Rare-Disease Researchers Could Teach Drug Makers Important Lessons

March 28th, 2014

In a recent column in Medical Marketing & Media, the founder and president of a digital relationship marketing firm with a long history in assisting those searching for remedies for rare diseases offers advice on how pharmaceutical companies could profit from the experience of those working on so-called “orphan” drugs.

Siren Interactive president and founder Wendy White notes fully a third of the novel drugs approved by the Food and Drug Administration last year are orphan drugs, treating rare diseases which each affect no more than 200,000 people in the U.S. The almost 7,000 rare diseases, however, collectively affect about 10% of the U.S. population. Since 80% of the conditions are genetic in origin, they are very likely to affect children. Only about 5% have an FDA-approved therapy.
A frequent author and speaker on rare-drug issues, White begins by noting that fully a third of the new drugs approved by the Food and Drug Administration last year were for rare remedies, and more are in the pipeline for expedited review.

In fact, two of the nine novel orphan drugs cleared by the FDA in 2013 practically set speed records: Genentech’s chronic lymphocytic leukemia drug Gazyva won approval in just a little over six months, and Janssen’s mantle cell lymphoma drug Imbruvica cleared the agency in just four-and-a-half months.

That record of success is partly due, White notes, to government incentives for rare-drug remedies, and to their profitability (in a market estimated to hit $127 billion by 2018, their return on investment is 1.7% higher than for conventional drugs, White notes, citing a 2013 report.) But, she adds, credit should also go to lessons those drug makers have learned that the overall pharmaceutical industry would do well to heed.

White points to five lessons learned that have benefitted orphan drug makers. First, she notes, large drug firms sometimes make the mistake of viewing their operations as compartmentalized, seeing clinical trials as separate from their manufacturing, marketing and other operations.

But rare drug firms, on the other hand, have discovered that, in White’s words, “brand building begins long before there is a brand.” So it’s crucially important for pharma firms to engage with affected communities early on, while planning and running clinical trials. What they do there in areas such as sharing data, providing drugs for compassionate use, and speedily reimbursing trial participants will do much to impair or enhance their reputation.

Orphan drug makers have also learned the importance of using social media in reaching close-knit rare drug communities. By failing to reach out through these widely shared media to tell their story, uncommunicative drug firms virtually guarantee someone else will tell it for them.

To gain maximum support, rare drug makers have also become expert in providing personalized support to those affected by the diseases they research. Leveraging their information and resources, they get involved in creating collaborative ways to educate the community and the public, and to help deliver services. By extending their view beyond the laboratory, such efforts pay dividends by building trust and cooperation, especially in times of trouble (as an example, she cites Genzyme’s active involvement with patient communities as helping the company weather some past supply problems).

Finally, White adds, rare drug companies recognize that patients look for innovations that improve their care and quality of life, and working together with patients, support groups and other stakeholders is the most effective way to achieve those outcomes.

E2E Method Urges Closer Links between Efficacy and Effectiveness Clinical Trials

March 24th, 2014

A new study urges greater integration of two types of clinical trials to ensure they are better coordinated and closer in time. Published online by the journal Nature Clinical Pharmacology & Therapeutics, the article “A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials” proposes earlier, closer integration of clinical drug trials for efficacy with those for effectiveness.

The E2E clinical trial design model proposed in the paper would more closely coordinate clinical trials for drug efficacy, which test a fairly homogeneous group of patients to determine whether a drug in development has a treatment effect under optimal conditions, with subsequent clinical tests for drug effectiveness, which aim to gauge whether observed treatment effects persist when the drug is used on a broader group of patients under protocols more closely resembling ordinary clinical treatments and standards.

The E2E model calls for using results from efficacy trials to design effectiveness trials, and to identify and test how clinical aspects and treatment effects may vary in typical care settings. This more seamless approach, the paper claims, would improve understanding of drug treatments, hold down start-up costs, and not delay regulatory review and approval.

The present practice of initial efficacy and later effectiveness trials, often done years apart, if effectiveness tests are done at all, means new drug introductions are frequently based on the results from optimized patient groups, tested under ideal conditions, without data on results from testing on larger, more diverse patient groups or under typical conditions. Once a new drug has been licensed based on efficacy test data, drug makers often hesitate to conduct effectiveness tests. Even if post-approval effectiveness trials are done, they may be without involvement of those who designed and ran the earlier efficacy trials, resulting in what the paper describes as an “uncertain and disjointed process.”

Besides enhancing medical benefits, the E2E advocates claim, better coordination could help make new treatments available earlier, since lack of data on the effectiveness and economic value of new proposed treatments in typical care settings often leads payers to deny coverage, restrict uses and increase co-payments.

While proposing the E2E model as likely to benefit both the public (by streamlining clinical trials and better identifying potential risks and benefits when the drug is used more widely) and to producers (by providing fuller, more timely data to support applications for marketing approval), the study recognizes a number of unanswered questions in its approach. Among these are: who will have the power to compel effectiveness trials for an already-approved drug, and who will pay; who would own the safety, efficacy and effectiveness of drugs already in use; and whether intellectual property rights might need revision.

Even so, say the study’s authors, a pre-planned, cost-effective adoption of the proposed E2E model for clinical trials hold promise for “improving the evidentiary basis for adopting treatments in practice, accelerating understandings of treatment effectiveness in subgroups with particular clinical features, and providing a framework for incorporating this information into the regulatory process.”

The paper was prepared by a team led by Dr. Henry Selker, dean of the Clinical and Translational Science Institute at Tufts University, and included an interdisciplinary group of researchers from MIT, the Harvard School of Public Health, other academics, several pharmaceutical firms, the FDA and the European Medicines Agency. The research project was supported by grants from the National Institutes of Health.

A veteran researcher who has served as president of the Society for Clinical and Translational Science and the Society for General Internal Medicine, Dr. Selker is well-known for studies on factors affecting outcomes in emergency care of cardiac patients, and in developing mathematical models that can be embedded into computerized electrocardiographs to aid clinical predictions and emergency treatments. He also advised legislators working on the Patient Protection and Affordable Care Act, and last year authored a book on the new law, incorporating the views of many of its principal drafters of its history and impact, and identifying further research areas.

What are the symptoms of Irritable Bowel Syndrome, also known as IBS? How do you know if you have IBS? Below is a list of common symptoms -

January 31st, 2013

Doctors often use a list of symptoms, known as the Rome III criteria, which was developed by an expert panel to distinguish IBS from other intestinal conditions. Please note that you do not have to have all of the symptoms listed below to have a diagnosis of IBS.
Here is the list of criteria:
- Your symptoms began at least 6 months ago.
- You have had abdominal pain or discomfort at least 3 days each month in the last 3 months and at least two of the following statements are true:
• The pain is relieved by having a bowel movement.
• The pain is linked to a change in how often you have a bowel movement.
• The pain is linked to a change in the appearance or consistency of your stool.
When you have IBS, your pattern of bowel movements may be different over time. Two or more of the following may happen:
• Bowel movements may occur either more often resulting in diarrhea, or less often resulting in constipation, than usual, such as having more than 3 bowel movements a day or less than 3 a week.
• Bowel movements may differ in size or consistency (may be hard and small, pencil-thin, or loose and watery).
• The way stools pass changes. You may strain, feel an urgent need to have a bowel movement, or feel that you haven’t completely passed a stool.
• You may have bloating or a feeling of gas in the intestines.

IBS is a functional gastrointestinal (GI) disorder, meaning it is a problem caused by changes in how the GI tract works. It is a common condition in women, estimated to affect 30 million women in the U.S. IBS is not a disease; it is a group of symptoms that occur together, but the GI tract does not become damaged. Some people with IBS have mostly diarrhea (IBS-D), others have mostly constipation (IBS-C), and some have a mix of the two (IBS-M). In patients with IBS-D, common symptoms include diarrhea, frequent bowel movements, and a strong urgency before bowel movements, along with abdominal pain or discomfort.

IBS – Irritable Bowel Syndrome Clinical Trials

November 21st, 2012

Irritable Bowel Syndrome Clinical Trials

Do You Suffer from Restless Legs?

June 6th, 2012

Do You Suffer from Restless Legs – does the discomfort you feel in your legs increase at night?

Many people do not even know they have Restless Legs Syndrome, also known as RLS, which is a disruptive neurologic disorder that seriously affects 2-3% of the adult population. RLS results in an irresistible urge to move the legs to relieve unusual or unpleasant sensations in the legs that may be described as creeping, tugging, or pulling. Because RLS most often occurs in the evening, it can severely disrupt sleep and reduce quality of life.

RLS, is a medical condition that can be treated. Several approved treatments exist for RLS, but despite their proven efficacy, not all RLS patients can take the currently approved drugs. Therefore, there is a need to develop new treatments for RLS.

This study is testing the safety and efficacy of an investigational drug in individuals suffering from moderate to severe RLS.

If you suffer from RLS, consider participating in this research trial. Study-related office visits, laboratory work, and medication are at no cost and you may be compensated for your time and travel.

Contact us today: 1-877-997-8839 or Visit ClinicalTrialSpotlight.com

Trivalent Vaccine for Sarcoma

March 15th, 2012

MabVax Therapeutics is actively recruiting patients for a Phase 2 sarcoma vaccine trial who have recently been treated for metastatic sarcoma and whose status is “No Evidence of Disease” or NED.

The patients in the study will be randomized to receive either a vaccine that is combined with an immune system stimulant or the immune system stimulant alone. The immune system stimulant is called OPT-821 and is an immunological booster. If the trivalent vaccine can stimulate the patient’s immune system to develop antibodies which recognize and target the GM2, GD2 and GM3 sugars that are over expressed on the surface of most sarcomas, then the patient’s antibodies could attack and kill any remaining sarcoma cells potentially preventing the recurrence of sarcoma.

For more information about the trial and to take a short questionnaire to see if you qualify click here.

If You Have SARCOMA…

February 17th, 2012

You may be eligible to participate in this clinicle trial for individuals recentrly diagnosed with metastatic (Stage IV) sarcoma or previously diagnosed and undergone surgery to remove all known sarcoma tumors.


This clinical trial is testing a novel vaccine desinged to instruct the immune system to destroy any sarcoma cells that remain in the body after surgery.

This vaccine is given as an outpatient treatment with the goal of preventing or delaying sarcoma recurrence.

Participants may receive, and no cost, study-related:

  • Medical care
  • Study vaccine
  • Laboratory work and evaluations

Participants must be within 8 weeks of their surgery and meet other eligibility requirements, agree to undergo the study required procedures and after a thorough discussion of the study, give written consent to participate in the study.

See if you qualify here

Is your Parkinson’s medication wearing off?

January 31st, 2012
Announcing Parkinson’s Clinical Trial

For Individuals Experiencing End of Dose Wearing Off

This trial is to evaluate the safety and efficacy of the investigational product tozadenant as adjunctive therapy in levodopa-treated patients with end of dose wearing off.

Parkinson's Disease Clinical Trials

Tozadenant has a novel mechanism of action which, if proven safe and efficacious and approved by the FDA, could represent the first new treatment modality for Parkinson’s disease in more than 20 years.

Participants must:

  • Be between the ages of 30 & 80
  • Have a good response to levodopa and be taking at least 4 doses of a levodopa containing medication per day
  • Be taking levodopa continuously for at least the previous 12 months and experiencing some daily (24-hour clock) OFF time

Study-related office visits, laboratory work and medication are at
no cost.

To learn more about this trial and see if you qualify to participate, Click Here

Parkinson’s Disease: The Wearing-Off Effect

January 23rd, 2012

Medications for Parkinson’s disease work very well. But symptoms can creep back when they wear off.  This is known as “The Wearing-Off Effect.”

Over the course of Parkinson’s Disease (PD), a substantial number of patients develop a wearing-off effect which refers to the fact that the benefits you receive from your dopaminergic medications wear off before you are due to receive your next dose. This means you may go for a period of time with relatively uncontrolled motor symptoms.

If your Parkinson’s medication is wearing off, consider participating in a clinical trial.  Visit www.clinicaltrialspotlight.com to learn about ongoing clinical trials for Parkinson’s.

Are you considering participation in a clinical trial as a treatment option for advanced prostate cancer?

September 21st, 2010

If so, here is information on a currently-enrolling research study designed to evaluate an investigational immunotherapy (ipilimumab) for advanced prostate cancer.

Why is it important to continue research for new treatment options for Advanced Prostate Cancer?
The American Cancer Society estimates that 32,050 men will die of advanced prostate cancer in 2010.  Prostate cancer is the second leading cause of cancer death in American men, behind only lung cancer, and accounts for about 11% of cancer-related deaths in men. Men of African or African-American descent are twice as likely to be diagnosed with prostate cancer as men of European descent, and are often diagnosed with advanced prostate cancer. These statistics indicate that we must continue to look for alternative treatment options for patients with advanced prostate cancer.
Clinical trials in advanced prostate cancer are an important means of discovering new treatment options. In 2004, the results of two Phase 3 studies (TAX327 and SWOG S9916) established docetaxel as a new standard for first-line therapy in patients with hormone refractory prostate cancer (HRPC). Docetaxel remained the only approved treatment option in HRPC until June of this year when cabazitaxel was approved for patients that had failed docetaxel. Once again this approval was based on Phase 3 clinical trial data (TROPIC). Both docetaxel and cabazitaxel were approved based on ~ 3 month increased overall survival in their respective patient populations. Although the approval of any drug that increases survival is welcome, clearly there is an ongoing need for clinical trials investigating new drug options using innovative mechanisms of action to fight the battle against advanced prostate cancer.

What is the investigational drug Ipilimumab and how does it work?
Ipilimumab is a member of a class of agents referred to as immunotherapeutics or immunotherapy agents. These agents are designed to use a patient’s own immune system to fight disease. One theory as to how cancer cells continue to grow is that they somehow hide themselves from immune cells or that the patient’s immune response is not robust enough to kill all the cancer cells. The goal of immunotherapy agents is to either boost the immune response or somehow make cancer cells more visible to the immune system so that they can be better targeted and killed.

Ipilimumab is a fully human monoclonal antibody, which means that it binds to a specific target (antigen) within the body.  Specifically, ipilimumab binds to a molecule found on T-cells that is called Cytotoxic T Lymphocyte Antigen 4 (CTLA-4). CTLA-4’s normal role is to act as part of a breaking mechanism that keeps our immune system from over responding. When a foreign antigen is presented to a T-cell, a response is generated so that the body’s immune system attacks and destroys the foreign cells. In order to keep the immune system from generating too much of a response, which could lead to immune cells attacking non-foreign or “self” cells, CTLA-4 works in conjunction with other molecules to put a “brake” on the immune response. By binding to CTLA-4, ipilimumab removes this “brake”, potentially leading to a more robust immune response. The clinical trial described below, sponsored by Bristol-Myers Squibb (BMS CA184043), is designed to determine if bone-directed radiotherapy combined with ipilimumab treatment leads to an increase in survival benefit for patients with metastatic prostate cancer that have received prior docetaxel.

How do I get more information on this Research Study?
About 800 patients throughout the world, approximately 300 in the United States, are expected to participate in this study. Participants may be in the study for 1 year or more, depending on how they respond to the study therapy. The study is currently enrolling in the US and globally, with enrollment expected to continue until July 2011.
Participants in this study may receive, at no cost, study-related: medical care, study medication, laboratory work and evaluations.
Medical insurance is not necessary to participate in this study, and study-related travel/parking may be reimbursed.

To participate, you must:
have a diagnosis of prostate cancer that has metastasized to the bone
have previously undergone at least 1 chemotherapy regimen containing docetaxel (Taxotere)
A study physician will discuss complete study criteria with you

For more information about this study including the location of study centers across the country, visit www.clinicaltrialspotlight.com or clinicaltrials.gov and search under NCT00861614, then speak with your physician to discuss if participating in this study might be of benefit to you.

Prostate Cancer Clinical Trial Locations

Prostate Cancer Alternative Treatment