Building on President Obama’s announcement in his recent State of the Union Address, the US Administration has unveiled details about the Precision Medicine Initiative, a bold new research effort to revolutionize how we improve health and treat disease. Launched with a $215 million investment in the President’s 2016 Budget, the Precision Medicine Initiative will pioneer a new model of patient-powered research that promises to accelerate biomedical discoveries and provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for which patients.
Through collaborative public and private efforts, the Precision Medicine Initiative will leverage advances in genomics, emerging methods for managing and analyzing large data sets while protecting privacy, and health information technology to accelerate biomedical discoveries. The Initiative will also engage a million or more Americans to volunteer to contribute their health data to improve health outcomes, fuel the development of new treatments, and catalyze a new era of data-based and more precise medical treatment.
Key Investments to Launch the Precision Medicine Initiative:
Complementing robust investments to broadly support research, development, and innovation, the President’s 2016 Budget will provide a $215 million investment for the National Institutes of Health (NIH), together with the Food and Drug Administration (FDA), and the Office of the National Coordinator for Health Information Technology (ONC) to support this effort, including:
- $130 million to NIH for development of a voluntary national research cohort of a million or more volunteers to propel our understanding of health and disease and set the foundation for a new way of doing research through engaged participants and open, responsible data sharing.
- $70 million to the National Cancer Institute (NCI), part of NIH, to scale up efforts to identify genomic drivers in cancer and apply that knowledge in the development of more effective approaches to cancer treatment.
- $10 million to FDA to acquire additional expertise and advance the development of high quality, curated databases to support the regulatory structure needed to advance innovation in precision medicine and protect public health.
- $5 million to ONC to support the development of interoperability standards and requirements that address privacy and enable secure exchange of data across systems.
ICON plc has announced a new global innovation hub which will create 200 new jobs in Ireland. The hub will foster the development of new technologies and clinical trial processes that will enable faster access to large volumes of clinical data and will enable clinical trial personnel to derive better insights from the data. The 200 new jobs will include roles in IT, Data Analytics, Clinical Science, Project Management, Finance and Human Resources.
Commenting on the announcement, Ciaran Murray, Chief Executive Officer, ICON plc, said: “Since our foundation in Dublin in 1990, ICON has grown to become a global leader in clinical research, employing 11,000 highly-skilled professionals in 40 countries. We have successfully built relationships with all of the world’s top biopharma companies and have been at the forefront of innovation in the design and conduct of global clinical trials. As innovation plays an increasingly important role in improving the outcomes of clinical trials, we are excited to reinforce our commitment to that goal by locating our global innovation hub in Ireland, one of the leading R&D and innovation centres in the world.”
Chugai Pharma has announced that it will establish a new division from April 1, specialising in early clinical development to accelerate the generation of proof-of-concept for in-house drug candidates.
Chugai’s mid-term management plan “ACCEL 15”, sets “Acceleration of global development” as one of the top priority challenges. Newly establishing the Translational Clinical Research Division as one of the measures to address those challenges will enable to build a development setup equipped with top-level quality and speed in the world, which would allow Chugai to obtain proof-of-concept at an earlier stage.
The FDA has published a list of new and draft guidance documents that it plans to publish during 2015.
Several topics stand out among the 90 announced as potentially important for the clinical research industry:
- Standards for Clinical Trial Imaging Endpoints
- Dose Selection in Drug Development
- Pharmacokinetics in Patients with Impaired Renal Function – Study Design, Data Analysis and Impact on Dosing and Labeling
- Multiple Endpoints in Clinical Trials (Statistical)
- Use of Electronic Informed Consent in Clinical Investigations Questions and Answers
- Investigational New Drug Applications Prepared and Submitted by Clinical Sponsor Investigators
ClinDev will continue monitoring the FDA website for newly-released guidance.
A number of medicines for which authorisation in the European Union (EU) was primarily based on clinical studies conducted at GVK Biosciences in Hyderabad, India should be suspended, says the European Medicines Agency (EMA). The recommendation is based on findings from an inspection that raised concerns about how GVK conducted studies at the Hyderabad site on behalf of marketing authorisation holders.
Upon the request of the European Commission, EMA’s Committee for Medicinal Products for Human Use (CHMP) looked at over 1,000 pharmaceutical forms and strengths of medicines studied at the GVK site. For over 700 of them, insufficient supporting data from other sources were available, resulting in the recommended suspension.
The inspection of GVK that led to the CHMP’s recommendation was carried out by the French medicines agency (ANSM). The inspection revealed data manipulations of electrocardiograms (ECGs) during the conduct of some studies of generic medicines. These manipulations appeared to have taken place over a period of at least five years. Their systematic nature, the extended period of time during which they took place and the number of members of staff involved cast doubt on the integrity of the way trials were performed at the site generally and on the reliability of data generated at that site. There is no evidence of harm or lack of effectiveness linked to the conduct of studies by GVK Biosciences.
The European Medicines Agency has announced a public consultation on how its new transparency rules should be applied to the clinical trial database specified in the 2014 EU Clinical Trial Regulation. Stakeholders are invited to send their comments before 18 February 2015.
The European Clinical Trial Regulation aims to create an environment that is favourable to conducting clinical trials in the European Union (EU), with the highest standards of safety for participants. The Regulation ensures that the rules for conducting clinical trials are consistent throughout the EU. It also transforms the level of information publicly available for each clinical trial carried out in the EU by requiring transparency on the authorisation, conduct, and results of the trial. The Regulation will apply to clinical trials that are registered once the Regulation is in operation (not before 28 May 2016).
Clinipace Worldwide has put in place a $50 million ‘war chest’ in a round of debt and equity financing to capitalize on a pipeline of corporate acquisitions. The company has announced that it plans to use the capital to continue building its therapeutic expertise and to further expand its capacity to deliver services to its clients.
Global clinical trial performance and efficiency are hampered by high turnover and noncompliance among principal investigators and wide variation in investigative site experience, according to a recently completed assessment by the Tufts Center for the Study of Drug Development.
Although the highest turnover rates are observed among the least active investigators, turnover rates have been getting progressively worse among more active investigators. At the same time, protocol noncompliance, the most common performance deficiency and one that has grown the fastest during the past decade, accounted for 46% of all investigative site deficiencies, Tufts CSDD said.
“Operating conditions for clinical trials have noticeably worsened in recent years,” said Ken Getz, associate professor and director of sponsored research at Tufts CSDD. “Most frustrating for drug sponsors and contract research organizations looking to achieve higher levels of predictable performance is the wide variation that exists in the infrastructure, stability, and experience levels of investigative sites conducting clinical trials globally.”
The US Institute of Medicine has published its report into transparency around clinical trial data. Somewhat behind policy developments in Europe on this topic, the US body first announced its intention to review the area in April 2014 and issued a discussion framework in January 2014.
In response to 23 public- and private-sector sponsors in the United States and abroad, the Institute of Medicine (IOM) assembled a committee to develop guiding principles and a practical framework for the responsible sharing of clinical trial data. In its report, Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk, the committee concludes that sharing data is in the public interest, but a multi-stakeholder effort is needed to develop a culture, infrastructure, and policies that will foster responsible sharing—now and in the future.
Specific recommendations in the report include that:
- Data sharing plans are registered with the trial registration before the trial begins
- Summary results are published within a year of a trial’s end
- Full data sets from a clinical trial should be shared within 18 months of the trial’s end
- Data sharing initiatives should be overseen by independent panels of experts and the public, and should be as transparent as possible.
The AllTrials campaign welcomed the report, but commented that its recommendations could have been stronger if they had included clear pathways to implementation and calls for audits of compliance.
On 1 January 2015 the new EMA policy on publication of clinical data entered into force. Under this policy, the Agency proactively publishes the clinical reports submitted as part of marketing-authorisation applications for human medicines. Since 2010 the Agency has been releasing clinical-trial reports on request, under its access-to-documents policy.
The policy will apply to clinical reports contained in all marketing-authorisation applications submitted after that date. The reports will be released as soon as a decision on the application has been taken.
The Agency embarked on this process because it believes that the release of data is about establishing trust and confidence in the system, because it will allow the public to better understand the Agency’s decision-making. In addition, academics and researchers will be able to re-assess data sets. The publication of clinical reports will also help to avoid duplication of clinical trials, foster innovation and encourage development of new medicines.