Category Archives: Global Clinical Research

Orphan Drug Development – Why They Are SO Important

Orphan drugs treat diseases so rare that sponsors are often unwilling to follow the usual pharmaceutical drug development marketing conditions. Most rare diseases are genetic, and therefore present throughout the sufferer’s life, even when symptoms are not immediately apparent. Many diseases appear early in life, and a total of 30 percent of children with orphan diseases die before the age of five. Global estimates put the number of different diseases between 5000 and 7000, with an average of five new conditions discovered every week. Unfortunately, in total, only 5 percent of orphan diseases have FDA approved treatment.?

Record Growth Shows Clear Commitment

Although the market is small for orphan drugs, pharmaceutical drug development in this area has seen notable growth in recent years. Currently, 350 orphan drugs are approved for sale in the U.S., including not only pharmaceutical and biological products but also medical devices and dietary products. A record number (10) of new drugs were approved by the FDA in 2011 along with five new drugs by the European Medicines Agency (EMA).

Proven Examples of Success

Orphan Drugs Find a Home

Orphan Drugs Find a Home at Criterium Global CRO

Many large pharmaceutical companies are expanding their orphan drug development and even establishing entire business units dedicated to rare diseases. Although orphan drugs treat only a small number of patients, they can receive a weighty revenue, and companies get a significant competitive advantage by being the first to market.?A good example is Rituxan? from Genentech, the second most profitable drug in the world, given orphan status to treat B-cell Non-Hogkin’s lymphoma. In 2010, it yielded $5.24 billion in sales for its use as an orphan drug and for extended usage for other types of cancer and rheumatoid arthritis.?It is quite possible to more than compensate for the smaller number of patients an orphan drug may help, through the increased market share, lower marketing costs, higher pricing, longer exclusivity period, and faster returns.

There is an even greater potential for profit when drugs have multiple orphan disease indications or if they can later go on to be used for more widespread non-orphan indications; for example, Gleevec? from Novartis Oncology resulted in sales of $2.4 billion in 2010. In addition, a number orphan drugs are biologics, meaning they are less likely to have generic equivalents, which extends the value to sponsors even after patent expiration.

Economically Feasible – Shorter Development Timelines

Investing in orphan drugs is at least as economically feasible as non-orphan pharmaceutical drug development due to the higher rates of approval and the shorter development times. For instance, the chances of approval for orphan drugs is very high, at 82 percent, compared to non-orphan drugs, at just 35 percent. Additionally, the time taken from Phase II to market is often shorter due to both smaller clinical trials and to the FDA Fast Track designation. On average, the timeline for orphan drugs is 3.9 years while traditional drugs typically take 5.4 years to reach the market.

With new orphan diseases being discovered every week, the potential for pharmaceutical drug development in this area is huge. While initially the market for these drugs may appear small, this is more than counterbalanced by the revenue opportunities and the quick time-to-market.

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The Advantages of Translational Research Methodology

Applying basic research knowledge to practical applications is a well-known obstacle to?progress?in science. This issue is seen most often in medical science. There is often a lag between emerging scientific findings and their application as treatments to patients who may benefit from them.Translational research is aimed at taking basic science? research to the next level.?It connects new discoveries to application in patients.

Multidisciplinary Collaboration
There are four basic steps to consider in this research tactic.

  • Development of techniques to translate new knowledge generated in a lab environment, or at the bench level, to human testing
  • Refinement of human study results for? clinical use
  • Transference of practices from academic medical centers to use in the care of patients in community practice
  • Continued evaluation of data from the previous steps to provide confirmation of the efficacy and tolerability of new therapies

The goal of translational research is to quicken the transition from the lab to the patients who may benefit from the basic science findings. Thus translational research is a collaborative effort among basic researchers, academic physicians and community health professionals to provide new treatments to appropriate patient populations.

Funding Options
Translational research can potentially expand the funding options for a project.

  • Researchers benefit by working with a center that is able to provide seed funding. They reduce overall project costs by utilizing basic science center personnel for development of new therapies that then can be applied to patients in the community..
  • For universities, setting up a translational research center is a tactical approach to improving funding options. Through the research center, academic organizations develop strategic plans to provide improvements in public health benefitting overall community.
  • Communities benefit by gaining a stake in the study. The community becomes a contributing member of innovative medical research.

Stoking Excitement in a Field of Study
The developing barriers between research and clinical applications tend to dim interest in research projects. Moving towards a translational approach expands these possibilities. The power to take new knowledge from the bench to a clinic setting and then back to the bench for analysis lifts some of the impediments to expanding interest in a project.

Translational Science Research

Physicians in the community are able to see
basic research translated into practical application. A project that was once unapproachable due to time constraints and the complexity of the study boundaries will now draw interest and participation.

Translational research is a bench-to-bedside approach to medical developments. With this two-fold methodology, basic science presents tools to clinicians that have the potential to improve patient care. Clinicians are able to make observations and provide valuable feedback about the progression of disease that will lead to more discoveries. A translational model benefits the community by speeding the application of new research findings to patients and thus drives the clinical research progress at a quicker pace.

The Oncology Consortia of Criterium believes this methodology is the best way to speed results of research to benefit the public – it’s why we say we are “Changing the Way Cancer is Managed”.? Read more about our approach at the Academic GI Cancer Consortium or at the Academic Myeloma Consortia web pages.

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Claire WyntersThu, 11 Apr 2013 15:07:00 GMT

Why run global clinical trials?

Clinical trials are a vital aspect of the pharmaceutical industry -?both as a source of validation for previously untested methods, as well as a way to publicly demonstrate the effectiveness of a developing product. As humans across the planet collaborate and network, global clinical trials are a necessity for maintaining accurate data across numerous demographics. Furthermore, conducting trials abroad positively influences the economies and well-being of developing nations.

Globe made of Flags_200All around the world, various ailments and negative conditions affect local populations in differing ways. To best treat the plethora of issues present in these wide-ranging communities, global clinical trials are needed to address the growing variable factors involved. It is a?recognized fact that minority groups and third-world citizens are vastly underrepresented in the modern medicine developed primarily by Westerners.?This is changing rapidly, as more and more clinical trials are funded and performed globally.

An important aspect of conducting global clinical trials is being aware of the differing regulations that exist in developed countries vs. under-developed countries.?Every trial has its own unique ethics and complexities that are carefully monitored and assessed by licensed physicians, and not shoe-horned into a universal set of regulations that may be inappropriate for any specific trial. By hosting testing locations in many?under-developed parts of the globe, a more local?flexibility can be used in research efforts for effective medicines in indigenous populations.

Additionally, conducting global clinical trials allows for researchers in smaller countries to work at the forefront of their field. While experts often travel to oversee the process, global trial activity means more employment and experience for the local doctors, investigators and clinical personnel. This provides ample opportunity for externally sponsored clinics to receive additional funding and investment which, in turn, can lead to a healthier and happier population overall.

Working in developing nations offers an opportunity to expand the scope of any trial ? both seasonally and with groups of people?Caduceus_311who have not been exposed to new medicines in a variety of therapeutic areas. Because of these na?ve patient populations, more extensive clinical trials can be executed and analyzed. Pharmaceutical corporations concerned about their bottom line have to carefully consider the best allocation of?their funds — and it?s important to understand that running trials in developing nations does not always provide a cost savings. Money must still be spent on securing locations, basic products, and housing and care of employees, and budgeting must suit the needs of the specific trial in that specific global location.

Medicine is a particularly important?and valuable branch of practical?science.? Seeking new, reliable and innovative information can be a timely, costly, and difficult process — and global clinical trials are much needed to improve the health problems of the world at large. Global trials provide more in-depth data across a wider demographic possibility, better freedom to experiment and explore, and more importantly, vital support of local native economies.

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Claire WyntersWed, 30 Jan 2013 14:45:00 GMT

Quick Takes: the Pharma C-Suite with John Hudak, President Hudak-President

john hudak quote_325We have successfully conducted trials for over 20 years – since 1991.
Criterium?s mission to provide our clients, staff, consultants and sites an extraordinary experience, with integrity.

Our focus, for every client, is simple, and we mean it:? Provide Quality Solutions.? Reduce Costs.? Accelerate Timelines.? It?s the root of our business philosophy.? We respond very quickly to clients from their first contact with us for a bid proposal to every aspect of the projects we win.? Every single person on our staff is aware of these company directives and works towards them.? This is a very real, daily, activity for us, not just words.? When I look ahead at the next twenty years, I am enthusiastic about what lies ahead in this field and committed to establishing better and more efficient ways to support drug development, to cut costs and to improve technology resources directly related to these initiatives.

We offer the broadest range of services in our market niche, because we CAN.? We have staff from all aspects of clinical research experience?? that allows us to do that.? It?s a unique mix of incredibly bright and dedicated people ? I am very fortunate to have them.? As a medium-sized CRO we can be more agile, and have the advantages of moving more quickly than the larger, leviathan CRO?s, starting and completing trials more efficiently, and giving clients a much more personalized, one-on-one experience. Our unique technology-based paradigm allows us to respond to sponsor needs in specific detail and to deploy trials rapidly throughout the world for fast, successful trials.

Our clients are among the top 10 pharma companies to small venture capitalized biotechnology and new formulation companies. They are USA domestic and they are also based in Europe, Israel and Asia. We have worked with generic companies when they need to have bioequivalence studies with clinical endpoints done.? We have a satisfied client list as varied as the therapeutic areas in which we have experience.? Our personalized service experience and ?Get to Know Us!? philosophy has proven to serve our clients well.?

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John HudakThu, 17 Jan 2013 22:45:00 GMT

Best Practices Series: Know What WE Know About ICH GCP

The ICH (international Conference of Harmonization) plays a major role in providing pharmaceutical standards, accepted by regulatory authorities across the globe, and GCP (Good Clinical Practice) is no different.? The ICH GCP guideline was developed with an aim to protect the rights and safety of human subjects while assuring credible and internationally acceptable clinical trial data.? Along with WHO (World Health Organization) recommendations, the international ethical and scientific standards for clinical trials involving human test subjects (GCP) from the regions of Europe (EU), United States of America, Japan, Australia, Canada, and Nordic countries were all taken into consideration.




Importance of GCP
It is safe to say that GCP in clinical trials requires a lot of time and resources. While following best practices protocols when conducting a research does yield more reliable data and strict ethical protocols protect the subject rights and safety, the GCP for any study was in the clinician?s domain until relatively recently. However, loose GCP protocols were not acceptable by regulatory authorities and The Declaration of Helsinki in 1964 provided a foundation for internal GCP guidelines for many organizations.

Under ICH GCP, all crucial aspects of best practices were unified under one guideline. For the independent researchers or noncommercial trials, this guideline can feel to restrictive and demanding, bringing extra paperwork and increased cost. However, the document represents the way of thinking that should be always present, when conducting trials on humans; the same rigorous quality, ethical, and safety protocols should be followed, regardless of the trial type or origin.

ICH GCP and Its Principals
Frequent poor data monitoring, flawed statistical design and analysis, and questionable ethical methods used in clinical trials in the past have all contributed to a growing public and professional concern about research misconduct, and have lead to misleading, and sometimes even hazardous trial results. ICH GCP plays a vital role in assuring sound clinical trials and reliable trial results while protecting the patients rights and safety.

Ethical Aspects?
Clinical trials should be conducted in accordance with the ethical principals of the Declaration of Helsinki, and a study should be conducted only if the potential benefit for the society outweighs the possible risks for the subject.

Clinical Protocol
The trials should have scientifically sound and clear objectives, supported by prior clinical and nonclinical information, and should be conducted in compliance with a detailed protocol, approved by IRB (institutional review board) or IEC (independent ethic committee). Freely given informed consents should be obtained from every subject in the study.

Record Keeping
Every individual involved in conducting a study should be properly educated and trained, all clinical trial data should be properly recorded, handled, and stored, and the subject’s confidential data should be protected.

Study Report
The study report needs to be prepared at the end of the study, regardless of the study phase or the number of participants that were included in the trial. Following ICH GCP principles is crucial to providing significantly increased quality of treatment.

While some of the procedures in the ICH GCP can seem tedious, past experiences shows all of them make sense. Not only do they provide a safe environment for the participants, and improved treatments for society, they ensure that negative trial results are well documented and can prove as useful as positive results in future clinical trials. Along with the obvious benefits of complying with the best practices, apparent hindrances, such as increased bureaucracy and cost, can be greatly reduced with proper study design and utilization of approved clinical trial system and experts.?

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Claire WyntersThu, 03 Jan 2013 20:57:00 GMT

The Expanding Global Reach of Pharmaceutical Clinical Research

The global pharmaceutical industry has continued to evolve over the last few years into different permutations aimed at maximizing research and development (RD) capabilities while minimizing the overall costs associated with the discovery, development, and launching of new drugs.

Mumbai at night_215The environment that has grown from this process has lead to the emergence of an innovative new approach to sustaining product development by integrating a global clinical research framework into the traditional RD landscape with the aim of keeping the industry’s momentum toward continued important pharmaceutical developments.

During this period, the pharmaceutical industry in India has devoted a great deal of focused attention on bringing the subcontinent’s RD infrastructure up to the standards expected by the world’s major enterprises. These efforts have led to over 70 FDA approved US pharmaceutical firms establishing facilities in India, where they now conduct ongoing RD toward finding unique investigational new drugs (IND).

The Evolution of Pharmaceuticals in India
While thepharmaceutical industry in Indiahas advanced in response to this new focus to become the world’s third largest, the fact remains that there are actually a fairly low number of Indian-owned pharmaceutical companies. The lion’s share of these enterprises in India areoperated and controlled as subsidiaries for a wide sampling of the world’s leading foreign companies.

India has developed astrategy to further develop a home grown industry that rests largely on its efforts to build state-of-the-art facilities and infrastructure, while simultaneously working with global bio-tech educators to revised the university curriculum in order to raise the professional level of pharmaceutical researchers in India.

Added to this is the adoption of attractive direct foreign investment regulations in India, which make it both easy — and potentially lucrative — for companies interested in global clinical research options to seriously include India in their calculations.

lab-test-tubes_227The Professional Level
The overwhelming majority of employees involved in the global clinical research aspects of these companies are Indians, freeing foreign enterprises from having to relocate staff to these facilities. The level of committed professionalism instilled by parent enterprises is consistently maintained. As early as 2005, the FDA was already receiving 20% of all submitted ANDAs from Indian based RD.

Foreign firms are able to take advantage of innovative local sourcing ideas derived from knowledge of traditional medicinal systems, as well as associations with local universities, bio-tech institutes, with a wide array of pharmaceutical expertise.

Integrating India as a global clinical research component into your pharmaceutical company’s strategic growth plan offers the opportunity to provide a quality solution that can meet any expectation of a clinical research process — from initial planning all the way through to clinical RD. And each element of this process can be achieved at considerably reduced costs. Our experiences on the ground in India can provide you with valuable guidance.

The bottom line is that incorporating a presence in India into a systematic expansion of global clinical research presents a forward thinking pharmaceutical enterprise with a real opportunity to reap a considerablereturn on their investment.

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Claire WyntersThu, 15 Nov 2012 15:46:00 GMT