Pharmaceutical Executive served as a lead media partner for the 2011 Prix Galien awards, held September 27 at the Museum of Natural History in New York City. The annual gala awards dinner, which recognizes innovation in products and agents that improve the human condition, brings together stakeholders and top influencers in the industry, including professional and scientific groups, academia, patient associations, and more for a night of celebration.

This year, top winners were Janssen’s Stelara and Amgen’s Prolia and XGEVA for Best Biotechnology Product; and Pfizer’s Prevnar 13 for best medical agent. Additionally, Dr. Paul Farmer—medical anthropologist, physician, Kolokotrones University Professor at Harvard University, Chair of the Department of Global Health and Social Medicine at Harvard Medical School, and founding director of international non-profit organization Partners in Health—took home the Pro Bono Humanum award.

“Dr. Farmer is an exemplary model of how one person can be a catalyst between science, business, and government to serve as a beacon of hope to those in need. Dr. Farmer’s dedication to combating deadly diseases while fighting poverty and inequality in developing countries is inspirational,” said Nobel Peace Prize winner and Prix Galien USA 2011 Award selection committee member Professor Elie Wiesel in a statement.

As another year of innovation in the industry comes to a close and is honored by Prix Galien, Gerry Weissmann, chairman of the committee of the Prix Galien, reflects on all that’s been accomplished: “After two centuries of tough, sometimes controversial experimentation, we’ve replaced pre-FDA home remedies and snake oil with drugs that address the basic mechanisms of human disease,” he said during the ceremony. “This year, the score of candidates for the Prix Galien USA shows what happens when we bring basic science from the bench to the bedside in a bottle or pill that’s been approved by the FDA … Teddy Roosevelt would have been proud that the industry he helped save from snake oil has made a difference in human life.”

Robert Epstein, chief clinical research and development officer at Medco, said too often pharmaceutical companies “show up in the middle of Phase III with a half-baked cake,” which inevitably raises questions. “What about sub-populations with side effects? What about an additional endpoint, an observational study or comparative information?” asked Epstein.

In response, panel moderator Richard Pasternak, an associate professor at Harvard Medical School and a former Merck VP, said “payers aren’t interested in early stage research, and pharma isn’t set up to listen to them.” Barry Gertz, SVP and head of clinical research and regulatory affairs at Merck, suggested that despite regulations and other challenges, “We have to force that dialogue to occur. The mechanisms haven’t evolved to include the needed communications, and the structure for payer/pharma communications.”

Epstein said R&D departments – not marketing departments – should be in direct contact with payers at the very earliest stages of development. Researchers, after all, are “more candid,” and they “ask the right questions.” Roger Longman, founder of Windhover Information and CEO at Real Endpoints, said Medco may be interested in early stage communications with pharmaceutical researchers, but “other payers aren’t, and R&D [researchers] aren’t.” R&D departments “are marketing-driven,” and health insurance payers have “very little incentive to dialogue with pharma early on,” said Longman. “Insurance companies wait and then say you don’t have the right data, we’re going to screw down the price, and government is banned from using cost effectiveness to determine how much it will pay” for a drug. Longman cited Italy as the “most aggressive” geography for value-based agreements involving payers in the drug development process. In Italy, “you have a drug, you bring it to a payer, an endpoint is identified, and if you meet the endpoint, you get paid,” he said.

Peter Pitts, president of the Center for Medicine in the Public Interest, wondered which R&D groups would be available for speaking with payers. “Are we talking about the Quintiles of the world? Who, inside pharmaceutical organizations, is left to think about clinical trial designs?” asked Pitts, somewhat rhetorically.

Regardless of what endpoints are pursued in clinical trials, Epstein said comparing a drug to placebo, as opposed to a comparable therapeutic product, “doesn’t work for us.” He cited a JAMA article published last May, finding that over “50% of all new drugs” approved by FDA come with comparative effectiveness data. The study also found that more than two-thirds of new molecular entities recently approved in therapeutic categories where alternative treatment options exist contain comparative effectiveness data.

The panel, titled “What is ‘Value’ and How Can it be Measured and Demonstrated in Therapeutic Innovations,” was part of a forum associated with the Galien Foundation’s Prix Galien ceremony, held last night in New York City. Prix Galien winners this year included Janssen’s (J&J) Stelara and Amgen’s Prolia, for best biotechnology products, and Pfizer’s Prevnar 13, for best medical agent.

At the gala ceremony for the 2009 Prix Galien USA award on October 1, a paradigm shift in drug development took center stage.

“The most successful new [cancer] therapies will be based on a precise molecular understanding of disease basis—that’s exactly what we did with Gleevec,” said keynote speaker Brian Druker, director of the Oregon Health and Science University Knight Cancer Institute, who played a major role in the discovery of the “magic cancer bullet” that is one of the most successful drug’s of the decade. Accordingly, Novartis’ Gleevec (imatinib mesylate), a kinase inhibitor that stops the protein that causes chronic myeloid leukemia, received the award for Best Pharmaceutical Product.

Druker’s speech considered the benefits of collaboration between the drug industry and academics in solving scientific conundrums. “Basic science can actually advance an undruggable target to a druggable target. It is my view that investment in science in large consortiums can move the ball forward much faster than each of us can do alone,” he said.

Druker even went so far as to propose a new system of conducting clinical trials: “We need a complete new branch of science—a branch that I’ll call human investigation—to investigate patients with the same level of scientific detail that we put into our basic science.”

Druker’s visionary proposals, delivered in the appropriately inspired setting of the American Museum of Natural History’s “Whale Room,” met with polite applause from the black-tie crowd celebrating the industry’s most innovative branded products. The translational-medicine model that brought Gleevec from lab bench to patient bedside in near-record time is the glittering exception that proves the glum rule of high-risk pharma R&D. Many of the drugs nominated this year are the product of decades-long research and billion-dollar investment, some working off of discoveries made nearly 50 years ago.

Two drugs approved last year by FDA for the treatment of the rare disease idiopathic thrombocytopenic purpura (ITP) shared the Best Biotechnology Product honor: Amgen’s, Nplate (romiplostin), and Promacta (eltrombopag), a collaboration between GlaxoSmithKline and Ligand Pharmaceuticals. Both products were developed under the orphan-drug designation.

In recognition of the increasing role that diagnostics play in clinical practice, the Prix Galien USA gave its first Best Medical Technology award. The winner was Veridex’s CellSearch System, the first device able to identify and count circulating tumor cells (CTCs) in blood samples to help predict survival in patients with metastatic breast, colorectal or prostate cancer.

The committee presented its Pro Bono Humanum Awards to two pioneers in the burgeoning global-health movement, Dr. Barry Bloom, an immunologist and professor at the Harvard School of Public Health, was recognized for his work to eradicate disease by understanding immune response to infectious diseases including leprosy, TB and malaria. Jeffrey Sachs, an economist and director of the Earth Institute a Columbia University, was awarded for his work on Millennium Villages, the U.N.’s public/private partnership project that helps rural African communities lift themselves out of poverty by providing financial and health care support.

The Prix Galien Award was established in 1970 by French pharmacist Roland Mehl and inaugurated in the United States in 2007. Pharmaceutical Executive is the media sponsor of the event.

Visit Prix Galien’s website for more information.

The Prix Galien USA committee was inaugurated in 2007 as the industry’s equivalent of a Nobel Prize. The award honors innovation by organizations and researchers developing drugs and therapy for worldwide use. The Pro Bono Humanum award is also given to a person or group that has improved the worldwide human condition through the use of pharmaceutical science. 2009 winners include Novartis, Amgen, and GlaxoSmithKline.

Bell opened the talk, discussing the lessons learned by Alexion during the development of Soliris, a groundbreaking drug that combats rare blood disease PNH. He found that innovation took place as a result of a well-designed long-term development process with public support, allowing the company to distribute the drug free of charge to the uninsured.

Sachs spoke next, discussing the difficulties developing economies face from the burden of disease. “We should not be leaving 8.8 million children to die each year,” he said of the developed world’s lack of support to public health in Africa. He continued to posit that if ten cents out of every hundred dollars made in the developed world were donated to support Africa, those 8.8 million deaths could be prevented.

Bloom followed by voicing his concern that rich countries have not solved the problem of social responsibility. Without rewarding researchers for investigating diseases that may not ensure a big payday at the of the development cycle, he said, true progress against disease cannot be made on a global level.

At the Prix Galien awards ceremony last month, I had the opportunity to interview the winners on camera. We’ve just posted edited versions of the interviews here. Inspiring stories, remarkable people (and some great-looking tuxes). Enjoy.

Click here to view a slideshow from Prix Galien 2008

The 2008 Prix Galien USA is in the books, and Pharm Exec was in attendance at New York’s Museum of Natural History to take in all the festivities. Pfizer, Merck, Alexion, Wyeth, and Celgene were all recognized for their work in advancing science in both the biotech and pharma industries. Charlie Rose played master of ceremonies to a packed house featuring a who’s who of the science elite and a handful of Nobel Laureates.

The Prix Galien USA 2008 winners are:
Best Pharmaceutical Agent (Small Molecule)

• Selzentry (Pfizer)
• Isentress (Merck)

Best Biotechnology Agent

• Soliris (Alexion Pharmaceuticals)
• INFUSE (Wyeth)

Special Therapeutic Development

• The Celgene Corporation for Revlimid

The evening ended with a moving speech by author and Nobel Prize winner Elie Wiesel and a presentation of the Pro Bono Humanum award to Population Council scientist Sheldon Segal. For more on the Prix Galien 2008, keep a look out for the October issue of Pharmaceutical Executive Magazine.