Multiple Myeloma Compound Gets Orphan Drug Status

Dr J S Macdonald MDDr. John S. Macdonald
Senior Consultant for The Academic GI Cancer Consultant Consortium (AGICC) & The Academic Myeloma Consortium (AMyC) of The Oncology Consortia of CRITERIUM, INC.

The US Food and Drug Administration has granted orphan drug status to an experimental compound for multiple myeloma known as KRN5500.

This is the second orphan drug designation for KRN5500, which is a novel, intravenous, nonopioid, nonnarcotic compound in phase 2 clinical development by DARA BioSciences.

Earlier this year, KRN5500 received orphan status for the treatment of chemotherapy-induced neuropathic pain refractory to conventional analgesics in patients with cancer.

“It is noteworthy in this regard that up to 20% of myeloma patients have intrinsic peripheral neuropathy, an incidence that increases to the range of 75% in patients treated with neurotoxic drugs such as thalidomide or bortezomib,” said David J. Drutz, MD, chief executive officer and chief medical officer of DARA BioSciences, in a statement.

In 2011, KRN5500 was granted a fast-track designation, a process designed to facilitate development and to expedite the review of drugs to treat serious conditions and fill an unmet medical need.FDA-Logo

The therapeutic potential of KRN5500 has been demonstrated in vitro and in vivo. The agent, which is a spicamycin derivative, exhibits antimyeloma effects by impairing both myeloma cells and osteoclasts, the company reports.

The orphan drug designation is “an important step toward the potential treatment of multiple myeloma and one of its major complications,” Dr. Drutz said. It will also help in the company’s “ongoing pursuit of partnering opportunities to assist in funding the clinical advancement and development pathway of KRN5500.”

Dr. John S. Macdonald comments on this important FDA decision…
“The fast tracking of this spicamycin derivative recognizes an agent that not only possesses anti cancer (multiple myeloma) activity likely to be beneficial in patients resistant to standard treatment, but also has the potential to decrease a very serious complication of myeloma, bone fractures because it inhibits osteoclast activity.”

SOURCE: Original article by Megan Brooks for MedScape – http://www.medscape.com/viewarticle/826882

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Immune Manipulation in Cancer Treatment: From Wishful Thinking to Tumors Shrinking

Colin Weekes MD PHD AGICCDr. Colin D. Weekes, Ph.D, PI at UC Denver for The Academic GI Cancer Consultant Consortium (AGICC) of The Oncology Consortia of CRITERIUM, INC.

The Holy Grail of cancer therapy has been to cause tumors in patients to disappear with minimal adverse effects on normal tissues. It has been clear, in concept, that one way to achieve this end would be to use the specificity implicit in the human immune system to recognize and destroy cancer cells while sparing normal cells.

Over the last 40 years there have been many attempts to stimulate the immune system in cancer patients in the hope that the this nonspecific stimulation would cause the immune system to recognize and destroy cancer cells. The literature is peppered with trials using non-specific immune stimulating adjuvants like BCG (Bacillus Calmette Guerin). Although some studies suggested some traces of antitumor activity, the overall assessment was negative. Dramatic and clinically meaningful responses didn’t occur. Other approaches to stimulate immunity by infusing patients with cells thought to have antitumor activity using auto and allogeneic stem cell transfer were both toxic and clinically disappointing. The adoptive transfer lymphoid cells, which had been ‘educated’ in vitro to recognize and destroy patient’s cancers (IL2/LAK and TIL infusions) tended to be very toxic. Although significant responses occurred, they were rare and were obtained with toxic side effects that were frequently very severe.

Recently, there have been examples of more significant, more specific and more sophisticated Chemistry behind cancer researchimmune manipulation that may be the leading edge of immune therapies that will be widely applicable to patients with cancer. What may be a ‘breakthrough strategy’ of immune manipulation in cancer has been demonstrated in melanoma. The FDA recently approved the monoclonal antibody Ipilimumab for use in patients with metastatic melanoma. This therapy is unique because its mechanism of action is to increase T-cell antitumor activity by blocking factors suppressing T-cell antitumor activity. Thus the intrinsic antitumor immunity in patients is allowed to function and results in meaningful clinical results (improved survival). Ipilimumab has the capability of producing autoimmunity since it blocks suppression of immune activity. However, clinical toxicity seems manageable. Other types of cancer may benefit from Ipilimumab or therapies with a similar mechanism of action.

The second example of a very intriguing immune manipulation in cancer has been reported this week from Rosenberg and colleagues work at the NCI. This group has been studying Adoptive Cell Transfer (ACT) in patients with metastatic cancer for the last 35 years. ACT, although frequently very toxic especially when given with high dose interleukin-2 (IL2), occasionally resulted in tumor regression. Rosenberg’s group now report results in a patient with metastatic bile duct carcinoma who had had very meaningful tumor responses to ACT using a subset of TIL (Tumor Infiltrating Lymphocytes) selected to be cytotoxic to cancer cells carrying a mutation, ERBB2, of an ERBB2 interacting protein. Toxicity of ACT in this setting is manageable and the patient’s performance status after therapy is excellent.

So why be excited about Ipilimumab and the admittedly, very early results of the newest iteration of ACT from the NCI group? First, the use of immunotherapy may be able to circumvent inherent chemotherapy resistance. Secondly one can imagine that ACT with specific tumorcidal T-cells might combine very nicely with Ipilimumab like agents which enhance T-cell mediated tumor cell death. Further evaluation of this strategy may warrant further testing.

Maybe, indeed, we are leaving the age of wishful thinking and are closing in on a new age of tumor shrinkage in the immune therapy of cancer.

For more information on the consortia model for drug development in oncology trials, please visit our webpage for AGICC and AMyC.

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A Novel Drug for Treatment of Necrotizing Soft-Tissue Infections

Criterium, Inc., a full-service CRO is proud to be the CRO of record as the supplier of services and support for this important trial. Further study details are provided by the pharmaceutical development sponsor, AtoxBio Ltd. on ClinicalTrials.gov

Clincial Trial Results ReportA complete article detailing the trial and its current progress was highlighted in JAMA’s April 2014 issue in print and online (http://archsurg.jamanetwork.com/article.aspx?articleid=1859986). This is a study to evaluate the safety and pharmacokinetics profile of different doses of AB-103 administered to patients diagnosed with Necrotizing Soft Tissue Infections that are scheduled for an urgent surgical intervention as part of their standard of care. The primary study hypothesis is that AB-103 can be administered safely to the patients presenting with Necrotizing Soft Tissue Infections. Secondary endpoints are efficacy by exploratory descriptive analyses of specific efficacy endpoints from three outcome domains to demonstrate treatment benefit of AB-103 in comparison to placebo in patients with Necrotizing Soft Tissue Infections.

The efficacy domains Lab-Beakersare:

  1. Clinical status domain
  2. Pharmacoeconomics domain
  3. Systemic and local inflammatory biomarker domain

Criterium, with extensive experience in dermatology and infectious disease indications, particularly with wound and burn treatments, provided services for data and project management, clinical and medical monitoring, biostatistics, safety, regulatory support, and medical writing.

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When LESS is MORE in Drug Development

Dr J S Macdonald MDDr. John S. Macdonald
Senior Consultant for The Academic GI Cancer Consultant Consortium (AGICC) & The Academic Myeloma Consortium (AMyC) of The Oncology Consortia of CRITERIUM, INC.

Cancer clinical therapy development is moving beyond the classical clinical research trial paradigm used to develop new oncology treatments. This paradigm was based upon the sequential use of Phase I trials to define toxicity and tolerable doses and schedules. Phase II trials then search for signals of efficacy in particular disease types. Phase III trials complete the paradigm and are designed to test new therapies against the current standards of care. Therapies successfully completing the Phase I, II, and III trials are presented to regulatory agencies for approval as widely available cancer treatments.

Although there will always be a role for some drugs to be developed using the classic Phase I-III Test Tube Glowingmechanism for development, many newer cancer treatments will be targeted not against a cancer type but rather against specific molecular targets that may be identified in cancers arising in various different organs. Therefore it doesn’t make sense to do Phase I-III clinical trials in large number of patients with a histologically defined cancer (lung cancer, breast cancer, renal cancer, for example). Rather one needs to test the efficacy of a targeted therapy in tumors that carry the target of interest.

An example of this scenario is the HER2 target. HER2 was originally identified in breast cancer and a monoclonal antibody targeting HER2 (Herceptin) was developed and shown to be efficacious in HER2+ breast cancer. More recently HER2 was identified in about 20% of stomach cancers. Herceptin was shown to be active in HER2+ gastric cancer but not in the roughly 80% of tumors that were HER2-.

Lab BeakerThe gastric cancer HER2 story illustrates important points about targeted therapy development. First, clinical trials taking all comers with a histologically defined cancer like stomach cancer makes no sense. The patient population chosen must be from the 20% of patients whose stomach cancers express HER2. Also full toxicity evaluations and dose finding studies do not need to be done since we have much of these data from the experience with Herceptin in breast cancer.

The bottom line here is that as we get increasingly more sophisticated in defining molecular targets in cancer, we will be dealing with smaller clinical trials performed in carefully defined patient populations. The molecular biology of the cancer and the targeted therapy will be come increasingly more important in cancer therapy development.

For more information on the consortia model for drug development in oncology trials, please visit our webpage for AGICC and AMyC.

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Quick Takes: the Pharma C-Suite with Ronny Schnel, Exec. Dir., Business Development & Client Services

Ronny Schnel Exec Dir Client Services

Importance of Client Services

When clinical trials project management is paired with effective client services it becomes the framework for the most crucial tasks a Clinical Research Organization (CRO) performs other than the trials it runs. Why is this imperative for executives in the Pharmaceutical development C-suite to focus on this?

The multi-million dollar price tags of the projects undertaken in the competitive environment of pharmaceuticals and drug development dictate the need for a strong liaison between the project management team and their customers. In addition, every CRO needs to adopt and maintain a service-oriented mindset focused on business development and sales, in order to be successful.

And not just the sales team ? a customer-centric focus starts with Client Services, but all employees who have contact with a customer need to keep this in mind:

It’s all About the Customers

Clinical Research Organizations who acknowledge the importance of making initial contact with valuable customers, keeping them as satisfied customers, encouraging them to return, and requesting referrals reap the benefits of a focused client services program. To accomplish this goal, a CRO must create strategic client services policies and procedures. Some of these might include the following:

  • Find and connect with the customers: Every CRO should maintain a database of contacts derived from networks, conferences and personal contacts.
  • Communicate with customers through quality content: It is of the utmost importance in order to attract the level of customers to compete in a tough pharmaceutical market. With today’s technology, digital content is always in front of consumers and it creates leverage for a CRO over competitors NOT engaged in digital content marketing, as well as help build long-term customer relationships.
  • Listen to needs and wants of customers: A recent large C-Suite study[1] involving over 23,000 executives revealed three major insights indicating that every CRO must: (1) acknowledge and act on customer influence and input; (2) implement digital strategies that connect with the customer base; and (3) improve the overall customer experience.?Client Services taking care of customers
  • Give customers reasons to remain: If a CRO remains in touch with the expressed needs, wants, and input from customers, those customers will remain loyal to the CRO and create repeat business — the key goal in the client services arena.
  • Request referrals from satisfied customers: It’s the same in nearly any business: keep customers satisfied, and they will provide word-of-mouth advertising and referrals in return.

Consider This

It’s simple, really: Consumers become customers of a company or organization because they want or need a product or service; they remain customers and refer others as long as that company or organization keeps them satisfied. If a CRO fails to satisfy its customers, they are likely to go elsewhere, just like any customer would. This is why every CRO needs to have effective client services in place as that bridge between the clinical trials and project management teams and their valued sponsors and customers.

[1] Source: http://www-935.ibm.com/services/us/en/c-suite/csuitestudy2013/

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How does Criterium Rescue a study experiencing recruitment obstacles?

Global Clinical TrialsOne of the major requirements for a clinical trial to be successful involves the recruitment of an adequate number of participants so that?accurate scientific conclusions can be made. “Adequate” means that an appropriate sample size has been calculated such that even when some drop out of the study, as is commonplace in clinical research, the remaining enrollment will be adequate to allow meaningful evaluation of data.

A problem arises when a study is in progress, but too many participants leave the program so that the remaining number is too small to provide the required amount of data to provide a statistically significant result. It is our experience that this occurrence often leaves the Sponsor in a position where they begin to seek alternative options in managing the study.

Analyzing the fall-off to get back on track

As a CRO we are experienced clinical trial project management specialists. We know how to turn a slow recruiting or high dropout study into one that fulfills the requirements of the statistical analysis plan. We return the study to a viable and productive state using the most cost effective methods. We analyze recruitment and retention plans and identify the problems.

Once identified, we provide suggestions and an implementation plan for how they can be resolved. Our step-by-step approach includes the following:

1) An intense review of the study with the study team. We develop a thorough understanding of all aspects of the clinical trial including the recruitment strategy that was implemented to understand the shortfalls.

2) We gather as much information as possible in order to identify what the recruitment and retention problems might be caused by through the conduct of the following types of field interviews.

  • People working at sites that are successful and sites that are struggling
  • Other identified knowledgeable sources that are not actively involved in the study but may provide valuable input,
  • Physicians and community groups who have the potential to provide referrals

3) We analyze the data we have collected in order to identify the factors that are responsible for recruitment and retention obstacles.

4) We provide you with a detailed report of our findings and the most cost effective solution(s).

5) We meet with you in person, whenever possible, to review, discuss and brainstorm ensuring that we rank the obstacles from most to least critical.

In this way, we prioritize the solutions — especially when dealing with budgetary constraints. Clinical Trial RescueBy gaining important feedback, using our own experience and by working together through a team approach, our clinical trials project management team is able to provide you with a complete understanding of the recruitment and retention obstacles faced by your study.

Our experience and confidence in this approach allows you to take immediate action to get your study back on track towards meaningful and significant results.

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The Value of Developing Generic Drugs

Generic drugs have the same chemical composition and non-active components as their brand name counterparts. Generics are prescribed in the same dosages, are administered in the same forms and act the same ways in the body. The difference to the consumer is the price. Generic drugs usually cost much less than the brand name product.

Generic Drugs
Generic Drugs are produced for all types of indications

Pharmaceutical drug development costs for generics are much lower than for brand name drugs because it is not necessary for them to conduct the extensive studies required of new medicines; they only asses the bioequivalence of their generic product to the brand name product. Producers of generic drugs still make a profit even though they sell their drugs at a much lower price than the brand name product.

Application for pharmaceutical drug development of generics is easier and less costly than for a new drug

When the patent for a brand name drug expires, other pharmaceutical manufacturers may develop their own generic forms of that drug. FDA approval is still required for the production and sale of generics, but this approval is through an Abbreviated New Drug Application, ANDA. It is a much quicker application process as it has has fewer requirements than does an application for a new brand name drug. Thus, the ANDA is faster to review and approve by the office of Generic Drugs.

Here are a few reasons why pharmaceutical drug development for generics is easier and less costly than in the development of new drugs:

  1. The chemical composition of the drug has already been established by the brand name company so there are lower costs incurred for research and development.
  2. Generic companies need only show bioequivalence. They are not required to conduct costly clinical trials to prove the safety of their generic drug or that the drug works in the way it is intended to work. These trials were conducted by the brand name company.
  3. Companies producing generics must prove their product is biologically equivalent to and performs in the same way as the brand name drug. This means the generic version has the same active ingredients delivered into a patient?s blood stream in the exact same manner and the exact same time as does the brand name drug.
  4. The generic drug must not look like the brand name drug. For example, it must have a different color even though its chemical composition must be the same.
  5. When the application is approved, the cost to the generic pharmaceutical company is limited to costs of producing and distributing the generic drug.

Other advantages of pharmaceutical drug development of generics

Generic Tablets in Blister Pack
Generic Tablets in Blister Pack

FDA approval of an application for production of a generic drug signifies that the drug has met the same rigid standards required of the brand name drug manufacturer. The FDA approval of the ANDA signifies the generic has been shown in limited clinical studies to be as safe as the brand name drug and works in exactly the same way.

Pharmaceutical companies that manufacture generics actually receive the benefit of the previous marketing campaigns of the brand name company. The public is familiar with the drug and is comfortable using it. When competition is presented by multiple companies now offering the same drug in just a cheaper version with a generic name, consumers are more likely to demand the less expensive drug.

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Global Clinical Research – Israel’s Development Environment

As clinical research is an essential step for pharmaceutical companies, Israel has become a hub for clinical research opportunities, and is becoming known as an excellent arena in which to initiate trials. Israel has made significant theoretical and practical contributions to the biotechnology revolution and has developed an advanced infrastructure of medical and paramedical research as well as bioengineering capabilities. Biotechnology, biomedical, and clinical research account for over half of all scientific publications. The country’s industrial sector has increased its activities in the medical field to capitalize on its extensive knowledge base. All research resources are provided to local pharmaceutical companies and related institutions.

Israel Pharma Development
Globally competitive with excellent resources and talent

From the Global Competitiveness Report 2012-2013:
Israel is rated 1st in R&D expenditure as % of GDP 1
Israel is rated 1st in quality of scientific research institutions 2
The country’s main strengths remain its world-class capacity for innovation (3rd), which rests on highly innovative businesses that benefit from the presence of the world’s best research institutions geared toward the needs of the business sector. Israel’s excellent innovation capacity, which is supported by the government’s public procurement policies, is reflected in the country’s high number of patents (4th). Its favorable financial environment, particularly evident in the ease of access to venture capital (3rd), has contributed to making Israel an innovation powerhouse.

Knowledge and Skill
What sets Israel apart from other potential options? One of the major reasons associated with Israel becoming an excellent research option relates to its knowledgeable workforce. The number of doctors and/or scientists per capita is high within Israel. Global clinical research performed within the nation succeeds because of the proficient talent on hand. These doctors and scientists include some of the finest minds in the world. 6 Israelis in the last 10 years have been awarded Nobel Prizes in the fields of Economics or Chemistry.

One of the fastest growing technology businesses in Israel is life sciences, with over 1,000 companies exporting over $6 billion in pharmaceuticals and medical devices. One well-known Israeli invention is Given Imaging‘s Pillcam, which allows physicians to examine a patient’s gastrointestinal tract with a swallowable camera. The generic pharmaceuticals giant Teva has played an important role in lowering the price of many drugs, as well as bringing to market novel drugs such as Copaxone for multiple sclerosis.

Israel provides an excellent Pharma development environment
Israel provides an excellent Pharma development environment

FDA Recognition
Why is Israel an outstanding option for your pharmaceutical development? It may have something to do with the nation being recognized by the FDA for all related clinical trials and associated research. FDA clinical trials can be launched immediately in Israel. International companies are enthusiastic to join Israel’s rise as a place for global clinical research. World renowned companies are launching ventures to produce high-quality testing facilities within Israeli cities. All research and manufacturing takes place within these facilities. Interested companies have access to their well-regarded doctors aiming at progress and innovation.

Efficiency Creates Quality
Clinical research is successful when there is an organized, efficient set up to help support it’s functioning. Israel is a cohesive nation that is controlled under one national health program. This enables access to all citizens in the form of their identity cards. All clinical trials launched and established in Israel get off the ground quickly in comparison to other nations around the world. Pharmaceutical, Biotechnology and Medical Device companies are typically approached by Israel to conduct clinical trials within the nation for clinical trials ranging from cardiovascular diseases to neurodegenerative disease. International companies are able to run thorough, professional research trials in an organized manner. This efficiency is important for companies aiming to deliver top-quality results in a short period.

Sources:
OECD Science, Technology and Industry Scoreboard 2011.
Israel’s Ministry of Foreign Affairs (www.mfa.gov.il)
1 International Institute for Management Development (IMD) Global Competitiveness Yearbook 2011.
2 World Economic Forum (WEF) Global Competitiveness Report 2012-2013.
Top photo of Jerusalem by Alex Bruda

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The FSP: Functional Service Provider in Pharmaceutical Research

A Functional Service Provider (FSP) offers a variety of clinical trial services on an “a-la-carte” type basis – as opposed to a full suite of services. This allows large Pharmaceutical companies to outsource only what they need to fit their budget (and other factors).

This blog article discusses how a COLLABORATIVE APPROACH to understanding the clients? needs and GOOD LISTENING SKILLS on the part of the service provider, make offering a partial selection of services (as opposed a full suite of services) a practical and cost effective approach to pharmaceutical drug development in this economic climate.

FSP Group Conference
FSP’s are able to plug-in to all parts of a study; Criterium’s a-la-carte menu of services offers sponsors the ability to get expert personnel for just what is needed.

As more and more pharmaceutical companies are outsourcing the management of clinical trials to Functional Service Providers (FSPs) there is a need for constant and competent communication between the Sponsor and the FSP?s who are providing specific clinical trial services.? This is not something to take for granted as communication will be the key to a successful project. The pharmaceutical company owns the project, while the FSP manages some aspect of it. Although a solid contract that is legally binding is always negotiated between the pharmaceutical company and the FSP, both entities will find that a good working relationship depends on open communication and collaboration.

Collaboration is a Must
Collaboration simply means working together to create something new. There are many different ways that the management of clinical trials can be outsourced to FSPs, but all require collaboration between at least two entities. The involved companies must have a shared plan as well as the right people and tools to work on the project.

When pharmaceutical companies outsource clinical trials, even though they still own the project, they must work together with the FSP. In fact, a pharmaceutical company may outsource different parts of a project to different FSPs. In order to create a satisfactory end product, they must all collaborate and set up parameters to delineate which entity is responsible for which tasks. In addition to emails, telephone calls and regular reporting, face-to-face meetings must be part of the collaboration process.

Need for Clear and Constant Communications
Collaboration requires clear communication. Just some aspects of the FSP agreement that must be communicated include:

  • The agreement may include the temporary transfer of certain employees knowledgeable about the project to temporarily work with the FSP. This limits the need for new workers to spend time and money to familiarize themselves with the project. When this happens, clear communication with the employees is imperative so they know who is in charge and what the expectations are for everyone on the project. This will relieve the FSP of potential personnel problems and concerns.
  • Plan in advance for contingencies. Anticipate potential problems during the management of clinical trials and establish protocol as to how those problems will be solved. For example, what happens if there is a lull in the workload at the FSP due to a glitch at the pharmaceutical company?
  • The FSP and pharmaceutical company need to agree on the vision for the project to be sure they are both on the same page headed for the same goal.
  • If the project is confidential, this needs to be clearly communicated to the FSP and all employees who will be working on the project.

Need for listening skills
As basic as it seems, communication and collaboration involve listening. When one entity owns the project and the other entity is the chosen vendor for outsourcing, conflict is not uncommon. Experience has shown that while the formal contract outlines the responsibilities of the entities and governs who has the authority, the key to the success of the project is the ongoing communication and the willingness of each entity to listen to the concerns of the other. It is this active communicative relationship that guarantees the ultimate success of the project.

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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 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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