PM360 April 2011

What Do You Think?

Pharma Feedback

You can probably surmise from the title that we want to hear back from you, but let’s start with another question: What do you know about the government’s plan to get involved in drug development? We queried industry experts about this latest development.

Last December an NIH advisory committee recommended establishing a new institute to be called the National Center for Advancing Translational Sciences (NCATS).

The goal of the center would be to catalyze the development and testing of novel diagnostics and therapeutics across a wide range of human diseases by helping to streamline the drug pipeline.

“A fundamental motivation behind NCATS is to look in a systematical way at the whole process of how you go from an idea to a success story,” NIH Director Dr. Francis Collins said during a webinar last month sponsored by FasterCures. “It is going to be focused explicitly on this idea of trying to reengineer the pipeline, identify ways to be more efficient and more prone to success going through. Of course real projects have to be put into the pipeline to test that, so NCATS will be supporting those, but it has a different flavor.”

The NIH is not proposing to become a drug development company or to start competing with the private sector. The goal of each project would be to push it just far enough along to make it commercially attractive for a company in the industry to pick up, according to Dr. Collins.

Another method revolves around gaining access to the hundreds of compounds currently sitting in freezers from research done by private industry, but for whatever reason, never made it to market. Dr. Collins believes this could be a win-win situation since the repurposing of existing compounds could save years of work and $100 million in investment and be financially beneficial to the company that holds intellectual property rights. Dr. Collins plans to meet with high-level pharma and biotech executives this month to discuss how this can be done.

The center is also meant to be a hub for both cross-NIH interactions and cross-sector interactions to encourage collaboration among the private industry, academia, nonprofits, advocacy groups, and even the FDA, according to Dr. Collins.

Although this new center is not set to open until October, its research and work is already being done. That’s because the creation of NCATS is basically a reorganization of NIH programs rather than the formation of completely new programs.

The advisory committee recommended that the following NIH programs be relocated into NCATS:
Molecular Libraries Program and Chemical Genomics Center. This center performs automated high-throughput screening of small molecules and provides biomedical researchers with chemical probes that can be used to better understand how those molecules function in biological systems.

Therapeutics for Rare and Neglected Diseases (TRND) Program. This program is designed to speed the development of new therapeutics for rare and neglected diseases. One example Dr. Collins mentioned in the webinar was a potential treatment for sickle cell disease that was discovered by researchers at VCU and out-licensed to AesRX, a Massachusetts-based biotech company. When the company ran out of money during pre-clinical trials, the product became a pilot for TRND. AesRx is now a partner with TRND and expects to start clinical trials in 12 to 18 months.

NIH Rapid Access to Interventional Development (RAID) Program. This program makes certain vital resources available for the development of new therapeutic agents. Services include production, bulk supply, GMP manufacturing, formulation, development of assays suitable for pharmacokinetic testing, and animal toxicology.

Clinical and Translational Science Awards (CTSA). This program is a national consortium of 55 medical research institutions, which receive $500 million a year from the NIH. While some of the programs are focused on therapeutic development, others are doing community-based participatory research such as determining the effectiveness of telemedicine in rural areas.

NIH-FDA Regulatory Science Initiative. This joint NIH-FDA initiative supports research designed to better inform scientists and regulatory reviewers about product safety as well as improve the evaluation and availability of new medical products. Current projects include research on nanoparticles and the development of a heart-lung model to test the safety and efficacy of drugs.

Cures Acceleration Network (CAN). This program was authorized by last year’s healthcare reform legislation to perform new translational research, but it has yet to receive any funding. The only new money that would go into NCATS is the $100 million set aside for CAN in the president’s 2012 budget, which had not been approved at press time.

The creation of NCATS also means another institute must go because by law the NIH can have only 27 institutes. The plan is for the National Center for Research Resources (NCRR) to be dissolved and for all of its programs to be integrated throughout the NIH. This has created a bit of a controversy within the research community.

“The controversy from the perspective of the national advisory council is that they had a working group that did an evaluation of how best to take on and create this new center but they completely ignored, or weren’t charged to look at, the collateral impact that it would have on the NCRR and the director apparently decided without any evaluation, certainly not with an open evaluation from the community, that it should be eliminated,” said Mark O. Lively, a biochemistry professor at Wake Forest and a member of the advisory board for the NCRR.

A working group was formed after the decision was made to dissolve the NCRR, but only to determine the best way to disperse its various programs. A feedback site (http://feedback.nih.gov/index.php/category/ncats/) was also set up to get the public’s opinion about NCATS and the NCRR. Among the concerns posted were fears about losing funding and complaints about separating NCRR programs into different institutes.

Some Reaction
The new center is also not appreciated by everyone in the industry. “It is a little tone deaf to suggest that the industry is incapable of coming up with this basic stuff and somehow we need someone—who’s never done it—to come in and show us how it’s done,” said an industry R&D expert. He added that the basic science research that has been the foundation of the NIH for years is an important part of the R&D continuum. “It’s a whole long process and what makes this country great is that we spend $31 billion on basic research at the NIH and we need that, we need that understanding of these models and genes and how they work and how disease impacts human beings,” said the R&D expert.

Dr. Collins commented during the webinar that he wanted to make it clear that there are no plans to take funds from other programs that do basic science research to fund NCATS.

Others in private industry as well as the research community are excited about NCATS’s possibilities. For instance, one of its goals is to make progress in rare and neglected diseases by assuming the risk of projects until the point at which companies would be willing to pick them up.
“Anytime there is an investment from the federal government into disease states where there is an unmet need, that complements the investment from industry, then this is win-win for all, especially the patients,” said Simon CS Lemmy, Senior Director Urology Franchise at Endo Pharmaceuticals. He added that oncology is always an area that could use more research, especially bladder cancer, which has had minimal investment over the past few years.

Dr. Ann C. Bonham, the Chief Science Officer for the Association of American Medical Colleges said: “When you think about the volume of new drugs that actually fail in the various phases of clinical trials to the extent that the pipeline can be made efficient and high quality, that’s going to increase the success of those potential drugs making it [all the way] through.”

Dr. Collins ended the webinar by acknowledging that this is not going to be easy. “This is science, so there will be some surprises along the way, but I think the time is right to do something that actually could be fairly historic and might be a major contribution to trying to advance the cause of medical research and to benefit patients who currently don’t have
the kind of interventions that they hope for.”

What’s Your Take?
Now it’s your turn to comment. We'd like to hear what you think and will publish your responses next month, so please include your name, phone number, and email address with your comments. Email us your feedback at editorial@pm360online.com