Open Notebook Science - In respectful memory of Dr. Jean Claude-Bradley - Open Notebook Science Pioneer and Practicioner

http://www.slideshare.net/hpiwowar/right-time-right-place-to-change-the-world

Open Notebook Science


From Wikipedia, the free encyclopedia:
Open Notebook Science is the practice of making the entire primary record of a research project publicly available online as it is recorded. This involves placing the personal, or laboratory, notebook of the researcher online along with all raw and processed data, and any associated material, as this material is generated. The approach may be summed up by the slogan 'no insider information'. It is the logical extreme of transparent approaches to research and explicitly includes the making available of failed, less significant, and otherwise unpublished experiments; so called 'Dark Data'.[1] The practice of Open Notebook Science, although not the norm in the academic community, has gained significant recent attention in the research,[2][3] general,[1][4] and peer-reviewed[5] media as part of a general trend towards more open approaches in research practice and publishing. Open Notebook Science can therefore be described as part of a wider open Science movement that includes the advocacy and adoption of open access publication, open data,crowdsourcing data, and citizen science. It is inspired in part by the success of open-source software[6] and draws on many of its ideas. Click for more.

Crowdsourcing: an overview and applications to ophthalmology

http://www.ncbi.nlm.nih.gov/pubmed/26761188

Jean-Claude Bradley is an organic chemist at Drexel University in Philadelphia. As with most scientists, Bradley used to be very secretive. He kept his research under wraps until publication and frequently applied for patents on his work in nanotechnology and gene therapy: Dr. Bradley has now become a practicioner of and outspoken proponent for open notebook science.

Links to Dr. Bradley's work:
Interview with Dr. Bradley on the "Impact of Open Notebook Science"
Dr. Bradley's USEFULCHEM open notebook science Wikispace
Dr. Bradley ONS 2012 presentation at Georgia Tech


Miscellaneous news and articles:


Power to the People: Participant Ownership of Clinical Trial Data: http://stm.sciencemag.org/content/3/69/69cm3.full

6/10/2013 Press Release: Bill Gates and Tenaya Capital Invest in ResearchGate to Advance Scientific Innovation Through Open Science
Dragoneer Investment Group, Thrive Capital, and existing investors Benchmark and Founders Fund join to fuel growth with 35 million USD Series C
http://www.fiercebiotech.com/press-releases/bill-gates-and-tenaya-capital-invest-researchgate-advance-scientific-innova

6/5/2013 Press Release: Research organizations agree to share genetic data: http://r.smartbrief.com/resp/eyitCmnjzmCjcOmcCidyfTDXBJoYZQ?format=standard
The NIH has joined about 70 medical, research and advocacy organizations across the globe in an initiative to create a shared database of genetic and clinical information. The coalition will agree on standards for representing and sharing genetic data as well as for obtaining patient consent. Researchers hope pooled data will allow them to understand and treat rare genetic mutations in cancer, pediatric diseases, heart disease and other conditions. Nature (free content)/News blog (6/5/2013)

Video - Changing the R&D Process:

Freda Lewis-Hall, a 40 year spouse of a rare disease patient and Executive Vice President and Chief Medical Officer at Pfizer, presented a keynote address at World Orphan Drug Congress USA 2013 on the topic, ‘Fast forward – thinking through the next generation of orphan drugs.’ World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development.


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An Audacious Goal?


What if we could evolve to a less than two year, less than 2 million dollar process that would move proven concepts from the lab to the patient and make targeted treatments for rare eye disorders available at a reasonable cost. This would accelerate availability of treatment for even small patient populations.
Patients and patient groups need to genetically identify themselves and connect with researchers, agencies, and industry. Patients must be involved in driving the process of attaining treatments and in an ongoing dialogue about risk vs. benefit from their perspective. Patients must educate themselves about regulatory patheways and assert their stakeholder rights to drive the process for treatment accessibility.
Researchers need to connect with other researchers and share knowledge, models, concepts, and methods where common disease characteristics exist to avoid duplication of costs, time, and effort. Virtual and physical disease specific cell, tissue, antibody, etc. banks need to be established to jump start new research into a disease or mechanism of disease. Collaborations can be established that study multiple diseases and therapies simultaneously, which can evolve into an ongoing physical and virtual infrastructure for both pre-clinical and clinical trials. This will leverage resources and avoid the “setup, startup, study, tear down” resource cycle that adds time and cost to developing and delivering treatments. Public/private partnerships would speed development and allow sharing of risks and rewards
The division of medicine by body part is becoming less relevant. We need collaboration with researchers of non-eye disorders, patient groups and other stakeholders to communicate and share when similarities exist in cause, disease mechanism, research methods, etc. True collaboration within and between agencies, institutions, and other entities will speed development and reduce cost.
Delivery systems such as viral and non-viral vectors and other technologies that can deliver genetic material or drugs need to be established as approved delivery methods, enabling quicker adaptation to specific genetic disorders, i.e. viewing the delivery method as a device and genetic material as a transplant.
The continuing increase in understanding the underlying causes and mechanisms of disease and targeting of treatments will allow greater emphasis on in vitro and ex vivo study, quantification of cellular response, qualification of biomarkers, etc. Diagnostic tools that will detect specific proteins, biomarkers, cellular response, visual function, etc. need to be further developed.
Regulatory agencies must develop new approaches to approval for highly targeted therapies. Working toward this goal will lay groundwork for reproducible and adaptable regulatory processes that can be leveraged for many eye and other disorders. Harmonization of requirements by various regulatory entities would further speed us toward this goal by reducing the number of trials necessary to make treatments available globally. The relatively new "Breakthrough Therapies" pathway is a giant leap in this direction, allowing the opportunity for conditional approval of qualifying therapies as early as after phase one studies. Patients should be empowered to determine whether the risk/benefit is justified under expanded access programs.
When the audacious goal is achieved, the impact would be......exponential acceleration of approval and real availability of treatments through creation of efficient processes in an “open source” type collaborative environment. This will result in more people receiving approved treatments for eye diseases under a sustainable economic and regulatory model sooner, rather than later, or never. Benefits will also extend to non-eye disorders.
There are many examples of what happens when closed systems open up – capability and availability increase while cost and time to market decrease. Even small patient populations could achieve access to treatments targeted to their specific disease. Reasonable treatment costs will increase payer acceptance, resulting in a sustainable economic model. Considering that only a fraction of rare disease treatments are available and their high cost, it is clear that the current economic and procedural framework cannot scale up to address the ever increasing number of identified rare eye and other diseases.