Clinical News Roundup: What To Expect From PDUFA VI

By Ed Miseta, Chief Editor, Clinical Leader

The FDA and stakeholders from industry and patient groups have outlined the agreement they forged on the next reauthorization of the Prescription Drug User Fee Act (PDUFA) that begins in 2017. The agreement focuses on what’s to come on pre-market reviews, post-market safety, regulatory decision tools, and other ways FDA is preparing for the future of drug development. The details are available in a paper published by RAPS (the Regulatory Affairs Professionals Society) titled PDUFA VI: Highlights on What to Expect.

The agreement follows the July 2016 release of the full details of the performance and procedural goals for fiscal years 2018 through 2022. The 46-page document offers perspective on the direction FDA is headed for PDUFA VI in terms of incorporating the patient voice into drug development, enhancing the use of real-world evidence, improving benefit-risk assessments, and expanding post-market safety data and evaluations. At the meeting, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research (CDER), highlighted the importance of enhancing patient input and integrating it into regulatory decision making, particularly on how to make their needs known and ensure they are met in the regulatory process.

Theresa Mullen, director of CDER’s Office of Strategic Programs, responded to concerns raised about FDA’s dissemination of guidance documents and the slow process by which they are finalized, noting FDA only has a handful of employees who have the expertise to write such documents, and a lot of these officials can be lured away from FDA by better paid industry jobs with more flexible schedules.

FDA and stakeholders also took care to craft new types of meetings between pharmaceutical companies and FDA in PDUFA VI, as FDA receives thousands of meeting requests annually and both sides might not have enough time for internal deliberations.

New Super Vaccine Will Guard Against Pneumococcal Disease

A new super vaccine with the ability to eliminate all 90 known strains of the world’s most deadly bacterial disease is being developed in Australia. The Lead reports G-PN, a whole cell pneumococcal vaccine that is inactivated by exposure to gamma irradiation, has been developed by researchers from the University of Adelaide in South Australia. G-PN is capable of preventing disease caused by all serotypes of pneumococcus (Streptococcus pneumoniae).

Lead researcher James Paton said G-PN would help to increase immune responses to pneumococcus because it kept the antigenic structure of protein surfaces on the bacterium. “Pneumococcus is a big deal in terms of disease and it is the biggest bacterial killer on the planet,” he said. “There are currently similar wholesale vaccines being developed, which have used chemical killing but that is nowhere near as good. The fact that gamma radiation is being used to inactivate it makes it a better vaccine.”

A paper on the pneumococcal vaccine was published in the journal Clinical Science earlier this year. Professor Paton and his team are awaiting an outcome of a National Health and Medical Research Council (NHMRC) funding application to fund the completion of preclinical studies. According to the World Health Organization about one million children die of pneumococcal disease every year.

What Trends Are Impacting Clinical Trials?

Chris McSpiritt, clinical practice lead for the Paragon Life Science R&D Domain, has published his insights on trends impacting clinical trials today. McSpiritt notes much of the conversation around improving clinical trials has focused on patient centricity, which involves three stakeholder groups: patients, partners, and sites.

Attracting and retaining patients remains a challenge for sponsors, and McSpiritt sees four areas where technology can add value. The first is patient recruitment, where social media, patient communities, and online trial registries can make it easier for patients to find information about clinical trials. Online surveys and apps can then support preliminary self-screening that allows patients to provide information.

Increasingly complex protocols and other demands on the patient have made enrollment and consent a greater challenge. McSpiritt believes enrollment can be improved by leveraging more sophisticated informed consent solutions that incorporate training components. This will shift the focus of the informed consent process from risk mitigation to educating the patient about the trial. Patient retention will also be improved by leveraging eConsent processes to better prepare patients for the trial and providing more frequent and personalized communications/schedule management via technology.

The fourth area is safety and outcome monitoring/analysis. This can be improved by collecting data from patients in both passive and active manners. Sponsors can take advantage of new technologies to passively monitor patient stats such as heart rate and activity. From an active perspective, sponsors can leverage new technologies such as Apple ResearchKit to collect data on patient ability (e.g., coordination, response time).

Other trends McSpiritt believes are having an impact on trials are site centricity, which involves improving site start-up, payment, and management; and partner collaboration, which includes TMF management and CRO oversight.

NextDocs Founder Launches VitalTrax To Change Trial Paradigm

Zikria Syed, who co-founded NextDocs before selling it to Aurea Software, has started a new company to support clinical trials. With VitalTrax, Syed hopes to change the paradigm for doing clinical trials.

As reported on MedCityNews, Syed and his team have developed a nimble interface for executing trials that more closely resembles social media than traditional clinical research documents. The interface still conforms to regulatory requirements.

The company’s software is used to collect data directly from patients. It also automates the tasks of participants in clinical trials such as informed consent, meeting appointments and reminders for both principal investigators and participants, and payment processing. Syed added that the platform is also designed to respond to the push to enlist digital health technology in clinical trials for remote monitoring of participants. The software is also designed to give the same user interface on a desktop as it does on an Android or iPhone or tablet and switch between them.

India Eases Rules On Clinical Trials

PHARMABIZ reports the Drugs Controller General of India (DCGI) has decided to do away with the restrictions on the number of clinical trials an investigator can undertake at one time. Currently, no investigator is allowed to conduct more than three trials at any given period. However, the health ministry empowered the ethics committee to make a final call on the number of clinical trials an investigator can do at a time after examining the risk and complexity of the trials.

The decision to relax the rules comes in the wake of recent widespread resentment against restrictions on clinical trials. Experts felt restricting the number of studies for an investigator was not justified in the interest of clinical research in the country. As a result, the health ministry convened several meetings on the issue with industry associations and other stakeholders. Two meetings were held last year under the Chairmanship of the Secretary, Ministry of Health and other experts.

Clinical trial approvals in India have almost come to a halt since 2012 with very few applications being cleared by the DCGI. The situation was a result of growing criticism on the way trials were being conducted in India by certain pharmaceutical companies and CROs. Between July and August 2013, the DCGI had received as many as 1,122 applications for clinical trials. The New Drug Committee approved only 285 applications but the DCGI office cleared only 162. In October 2013 the Supreme Court issued an order staying all the 162 approvals granted by DCGI.