Download our eBook ‘Supplying global clinical trials: Keys to avoiding costly delays’

Posted under Blog, Catalent Pharma Solutions, clinical trial, clinical trial management, Clinical Trials, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by selvidge

Getting clinical trials right, and right the first time, is crucial for biopharma companies, especially as the cost of getting a drug to market is skyrocketing. Matthew Herper estimates in "The Truly Staggering Cost Of Inventing New Drugs"--which ran in Forbes in February--that drug development costs have reached the eye-watering level of somewhere between $4 billion and $11 billion. R&D returns have therefore fallen drastically and are around half what they were 10 years ago, explains Ian Shott, managing director and principal consultant at Shott Consulting: "Attrition is the overriding driver of cost, with a preclinical success rate of 0.01% and a success rate in the clinic of 10% or less."

The larger part of the cost of clinical-stage drug development comes from clinical trials. According to Timothy Scott, president of the U.S.-based pharmaceutical chemistry development organization, Pharmatek Laboratories, Phase II costs can range from $4,000-$20,000 per patient. So, the last thing a drug developer wants is any kind of interruption of a trial, or to have to start a trial all over again, and this makes the reliable supply of clinical trial material (CTM), whether manufactured in-house or outsourced to a contract manufacturing organization (CMO), absolutely critical.

"An interruption in the provision of the clinical trial material … could require that trial to be conducted over again," Scott says. "And … there is the cost of missed opportunity in the marketplace. If the drug being developed has the potential of being a billion-dollar drug, that is nearly $3 million in lost revenue every day the drug is not on the market."

Clinical trial supply isn't just an issue for investigational drugs. Some clinical trials compare the study drug against a placebo; however, others, particularly those for serious diseases for which it would be unethical to withdraw active treatment, compare with a marketed drug or another investigational drug. Other trials combine an investigational drug with another drug in a combination therapy. It's vital for these trials that there is a constant supply of the comparator or combination drug as well as the investigational drug. Maintaining this kind of reliable clinical trials supply needs a robust supply chain. "This requires complex planning to orchestrate the various elements that need to be brought together to have the right supplies ready in the right place at the right time," says Gerry Hepburn, chief operating officer, vice president and general manager, clinical supply services, Catalent Pharma Solutions. Click here to download the full ebook>> -- Suzanne Elvidge (email)

Bill calls for cancer clinical trials results to be reported–or else

Posted under Blog, clinical trial, Clinical Trials, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Regulatory, Startups, Tom Reed, transparency, Universities, Videos by rmcbride

Fed up with the lack of transparent clinical trial reporting, U.S. Rep. Tom Reed, a Republican from the state of New York, has introduced a bill that adds new teeth to requirements for trial results to be reported. The bill aims to require groups to publish both positive and negative results of cancer clinical studies, as part of a larger effort to find cures.

Drug developers often hide their clinical trial failures, and current rules for reporting results from federally funded research often aren't followed. But if Reed's bill became law, groups that fail to report the results of federally funded cancer studies would have to repay their grants to the Treasury and lose eligibility for future government research support, according to a statement. Good or bad, Reed wants the results published on ClinicalTrials.gov, the U.S. government's online directory of clinical trials.

Advocates for transparency in clinical trial outcomes argue that access to study results could help advance scientific knowledge and lead to faster development of new therapies. Reed's bill focuses on the largest area of drug development today--cancer treatment--and the premise of his bill is that wider sharing of study data could result in cancer cures. While this sounds like an oversimplification of efforts to combat a vastly complex disease, there's no denying that researchers often conduct redundant studies because they don't know about previous results of the same experiments.  

"Public reporting of both positive and negative results will help other research be more effective and less duplicative," Reed said in a statement. "Enforcing the reporting requirement will not only lead to more available cancer research data, but also help every taxpayer dollar spent on research go further toward finding a cure."

- here's Reed's release
- check out Pharmalot's article
- and the report from FDA Law Blog

Related Articles:
Investigators chastise developers for leaving trial data in the dark
Developers should fess up about drug trial failures, researchers say

Fox Trial Finder and You: Use It to Recruit for Your Trials or to Get Involved Yourself!

Posted under Blog, clinical trial, Companies, Diagnostics, drug development, Funding, Health, Health Care, healthcare, Medical Devices, Medical Supply, Parkinson's, Pharmaceuticals, Startups, Universities, Videos by biotechnow@bio.org (Biotechnology Industry Organization)

From The Michael J. Fox Foundation for Parkinson’s Research

The Michael J. Fox Foundation has launched Fox Trial Finder, a new Web tool that uses state-of-the art technology to connect potential study volunteers with the coordinators of clinical studies that need someone with their specific medical history. The goal is to benefit both study sponsors and patients by getting people into clinical trials and studies faster — speeding the development of new treatments and ultimately a cure for Parkinson’s disease.

“This is an example of how technology can enable us to bring new strategies to address a long-standing problem,” says Deborah W. Brooks, co-founder and executive vice chairman of The Michael J. Fox Foundation for Parkinson’s Research.

Use It to Recruit for Your Trials

Fox Trial Finder enables faster and more efficient connections between willing volunteers and trial teams. Registered volunteers and coordinators, research assistants and investigators can review potential matches and connect directly – online and anonymously – to explore next steps. Fox Trial Finder is open to all recruiting PD trials and observational studies with regulatory approval. The site currently operates in the United States, UK, Canada and Australia. Expansion to additional international locations is expected later this year.

-Volunteer matches are customized for your trial: Fox Trial Finder compares key clinical trial eligibility criteria (such as current and past medication use, location, and Hoehn and Yahr stage) to information provided by patient and control volunteers to suggest possible matches.

-Two-way messaging with de-identified volunteers: Volunteers, who appear as an anonymous profile, can message directly with trial teams recruiting for the nearest site of a trial matches. Trial teams can also initiate communication with volunteers on the site.

-Advanced search enables tailored results based on a trial protocol: In addition to trial matches suggested based on a standard match algorithm, trial teams can search the FTF volunteer database based on specific criteria.

-Ongoing email alerts: An alert system notifies users via email of new matches and messages received on Fox Trial Finder.

Contact Tara Guastella at tguastella@michaeljfox.org to start recruiting for your trial with Fox Trial Finder.

Use It to Get Involved Yourself!

MJFF’s Brooks has not only been instrumental in conceiving, structuring and fundraising for the launch of Fox Trial Finder; she also is putting her money where her mouth is by volunteering for a clinical study herself. She is a control participant in the Parkinson’s Progression Markers Initiative (PPMI), the MJFF-sponsored observational study seeking biomarkers of Parkinson’s disease progression.

This is the first time Brooks has volunteered for clinical research, and she is blogging about her experience. In her first video entry, posted on the eve of her PPMI screening visit, she reflects on her expectations for the coming day, and the journey ahead:

“As an observer and someone who professionally is sitting around the table and thinking about how to get to these answers and do what we can to make clinical research more available and relevant for patients, it’s just not enough for me to sit back and think about what other people can do. I decided I actually should do it myself; I wanted to learn firsthand what participation in a clinical trial would be all about.”

While Debi’s story is unusual, it’s not unique. Others in the MJFF family have similarly been inspired to get involved in the search for a cure. Chris Coffey, PhD, heads the PPMI Statistics Core at the University of Iowa. In fall 2011, he decided to run a marathon to raise funds for Team Fox, MJFF’s grassroots community network raising funds and awareness for the Foundation’s research programs. He writes:

“Like a lot of researchers, I’ve been inspired by The Michael J. Fox Foundation. I knew early on that I wanted to go beyond my scientific relationship with MJFF — I decided to run a marathon, something I’d never done before, to raise funds as part of Team Fox.   Reading through the inspirational stories from other Team Fox members, I get the sense that I’m not the typical participant. Many have a personal connection to the disease.  Although I know people who have had PD, I don’t have an immediate family member with the disease, nor have I ever been a caregiver for someone with PD.

“I ran the marathon to honor the individuals who are volunteering as control participants in PPMI.  It takes a special kind of courage to dedicate oneself to the commitment required of this study when one doesn’t have the disease oneself.”

However you choose to get involved, you’ll be helping speed a cure for Parkinson’s disease. Visit www.michaeljfox.org or www.foxtrialfinder.org to learn more and take action today.

Roche headlines autism alliance

Posted under autism, Blog, clinical trial, Companies, Diagnostics, Eli Lilly, Fragile X, Funding, Janssen, Medical Devices, Medical Supply, Pfizer, Pharmaceuticals, Roche, Servier, Startups, Universities, Videos by Mark Hollmer

A European research effort led by Roche ($RHHBY) and others to tackle autism spectrum disorders nailed down $38.7 million in financing for what will be a massive, 5-year project seeking to come up with new diagnostic and treatment options.

According to Genetic Engineering News, Eli Lilly ($LLY), Servier, Janssen Pharmaceutica and Pfizer ($PFE) are among other bio and pharma partners in the substantial collaboration. But Roche is leading the drug-company side of things. King's College London in the U.K. and the non-profit organization Autism Speak are the other head partners. Add in about 14 additional "scientific centers," and you have lots of parties working on autism research.

Roche's involvement is interesting because it has a relatively targeted focus on Alzheimer's. The Swiss drugmaker launched a Phase II study two years ago of a drug to treat Fragile X syndrome, a rare genetic condition thought to be among the most common causes of autism. But the company has more drugs in the pipeline, by comparison, for neurological diseases such as Alzheimer's and schizophrenia, according to its web site.

The collaboration will focus on developing and testing new autism treatments, according to the story. But the consortium also plans to pick and launch multiple autism clinical trial sites throughout Europe, pursue a number of lab and animal tests and identify genetic or proteomic biomarkers to aid in more precise diagnoses and treatments. They face the challenging reality, however, that autism remains hard both to diagnose and to successfully treat.

This massive partnership even has a name: European Autism Interventions--A Multicenter Study for Developing New Medicines, or EU--AIMS, for short. The European Union's Seventh Framework Program, Autism Speaks and the members of the European Federation of Pharmaceutical Industries and Association funded the program, according to the story.

- here's the Genetic Engineering News story
- read the release

Related Articles:
NIH buddies with Afraxis to escort Fragile X program through "valley of death"
Roche begins Ph II Fragile X studies