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David Altman

Group handshake representing a consortiumDavid Altman is manager of marketing and communications in Boston Children’s Hospital’s Technology and Innovation Development Office.

Successful therapeutic development requires multiple stakeholders along the path from discovery to translation to clinical trials to FDA approval to market availability. At various points along this path, academia, industry, government, hospitals, nonprofits and philanthropists may work together. Would bringing these stakeholders together from start to finish lead to greater success?

A growing number of private-public consortia are launching in defined “pre-competitive” spaces where potential rivals collaborate to generate tools and data to accelerate biomedical research. In 1995, consortia were rare in health care: Only one was created. In 2012, 51 new consortia were launched, according to the organization Faster Cures.

Why? you may ask. Banding together in consortia can reduce costs, minimize failures and shorten the timeline to approval for new drugs. Full story »

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Tangled roots

Drug approval is increasingly intertwined with pricing questions.

At last month’s BioPharm America conference, what I originally thought would be a run-of-the-mill panel wound up being a frank discussion about regulatory and pricing challenges that pharma and biotech companies are facing today. I hadn’t realized these two challenges are intertwined so closely.

The regulatory and pricing paths for new drugs in the United States have become increasingly difficult to navigate. Due to outside policy pressures, the FDA is scrutinizing drugs more than in the past, requiring much more data. Even when a drug is approved, there is no guarantee that payers will cover its full cost, as they are starting to consider the drug’s overall value—improving quality of life and decreasing costs—along with its effectiveness.

Meanwhile, in many European single-payer countries, pharmaceutical companies are being told how to price their drugs before they are considered for approval by the regulatory agencies. The likely effect is less return on investment on new drugs, which could in turn decrease the pace of innovation.

Vaughn Kailian, managing director of MPM Capital, a health care venture capital investment firm, led an eye-opening conversation around these topics. Full story »

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Neuron_in_tissue_culture

Neuron in tissue culture (Wikipedia creative commons)

Translational neuroscience research has seen a disappointing streak of failed clinical drug trials. While the need for therapeutics that target the nervous system is growing, recent results in diseases like Alzheimer’s and autism have disappointed, and many companies have begun to downsize their R&D investments. Prospects are glum for patients who need new therapies to help manage their disorders.

The frustration is that drug candidates that have shown promise in animal models have not demonstrated efficacy in humans. Mouse models are not proving to be sufficient surrogates for human neurologic disease. Human brains and brain cells are built and function differently, and many neurodevelopmental disorders—hard enough to diagnose in human children—don’t have identifiable behavioral counterparts in mice. As I hear over and over from scientists, there is no such thing as a mouse with autism.

A study, published in Cell Stem Cell this June and conducted by Clifford Woolf, MD, PhD, et al, is among the first to demonstrate the power of an alternative technique: modeling disease in neurons derived from induced pluripotent stem cells (iPS cells). Full story »

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Thalidomide advertisement, c. 1961

Thalidomide advertisement, c. 1961

In times past, a pharmaceutical chemist’s main focus was to synthesize novel molecules to treat diseases. Today, an increasingly popular alternative is to re-engineer an existing drug—and continually improve it even after FDA approval. That’s how Robert D’Amato, MD, PhD, developed Pomalyst®, recently approved to treat multiple myeloma and the most potent analog to date of thalidomide.

Pomalyst_CelgeneCorpThalidomide has its own fascinating history. Originally developed by Chemie Grünenthal GmbH in the 1950s, it was the result of a search for an anti-anxiety drug to compete with Valium, and was approved for use in Europe as a sleep aid and depression treatment. Eventually, doctors found it useful for treating nausea, and started prescribing it off-label to pregnant women with morning sickness.

The results were disastrous. Thalidomide turned out to be a teratogen, causing severe birth defects.  Full story »

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In a four-way collaboration, skin cells from patients with autism will be used to make pluripotent stem cells. These will be made into neurons -- for study of what goes awry at the cellular level in autism, and for testing of drugs. (Miserlou/Wikimedia Commons)

In recent years, creative new partnerships have demonstrated big pharma’s recognition that academic medical centers hold many important cards in clinical research: scientific expertise, animal models of disease, patient samples and phenotypic data.

Increasingly, these partnerships involve academic and company researchers developing joint grant proposals in targeted areas, selected (by joint agreement) for company sponsorship. Some, like the Immune Disease Institute’s $25M arrangement with GlaxoSmithKline, are specific to one academic institution; others, like Pfizer’s Centers for Therapeutic Innovation (CTI) program, provide the same resources under the same deal structure to multiple institutions. Each new deal advances the interaction and understanding between academia and pharma around the common goal of finding new compounds and bringing them to clinic.

Now, in an exciting twist on its track record of partnerships with academic institutions, Roche has brought together three Harvard-affiliated organizations to screen and identify new drugs for the treatment of autism spectrum disorders (ASDs). Full story »

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Even a small idea, given a small boost, can have a high impact. (Rick Kimpel/Flickr)

When I tell people I work at the Technology and Innovation Development Office at Children’s (TIDO), they usually think I work to commercialize patented blockbuster drug candidates. But many of the most satisfying projects I help promote are innovations that don’t involve as much risk, time and investment, yet make a big difference for patients. Commercializing these innovations can help the greater good, and is part of what propels me to work at a licensing office at a pediatric hospital.

And sometimes it doesn’t take much to help them along.

The Sonnewheel Body Mass Index Calculator and the Vidatak communications board for patients unable to speak or write are some products supported by TIDO without income being the primary goal. Another great example, which we blogged about recently, is helping make routine blood draws less stressful for kids with learning differences and their parents.

The Blood Draw Learning Kit grew out of a serendipitous meeting. Full story »

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Sometimes promoting a non-money-maker is just the right thing to do. (Photo: iChaz/Flickr)

An article popped up in my Google alerts that gave me some excitement.  A survey from the Association of University Technology Managers, reported in Mass High Tech, placed Children’s Hospital Boston fifth in licensing income among all U.S. hospitals. We were ranked just below the Mayo Clinic, which has more than double the research funding of Children’s. Massachusetts General Hospital was second on the list and Brigham and Women’s was eighth.*

I don’t often get to see quick financial results from my work (I’m the marketing and communications specialist in Children’s Technology and Innovation Development Office (TIDO), which licenses the Hospital’s technologies). But what I do get to see regularly is just as important to our mission: small advances that barely impact the hospital’s bottom line but have a large significance to our patients. Full story »

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Streptococcus pneumoniae kills over a million young children a year, most of them in the developing world. Yet currently available pneumococcal vaccines are only effective against strains circulating in Europe and the US.  How can we make affordable vaccines that work globally? Full story »

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