|By PR Newswire||
|August 6, 2014 03:00 PM EDT||
Input will help shape the role of public contributions to orphan drug approvals
OTTAWA, Aug. 6, 2014 /CNW/ - The Honourable Rona Ambrose, Minister of Health, announced today that the Government of Canada has launched a pilot project targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs. As announced in October of 2012, Health Canada has been developing an Orphan Drug Framework to spur innovation and research into new treatments for rare diseases and also to encourage patient participation. The Pilot Project will simulate how input from patients will be gathered and incorporated into the drug submission review process once the Orphan Drug Framework is in effect.
Drug manufacturers, Hoffmann-La Roche Limited and Hyperion Therapeutics, Inc have agreed to participate in the pilot with two of their drug review submissions. The review of Hoffmann-La Roche's medication obinutuzumab for the treatment of chronic lymphocytic leukemia (CLL) will be used for the pilot. Hyperion Therapeutics Inc.'s review is for Ravicti ® (glycerol phenylbutyrate) Oral Liquid which is used for the treatment of urea cycle disorders. Hoffmann-La Roche and Hyperion's participation will serve as models for how patient input may inform drug authorization decisions.
Patients will be asked to comment on the following:
- how the rare disease affects their ability to manage their day-to-day lives;
- what treatments are currently available (if any);
- what therapeutic benefits are most important to them; and
- their risk tolerance for any new treatments.
Health Canada will be asking patient advocacy groups including the Canadian Organization for Rare Disorders to assist in engaging specific Canadian patients affected by CLL or urea cycle disorders to participate in the project. The patient feedback provided during the pilot will assist Health Canada in assessing and refining its approach to gathering patient input. This will ensure that patient perspectives are considered in future orphan drug authorizations.
- Orphan drugs are those drugs used to treat rare diseases. A rare disease is a life-threatening, seriously debilitating, or serious chronic condition that only affects a very small number of patients (typically less than 5 in 10,000 persons).
- When the Orphan Drug Framework is fully implemented, Canadian patients will be invited to participate in future orphan drug authorizations through Health Canada's website.
Patients with CLL or urea cycle disorders, their current or former caregivers or healthcare professionals, and others with first-hand knowledge of CLL or urea cycle disorders are encouraged to contact Health Canada by email if they wish to participate at: [email protected].
"No one has a better understanding of what it means to have a rare
disease than the patients who are affected by them. The input of these
patient volunteers is absolutely invaluable and our government has
committed to making their experiences and perspectives an important
addition to our scientific approach to drug assessments for rare
Minister of Health
"Roche recognizes that patients and patient organizations are playing an
increasingly important role in drug review and health technology
assessment and bring valuable experience, perspective and insight to
the process. As an organization committed to patients, Roche is pleased
to participate in this Health Canada pilot program that is seeking
patient input within the regulatory review process."
Vice President, Medical & Regulatory Affairs, Hoffmann-La Roche Limited
"As part of our full commitment to serving the urea cycle disorder
community, we're looking forward to participating in Health Canada's
Patient Involvement Pilot Project. Orphan diseases have a dramatic
impact on the lives they touch, making this initiative critical to the
process of drug review and approval."
Dr. Bruce Scharschmidt
Senior Vice president, Chief Medical and Development Officer, Hyperion Therapeutics, Inc.
"Kudos to Health Canada for recognizing the value of the direct patient
voice in the drug review process, for designing a user-friendly
template that will encourage patient engagement, and for piloting this
in advance of the orphan drug regulations. The Canadian Organization
for Rare Disorders is pleased to have had the opportunity to
collaborate on this important initiative and to promote this in the
President, Canadian Organization for Rare Disorders
SOURCE Health Canada
In their Live Hack” presentation at 17th Cloud Expo, Stephen Coty and Paul Fletcher, Chief Security Evangelists at Alert Logic, will provide the audience with a chance to see a live demonstration of the common tools cyber attackers use to attack cloud and traditional IT systems. This “Live Hack” uses open source attack tools that are free and available for download by anybody. Attendees will learn where to find and how to operate these tools for the purpose of testing their own IT infrastructu...
Aug. 30, 2015 02:00 AM EDT Reads: 384
WebRTC has had a real tough three or four years, and so have those working with it. Only a few short years ago, the development world were excited about WebRTC and proclaiming how awesome it was. You might have played with the technology a couple of years ago, only to find the extra infrastructure requirements were painful to implement and poorly documented. This probably left a bitter taste in your mouth, especially when things went wrong.
Aug. 30, 2015 01:00 AM EDT Reads: 426
It’s been proven time and time again that in tech, diversity drives greater innovation, better team productivity and greater profits and market share. So what can we do in our DevOps teams to embrace diversity and help transform the culture of development and operations into a true “DevOps” team? In her session at DevOps Summit, Stefana Muller, Director, Product Management – Continuous Delivery at CA Technologies, answered that question citing examples, showing how to create opportunities for ...
Aug. 30, 2015 01:00 AM EDT Reads: 470
Whether you like it or not, DevOps is on track for a remarkable alliance with security. The SEC didn’t approve the merger. And your boss hasn’t heard anything about it. Yet, this unruly triumvirate will soon dominate and deliver DevSecOps faster, cheaper, better, and on an unprecedented scale. In his session at DevOps Summit, Frank Bunger, VP of Customer Success at ScriptRock, will discuss how this cathartic moment will propel the DevOps movement from such stuff as dreams are made on to a prac...
Aug. 30, 2015 01:00 AM EDT Reads: 205
SYS-CON Events announced today that IceWarp will exhibit at the 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. IceWarp, the leader of cloud and on-premise messaging, delivers secured email, chat, documents, conferencing and collaboration to today's mobile workforce, all in one unified interface
Aug. 30, 2015 01:00 AM EDT Reads: 381
With the proliferation of connected devices underpinning new Internet of Things systems, Brandon Schulz, Director of Luxoft IoT – Retail, will be looking at the transformation of the retail customer experience in brick and mortar stores in his session at @ThingsExpo. Questions he will address include: Will beacons drop to the wayside like QR codes, or be a proximity-based profit driver? How will the customer experience change in stores of all types when everything can be instrumented and a...
Aug. 29, 2015 11:45 PM EDT Reads: 427
Red Hat is investing in Tesora, the number one contributor to OpenStack Trove Database as a Service (DBaaS) also ranked among the top 20 companies contributing to OpenStack overall. Tesora, the company bringing OpenStack Trove Database as a Service (DBaaS) to the enterprise, has announced that Red Hat and others have invested in the company as a part of Tesora's latest funding round. The funding agreement expands on the ongoing collaboration between Tesora and Red Hat, which dates back to Febr...
Aug. 29, 2015 10:00 PM EDT Reads: 321
The Internet of Things (IoT) is about the digitization of physical assets including sensors, devices, machines, gateways, and the network. It creates possibilities for significant value creation and new revenue generating business models via data democratization and ubiquitous analytics across IoT networks. The explosion of data in all forms in IoT requires a more robust and broader lens in order to enable smarter timely actions and better outcomes. Business operations become the key driver of I...
Aug. 29, 2015 07:30 PM EDT Reads: 378
SYS-CON Events announced today that DataClear Inc. will exhibit at the 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. The DataClear ‘BlackBox’ is the only solution that moves your PC, browsing and data out of the United States and away from prying (and spying) eyes. Its solution automatically builds you a clean, on-demand, virus free, new virtual cloud based PC outside of the United States, and wipes it clean...
Aug. 29, 2015 07:15 PM EDT Reads: 371
Culture is the most important ingredient of DevOps. The challenge for most organizations is defining and communicating a vision of beneficial DevOps culture for their organizations, and then facilitating the changes needed to achieve that. Often this comes down to an ability to provide true leadership. As a CIO, are your direct reports IT managers or are they IT leaders? The hard truth is that many IT managers have risen through the ranks based on their technical skills, not their leadership ab...
Aug. 29, 2015 04:00 PM EDT Reads: 327
A producer of the first smartphones and tablets, presenter Lee M. Williams will talk about how he is now applying his experience in mobile technology to the design and development of the next generation of Environmental and Sustainability Services at ETwater. In his session at @ThingsExpo, Lee Williams, COO of ETwater, will talk about how he is now applying his experience in mobile technology to the design and development of the next generation of Environmental and Sustainability Services at ET...
Aug. 29, 2015 03:45 PM EDT Reads: 111
Cloud and datacenter migration innovator AppZero has joined the Microsoft Enterprise Cloud Alliance Program. AppZero is a fast, flexible way to move Windows Server applications from any source machine – physical or virtual – to any destination server, in any cloud or datacenter, using its patented container technology. AppZero’s container is also called a Virtual Application Appliance (VAA). To facilitate Microsoft Azure onboarding, AppZero has two purpose-built offerings: AppZero SP for Azure,...
Aug. 29, 2015 01:00 PM EDT Reads: 148
WSM International, the pioneer and leader in server migration services, has announced an agreement with WHOA.com, a leader in providing secure public, private and hybrid cloud computing services. Under terms of the agreement, WSM will provide migration services to WHOA.com customers to relocate some or all of their applications, digital assets, and other computing workloads to WHOA.com enterprise-class, secure cloud infrastructure. The migration services include detailed evaluation and planning...
Aug. 29, 2015 12:00 PM EDT Reads: 145
This Enterprise Strategy Group lab validation report of the NEC Express5800/R320 server with Intel® Xeon® processor presents the benefits of 99.999% uptime NEC fault-tolerant servers that lower overall virtualized server total cost of ownership. This report also includes survey data on the significant costs associated with system outages impacting enterprise and web applications. Click Here to Download Report Now!
Aug. 29, 2015 11:45 AM EDT Reads: 153
IBM’s Blue Box Cloud, powered by OpenStack, is now available in any of IBM’s globally integrated cloud data centers running SoftLayer infrastructure. Less than 90 days after its acquisition of Blue Box, IBM has integrated its Blue Box Cloud Dedicated private-cloud-as-a-service into its broader portfolio of OpenStack® based solutions. The announcement, made today at the OpenStack Silicon Valley event, further highlights IBM’s continued support to deliver OpenStack solutions across all cloud depl...
Aug. 29, 2015 11:30 AM EDT Reads: 202