Tag Archives: drugs

The changing landscape of pharma: a new route for PhDs?

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The pharmaceutical industry is changing – challenging for some, but an open road to opportunity for others. And the latest opportunity in the world of pharma comes from a rapidly growing demand for Medical Science Liaisons (MSLs), says Dr. Martijn Bijker.

As more drugs come to market and pharma’s business model shifts from selling drugs directly to fostering earlier engagement, companies are looking for highly skilled scientifically- and clinically-trained candidates. Could this be an outlet for the overflowing pool of PhD (and MD) graduates produced every year?

So what is an MSL?

An MSL is the scientific and clinical disease expert within a pharmaceutical or biotech company. They’re the go-to person for any complex questions about a specific drug. That could include questions about the science behind the drug, the mode of action, the competitors’ drugs, side effects, clinical trials, research opportunities, and disease-related questions.

MSLs work at the interface between internal stakeholders in the company and external stakeholders in the field – called Key Opinion Leaders (KOLs). MSLs help to bring innovative new drugs to the market and provide education about the proper use of drugs that are already on the market. KOLs – broadly defined as leaders in their field – can be heads of departments at teaching hospitals, heads of pharmacies, professors of medicine, the CEO of a patient organisation, physicians involved in pharmaceutical clinical trials and sometimes clinical scientists themselves. In short, MSLs work with the most influential stakeholders in a therapeutic area.

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MSLs help to bring innovative new drugs to the market and provide education about the proper use of drugs that are already on the market.

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Drug approval (and rejection) highlights from 2012

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Another year, another round of approvals, mixed reviews and high-profile failures. We look back on which medicines made the headlines.

GREEN LIGHT

Xeljanz (tofacitinib)

In November, the US Food and Drug Administration (FDA) approved the first oral disease-modifying drug for rheumatoid arthritis in more than a decade. Pfizer’s twice-daily pill—which works by blocking enzymes, called Janus kinases, that are involved in joint inflammation—is also being tested as a treatment for psoriasis, colitis and other diseases.

PCSK9 inhibitors

A new class of agents directed against proprotein convertase subtilisin/kexin type 9 (PCSK9), a key regulator of cholesterol metabolism, is moving into the last stage of clinical development. In phase 2 trials reported this year, experimental PCSK9 inhibitors from Amgen, Pfizer and Regeneron Pharmaceuticals (in partnership with Sanofi) each reduced low-density lipoprotein levels by 40–60%, on average, in various patient populations.

Kalydeco (ivacaftor)

The first drug designed to treat the underlying cause of cystic fibrosis won approval in 2012 from both European and US regulators. Although the approved therapy will only benefit those 4% of cystic fibrosis sufferers who have a specific genetic defect known as the G551D mutation, Vertex Pharmaceuticals reported interim data this year showing that the drug, marketed as Kalydeco, helped improve lung function in people with the most common mutation associated with the disease when administered in combination with an experimental agent called VX-809.

Glybera (alipogene tiparvovec)

European regulators in November approved the first gene therapy product to be used in the Western world. UniQure’s Glybera relies on an adeno-associated viral vector to deliver a functional copy of the gene that is deficient in individuals with a rare lipid-processing disease called lipoprotein lipase deficiency, which affects around one in a million people worldwide.

Aubagio (teriflunomide) and BG-12 (dimethyl fumarate)

People with multiple sclerosis now have a second oral drug option—and a third could be just around the corner. In September, the FDA approved Sanofi’s once-a-day tablet Aubagio for the treatment of relapsing forms of the disease. A week later, Biogen Idec posted positive phase 3 trial data for its own oral agent, BG-12. Both pills face competition from Novartis’s Gilenya (fingolimod), which hit the market in 2010.

T-DM1 (trastuzumab emtansine)

In November, the FDA granted priority-review designation to an antibody-drug conjugate developed in a partnership between Roche and ImmunoGen. Earlier in the year, phase 3 data showed that the therapy—a combination of the monoclonal antibody Herceptin (trastuzumab) linked to the cytotoxic agent mertansine—prolonged median survival by about six months longer than standard therapy did among women with advanced HER2-positive breast cancer.

Truvada (enofovir/emtricitabine) and Stribild

Gilead’s combination antiretroviral pill Truvada received the blessing of US authorities in July as a strategy for reducing the risk of HIV infection among adults at high risk of sexually acquired infection. A month later, Gilead scored another FDA blessing for its four-in-one drug Stribild, which packages Truvada together with two newer ingredients, elvitegravir and cobicistat, to combat the virus in HIV-positive individuals who have not received prior treatment.

Odanacatib

In July, Merck announced an early close to a 16,000-person clinical trial of odanacatib after a review of early results reportedly showed that the experimental osteoporosis drug significantly reduced the risk of bone fractures among post-menopausal women. Phase 2 data released this year similarly demonstrated that the investigational cathepsin K inhibitor increased bone mineral density by significantly more than standard therapy. Merck plans to file for regulatory approval early next year.

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Open Season

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Misha Angrist is the author of Here is a Human Being: At the Dawn of Personal Genomics (HarperCollins), now out in paperback. He teaches at Duke University and blogs at blogs.plos.org/genomeboy.

Us…and Them

And after all we’re only ordinary men.

Roger Waters

As a graduate student, I studied the genetics of Hirschsprung disease, a congenital disorder of the nervous system in the gut (and, as I describe in my book, a disease that would affect my own family many years later). Among the things I found to be most gratifying (and yes, occasionally frustrating) in my doctoral studies were the interactions with Hirschsprung patients and families. We students had pledged our fealty to Science writ large, yes, but we weren’t studying roundworms or fruit flies. Our “subjects” (a descriptor of research participants that, in my opinion, is condescending and should be retired ASAP) were thinking feeling human beings. If we found a highly penetrant mutation in their DNA, it had the potential to alter their reproductive decisions and their lives. It meant something to them.

But even if it didn’t, shouldn’t life scientists-in-training, especially those whose model organism is Homo sapiens, have some sort of mandatory exposure to, you know, life? Should there not be some inevitable, meaningful exchange between researcher and researchee?

Increasingly, community members are beginning to assert this right in various ways: Open Science, PatientsLikeMe, the Society for Participatory Medicine and the Sage Bionetworks Commons are just a few manifestations. The recent ScienceOnline meeting, which embodies the same sort of grassroots ethos, is my favorite science gathering for exactly that reason.

But of course participants in these endeavors are self-selected. How do we reify their approaches on a massive scale? Virally spreading the word, certainly. But it will also require bravery and iconoclasm. Recently I read The Cure, Geeta Anand’s heartbreaking 2006 book about John Crowley’s tireless struggle in the early 2000s to get a treatment developed for his kids, two of whom have the devastating lysosomal storage disorder Pompe disease (the book was the basis for the movie Extraordinary Measures). At one point Crowley is trying to impress upon members of the drug development team at Genzyme the urgency of their task. He organizes a “Pompe Summit,” to which the 200 employees working on the disease are invited, as are patients and their families. “How many of you have ever met a patient?” he asks the Genzymers. Only a handful of hands go up. Even the doc leading the company’s Pompe trial had never met a patient.  Imagine an automotive design engineer never having driven a car. Extraordinary measures indeed.

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