As more companies try to ensure that their validation activities are compliant and cost-effective, it has become increasingly important for quality professionals and validation technicians to manage costs and reduce downtime by accurately evaluating their validation equipment needs. This is particularly the case in validation/mapping studies. Pharmaceutical Technology will hold a live webcast, “Optimizing Validation/Mapping Studies for GMP Compliance,”  on Wednesday April 10th at 11:00 AM EST to address this issue. Read more »

Pharmaceutical Technology interviewed some of the speakers for the upcoming INTERPHEX 2013 conference to get a “sneak peek” of their presentations. The first of these, an interview with Par Almhem, president of ModWave and Modular Partners, discusses advances in modularization. Check out the media player on the PharmTech website or click here to listen to the podcast, and look for more to come soon. If you’re at the show, be sure to stop by PharmTech’s booth (Booth #4139).

In recent years, large pharmaceutical companies have launched a variety of initiatives to restock ailing pipelines and boost business performance including mergers and acquisitions, diversifying business portfolios to non-pharmaceutical products, downsizing, spinoffs, and entering the biopharmaceutical arena.

Whatever the approach, pharmaceutical companies want balanced portfolios with programs at various stages and risk profiles, says Melinda Richter, founder and CEO of San Francisco-based Prescience International in a BioPharm International podcast.

To date, most Big Pharma companies have partnered or acquired assets of biopharmaceutical companies with products in late-stage development, says Richter. However, as the availability of late-stage development opportunities shrink and the landscape becomes more competitive, Big Pharma is turning to more early-stage partnerships with academia and early-stage companies.

It is attractive to for the pharmas to go after early-stage companies because “by nature, they are smaller, they are nimbler, and they are willing to take the risks that the large pharmas just can’t. These small companies have to swing for the fences and they have to win. Pharmas have a lot to protect. They have to be more conservative,“ says Richter.

More scalable innovation opportunities are another part of the story, says Richter.

For example, last year, Merck announced a $90 million, seven-year commitment for the California Institute for Biomedical Research (Calibr), an independent, not-for-profit organization established to accelerate the translation of basic biomedical research to innovative new medicines.

However, for hands-on research, startup companies need laboratory and office space, as well as specialized equipment. Janssen Labs, located on the West Coast Research Center of Janssen Research & Development in La Jolla, Calif., offers short-term leases on wet laboratory and office space.  Tenants also have access to core research facilities and instruments.

The facility, operated by Prescience International, has a “no strings attached” policy. Janssen R&D does not take an equity stake or first right of refusal in the work of tenants, protecting the entrepreneurial rights of startup companies that choose independence.

Janssen Labs and Calibr are two options offered by Big Pharma that will be explored in the session “And Now for Something Completely Different: How Will Pharma Access External Early-Stage Innovation?” at the 2013 BIO International convention on April 23, 2013.

Stem cells are being developed to treat a diverse set of conditions, including spinal cord injury, amyotrophic lateral sclerosis, macular degeneration, Parkinsons disease, and Type I diabetes. But the challenges in moving from the laboratory to the clinic are formidable.  The California-based biotech company, Geron, pioneered clinical trials using embryonic stem cells, with a Phase I trial using oligodendrocyte precursors derived from embryonic stem cells to treat spinal cord injury approved in 2009. In November 2011, however, Geron announced it would be discontinuing all of its stem-cell development work, citing cost and regulatory complexity as factors in that decision.

At the BIO International Convention, a panel will convene to discuss the current landscape for developing stem cell therapies in a breakout session titled Opportunities and Challenges in Developing Innovative Stem Cell Therapies. BioPharm International spoke with one of the session’s presenters, Dr. Armand Keating, Epstein Chair in Cell Therapy and Transplantation, University of Toronto about the challenges facing developers.  According to Keating, funding remains the biggest challenge in moving from laboratory to clinic.  He points out that unlike small-molecule development, stem-cell development is still very much rooted in academia. The traditional sources of funding available to academics are ill-suited or inadequate to fund preclinical validation studies, manufacturing scale-up studies, or the clinical trials themselves. While an organization such as the California Institute for Regenerative Medicine is able to provide some funding   for early-stage clinical trials, later-stage trials will be far too expensive to be carried out at academic research centers, he says.

Clarification of statement in podcast: There have been approximately one million bone marrow transplants performed worldwide since 1959.

INTERPHEX 2013 begins later this month and Pharmaceutical Technology will be previewing the action in the weeks to come.

Look for posts from our Manufacturing Editor Jennifer Markarian, as well as PharmTech’s Editorial Director Rita Peters and Executive Editor Patricia Van Arnum.

And be sure to stop by PharmTech’s booth (Booth #4139) during the show!

Representatives from Merck, Pfizer, and Novartis shared their recent efforts in applying quality-by-design (QbD) concepts to analytical methods, and Todd Cecil from USP explained the related new draft USP chapter in a symposium at Pittcon on Monday, March 18, 2013. The session, “Understanding Analytical Method Variance and the Impact for QbD Filing for Pharmaceutical Products,” was sponsored by the American Chemical Society’s Division of Analytical Chemistry (ACS ANYL) and the American Association of Pharmaceutical Scientist’s  Analysis and Pharmaceutical Quality Section (AAPS APQ). Read more »

The European Commission (EC) has released the draft revision for four chapters of its good manufacturing practice (GMP) guidelines—chapters 3, 5, 6 and 8.  A public consultation was launched earlier this year with comments due on 18 July 2013. The EC stated that these revisions were needed to reflect the latest thinking relating to best practices. Read more »

The UK government has confirmed that from 2014 onwards, the National Institute for Health and Clinical Excellence (NICE), which is the country’s cost-effectiveness watchdog, will play a central role in the new value-based pricing system for assessing the costs and benefits of medicines. Read more »

After less than a year on the job, the head of FDA’s Office of Generic Drugs (OGD) has announced his departure, a sign that all is not well with plans for major organizational changes at the Center for Drug Evaluation and Research (CDER). Read more »

The greatly feared federal budget sequestration mandate went into effect Mar. 1, 2013, and, initially, the impact was fairly muted. The stock market soared, employment rose and government workers continued on their jobs. Federal agencies, including the Food and Drug Administration (FDA), launched initiatives to comply with the mandated 5% across-the-board cut in spending (in reality a 9% cut that exceeds $200 million) to minimize the impact on basic operations. That means curbs on training and staff travel, no new hires and a delay in launching new programs. Read more »