Pipeline Depends on Public-Private Partnerships

WASHINGTON — The National Institutes of Health needs to partner more with the pharmaceutical industry in order to create a better pipeline for new drugs, Dr. Francis Collins said at the annual meeting of the Endocrine Society.

Dr. Collins, the former head of the National Human Genome Research Institute who at press time was rumored to be President Obama's pick for NIH director, said that with all the genomic research developments, “pharmaceutical companies are a little overwhelmed about where to start” when it comes to figuring out which genes would make good targets for drug therapy.

“Academic investigators should get more intentionally involved in the translational process of going from basic research to drug development,” Dr. Collins said. “There is an opportunity now, more than ever, to bring together academic investigators and the private sector to put together a really exciting version of a drug development pipeline.” Such a collaboration “involves more of academics taking the front-end risk of developing promising compounds so they become attractive and licensable by the private sector.”

Dr. Collins noted that many academic researchers are identifying promising targets for drugs, “but relatively few are taking that information and turning it into an assay … to see if there is something promising that might turn out to become a therapeutic.”

Some targets start out looking promising, but when they get to a point where they need support for preclinical development, “that's where things often die,” he said. “Congress just a few months ago put $24 million into the fiscal year 2009 budget to start this process in an NIH-funded way, and I hope the money will go up substantially in the next 5 years.”

With such a pipeline, conflicts of interest on the part of pharmaceutical companies “would have to be factored in,” Dr. Collins said in an interview. Drugmakers' interest in commercialization would be a factor. “You want to start a project that is going to get somewhere,” he said. “But there are companies across the board that are interested in almost any disease—even very rare ones—as long as it won't cost a fortune to get that drug approved. For the rare diseases, you may have to push things further down the pipeline with public money before the company says, 'Okay, I'll start with that one now,' but I don't know that that should discourage consideration of working on even a very rare disease in this pipeline.”

During a question-and-answer session, Dr. Collins was asked whether he would list his priorities for NIH “as a private citizen,” since he couldn't address any possible nomination for the head job, a question that elicited laughter and applause from the audience.

“We have an opportunity to take [the new technologies] that have started to appear and apply them in a vigorous way to understand fundamentals of biology; that would include genomics and nanotechnology and a wide variety of approaches to epigenetics,” he said.

“I would also think we need to take seriously the charge coming from the Congress and the Administration to provide useful information to guide health care reform. That would mean, in some instances, comparative effectiveness research, but we need to be careful not to lose the personalized aspects of individual [health] along the way.”

“The U.S. is in a position to spread more soft power instead of hard power around the world; NIH ought to be able to play a useful role in that. And we should encourage the research community, including young investigators, and increase the diversity of our workforce, to make it vigorous and effective.”

WASHINGTON — The National Institutes of Health needs to partner more with the pharmaceutical industry in order to create a better pipeline for new drugs, Dr. Francis Collins said at the annual meeting of the Endocrine Society.

Dr. Collins, the former head of the National Human Genome Research Institute who at press time was rumored to be President Obama's pick for NIH director, said that with all the genomic research developments, “pharmaceutical companies are a little overwhelmed about where to start” when it comes to figuring out which genes would make good targets for drug therapy.

“Academic investigators should get more intentionally involved in the translational process of going from basic research to drug development,” Dr. Collins said. “There is an opportunity now, more than ever, to bring together academic investigators and the private sector to put together a really exciting version of a drug development pipeline.” Such a collaboration “involves more of academics taking the front-end risk of developing promising compounds so they become attractive and licensable by the private sector.”

Dr. Collins noted that many academic researchers are identifying promising targets for drugs, “but relatively few are taking that information and turning it into an assay … to see if there is something promising that might turn out to become a therapeutic.”

Some targets start out looking promising, but when they get to a point where they need support for preclinical development, “that's where things often die,” he said. “Congress just a few months ago put $24 million into the fiscal year 2009 budget to start this process in an NIH-funded way, and I hope the money will go up substantially in the next 5 years.”

With such a pipeline, conflicts of interest on the part of pharmaceutical companies “would have to be factored in,” Dr. Collins said in an interview. Drugmakers' interest in commercialization would be a factor. “You want to start a project that is going to get somewhere,” he said. “But there are companies across the board that are interested in almost any disease—even very rare ones—as long as it won't cost a fortune to get that drug approved. For the rare diseases, you may have to push things further down the pipeline with public money before the company says, 'Okay, I'll start with that one now,' but I don't know that that should discourage consideration of working on even a very rare disease in this pipeline.”

During a question-and-answer session, Dr. Collins was asked whether he would list his priorities for NIH “as a private citizen,” since he couldn't address any possible nomination for the head job, a question that elicited laughter and applause from the audience.

“We have an opportunity to take [the new technologies] that have started to appear and apply them in a vigorous way to understand fundamentals of biology; that would include genomics and nanotechnology and a wide variety of approaches to epigenetics,” he said.

“I would also think we need to take seriously the charge coming from the Congress and the Administration to provide useful information to guide health care reform. That would mean, in some instances, comparative effectiveness research, but we need to be careful not to lose the personalized aspects of individual [health] along the way.”

“The U.S. is in a position to spread more soft power instead of hard power around the world; NIH ought to be able to play a useful role in that. And we should encourage the research community, including young investigators, and increase the diversity of our workforce, to make it vigorous and effective.”

WASHINGTON — The National Institutes of Health needs to partner more with the pharmaceutical industry in order to create a better pipeline for new drugs, Dr. Francis Collins said at the annual meeting of the Endocrine Society.

Dr. Collins, the former head of the National Human Genome Research Institute who at press time was rumored to be President Obama's pick for NIH director, said that with all the genomic research developments, “pharmaceutical companies are a little overwhelmed about where to start” when it comes to figuring out which genes would make good targets for drug therapy.

“Academic investigators should get more intentionally involved in the translational process of going from basic research to drug development,” Dr. Collins said. “There is an opportunity now, more than ever, to bring together academic investigators and the private sector to put together a really exciting version of a drug development pipeline.” Such a collaboration “involves more of academics taking the front-end risk of developing promising compounds so they become attractive and licensable by the private sector.”

Dr. Collins noted that many academic researchers are identifying promising targets for drugs, “but relatively few are taking that information and turning it into an assay … to see if there is something promising that might turn out to become a therapeutic.”

Some targets start out looking promising, but when they get to a point where they need support for preclinical development, “that's where things often die,” he said. “Congress just a few months ago put $24 million into the fiscal year 2009 budget to start this process in an NIH-funded way, and I hope the money will go up substantially in the next 5 years.”

With such a pipeline, conflicts of interest on the part of pharmaceutical companies “would have to be factored in,” Dr. Collins said in an interview. Drugmakers' interest in commercialization would be a factor. “You want to start a project that is going to get somewhere,” he said. “But there are companies across the board that are interested in almost any disease—even very rare ones—as long as it won't cost a fortune to get that drug approved. For the rare diseases, you may have to push things further down the pipeline with public money before the company says, 'Okay, I'll start with that one now,' but I don't know that that should discourage consideration of working on even a very rare disease in this pipeline.”

During a question-and-answer session, Dr. Collins was asked whether he would list his priorities for NIH “as a private citizen,” since he couldn't address any possible nomination for the head job, a question that elicited laughter and applause from the audience.

“We have an opportunity to take [the new technologies] that have started to appear and apply them in a vigorous way to understand fundamentals of biology; that would include genomics and nanotechnology and a wide variety of approaches to epigenetics,” he said.

“I would also think we need to take seriously the charge coming from the Congress and the Administration to provide useful information to guide health care reform. That would mean, in some instances, comparative effectiveness research, but we need to be careful not to lose the personalized aspects of individual [health] along the way.”

“The U.S. is in a position to spread more soft power instead of hard power around the world; NIH ought to be able to play a useful role in that. And we should encourage the research community, including young investigators, and increase the diversity of our workforce, to make it vigorous and effective.”