Stories about: drug discovery

The ever-changing, interwoven regulatory and drug-pricing landscape

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.

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Developing cures for neglected diseases: Overcoming the barriers

(scottchan/Fotolia.com)
Who will invest in the clinical development of drugs that offer limited commercial opportunities?(scottchan/Fotolia.com)
The desire to impact areas of great need drives many academic medical researchers. Unfortunately, a variety of challenges can prevent even the most promising innovations and technologies from reaching the patients who would benefit most. When the target population is primarily in the developing world, these challenges are magnified. Only a fraction of research and development funding goes toward treatments that target neglected diseases and the needs of low- and middle-income countries, posing a particularly frustrating situation.

The Universities Allied for Essential Medicines (UAEM)’s recent forum on Global Access Licensing of Biomedically Relevant Technologies delved into this pressing issue. According to the UAEM philosophy, the accessibility of medicine to developing nations “depends critically on how universities manage their intellectual property.” Further, the UAEM suggests that obtaining patents means that “anyone who can’t afford the asking price will be unable to access the product” and that “further innovation is hampered or outright blocked.”

In contrast, many of the panelists at the forum didn’t see intellectual property licensing as the primary obstacle—rather, they viewed it as a requirement to attract industry partners.

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Big things from small places: Modified RNAs eyed as a way to make new drugs

A technology from a small research institute, originally developed as a safer way to make embryonic-like stem cells, just hooked a very large fish. As The New York Times reported yesterday, pharma giant AstraZeneca is betting at least $240 million that this technology could be the source of a variety of new drugs—drugs that spur the body itself to make what it needs.

In 2010, the lab of Derrick Rossi at the Immune Disease Institute, which is now the Program in Cellular and Molecular Medicine at Boston Children’s Hospital, reported that they could reprogram ordinary cells into pluripotent stem cells by simply injecting them with messenger RNAs. The mRNAs reprogrammed the cells up to 100 percent more efficiently than other techniques, and did so without becoming part of the cell’s genome, greatly reducing concerns about cancer associated with other methods.

Key to the discovery were the chemical modifications made to the mRNAs so that cells wouldn’t “see” them as viruses and attack them. This video and this article describe the modified mRNA technique, also described in Cell Stem Cell:

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The problem with modeling metastatic cancers: Is it the mouse’s fault?

While the mouse is widely used to model many diseases, including cancer, the results we gain from it rarely translate well to people. But whose fault is it? (Wednesday Elf - Mountainside Crochet/Flickr)

The humble house mouse (or Mus musculus) is probably the most widely used model animal in biomedical research (beating out my favorite, the zebrafish, by a long mile). Millions are studied around the world every year, helping us understand the genetics of health and disease as well as the biology of cancer, diabetes and a host of other conditions. Mouse modeling is also often a major step in developing and getting FDA approval for new drugs.

But the mouse sometimes gets a bad rap in the research world. While it can be an effective and affordable model, and 95 percent of its genes are similar to ours, it is less than ideal for some of the diseases we study with it.

Take cancer, for instance. It’s relatively easy to cure cancer in a mouse; we’ve done it millions of times over. (The late Judah Folkman, MD, founding father of Boston Children’s Hospital’s Vascular Biology Program (VBP) and of the field of angiogenic research, famously said, “If you’re a mouse and you have cancer, we can take good care of you.”) Mouse and human tumor cells are fundamentally different in many ways. And the way that tumors behave in mouse models doesn’t necessarily reflect the way they behave in their natural environment (that is, in us)—a major consideration, especially when it comes to looking for new treatments for cancer that has spread (aka metastasized). More often than not, drugs that are successful in mouse models fail in the clinic.

But is it the mouse’s fault? Or is the problem the way we develop our models and run our experiments?

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Let’s liberate clinical trial data

The Gutenberg press disseminated ideas to a wider society. But in the clinical world, much information is still on "lockdown." (Wikimedia Commons)

The best things in life are free: friends, sunny days, beautiful vistas. Wouldn’t it be nice if knowledge were also free? Historically, libraries promulgated knowledge sharing because it was for the public good. We see this spirit increasingly embraced on the Internet – take the recent announcement of a collaboration between Harvard and MIT to make their courses freely available to users around the world via the edX platform.

But have we made all useful knowledge available in a way that allows for the greatest societal advancement? Not really. According to Ken Mandl, MD, MPH, director of the Intelligent Health Laboratory at the Children’s Hospital Informatics Program (CHIP), one important source of information still on lockdown is clinical trial data. In an article called, “Learning from Hackers: Open-Source Clinical Trials” published this month in Science Translational Medicine (not currently available in full text), Mandl and his coauthors call for making raw, de-identified clinical trial data free to the public.

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A good deal: Pharma and academia team up to use stem cells to find autism treatments

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).

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Ideas fly at Proteomics 2011

“What is proteomics?” Answering this simple question was the motivation for the Proteomics 2011, an annual symposium hosted by Judith and Hanno Steen of the Steen & Steen lab and The Proteomics Center at Children’s, featuring global innovators and local advances in proteomics at Children’s Hospital Boston, held last week. As a video at the start of the symposium showed, it’s a question that elicits a wide range of answers:


Video courtesy of the Carino Agency.

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