letters to institutions that have failed to comply with the country’s legal mandate to report trial results.

A new report by nonprofit AllTrials found that fewer than nine percent of 151 trials studied have posted all their trial results on the EU clinical trials register. More than 25 percent have failed to report any trial data at all.

The AllTrials study comes almost exactly a year after a report by the UK’s parliamentary Science and Technology Committee showed that only half of trials had reported their results within the required one year of the trial’s end (CenterWatch Weekly, Nov. 12, 2018).

A letter sent by the committee to noncompliant institutions in January warned that they had six months to improve their reporting. The committee, which has scheduled an Oct. 29 meeting to review the lack of progress, has urged the NHS Health and Research Authority to enforce rules and impose penalties on trial scofflaws.

Ken Getz’s New Edition of “The Gift of Participation” Released

Ken Getz, founder of CenterWatch and director of the Tufts Center for the Study of Drug Development, has released the third edition of his book, “The Gift of Participation.”

The book focuses on the value of participation in clinical trials, what participation means for patients and how it can help advance science.

Practical information is included on subjects such as insurance coverage, compensation and tax ramifications for clinical research volunteers.

Additions to the book include:

• The role that social and digital media play in clinical research;
• The collection of biomarker data and genetic material in clinical research;
• Evolving rules on clinical trial results disclosure; and
• The patient engagement movement.

For more information, click here: https://bit.ly/2MQ2tgl.