Seeing the surprising success of “poop pills” in gastrointestinal C. difficile infection, pharma companies and startups are embracing the microbiome as a new therapeutic target for an astonishing range of maladies. To learn what pioneering companies in the space are thinking about the hope and the hype, Vector recently attended a panel on microbiome therapeutics at the MassBio Annual Meeting. …
Academic researchers and physician innovators are great at making research discoveries and developing inventions at an early stage. But if you were to fund them to turn their research findings into a product, would they have the expertise and experience needed to be successful? Most would not.
The investment community talks about the innovation funding gap, a.k.a. the “valley of death.” But there is also a knowledge gap on the part of academic researchers when it comes to transforming their technologies into therapeutics. Most want their findings to lead to new treatments for patients, but they lack the experience and expertise that companies have to advance early-stage research to a clinical stage. That includes expertise in designing pre-clinical experiments and navigating regulatory pathways for commercial development.
Academics often enter agreements with pharmaceutical companies, many of which are early-stage research grants. Often, these industry-sponsored research projects end with a scientific publication and are unsuccessful in generating new therapeutics—a subpar outcome for the company investor. …
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.
“Value is more important than innovation,” declared Angus Russell, CEO of Shire Pharmaceuticals, in his opening keynote address at Biopharm America last week. At a drug development conference, where attendees typically focus on interesting new ways to address therapeutic problems, that sounded a bit heretical.
But it was a telling example of how cost pressures are now manifesting in pharma. Some complex treatments can be new and innovative, but not provide measurable improvement in patient outcomes. Can we conclude that they have no value? This has been one of the central questions fueling the debate over U.S. healthcare reform, and will influence development of medicines into the future.
My summary of BioPharm America 2011: We are a family and we just need to work together. As stakeholders in developing new treatments, we each have our own shortfalls and strengths, we’re under pressure, and our roles are changing over time.
Here’s the panelists’ take on the different players.
Big pharma: The old business model is broken. Pharma is cutting R&D and other programs that aren’t generating enough return. Companies now approach markets differently, said Angus Russell, CEO of Shire. A new product doesn’t have to be a first-line therapy to justify market entry; there’s a business case for selling a targeted drug to patients who don’t respond to generics and have no other solution. …
When the clinical and research enterprises at an academic medical center are strategically and tactically aligned, they enter a “virtuous cycle,” described by Stephen Wartman, president of the Association of Academic Health Centers. Clinician-researchers embody it: They know their patients’ needs, what treatments are available and the frustration when those options are insufficient. They take clinical problems back to the lab to figure out solutions, and are highly motivated to develop products because their goal begins and ends with helping the patient.
The academic medical center is the epicenter of therapeutic development. It’s where potential drug targets are identified and mapped from animal models to human tissues, and where clinical trials will ultimately take place. Yet, in the past, pharmaceutical and biotech companies have driven drug development, while academia has been a piecemeal contributor …
In almost every conversation I have these days with potential industry partners, I hear what seems to be the new buzz phrase: “Clear path to clinic.” A molecule with a “clear path to clinic” can become a medicine quickly, by virtue of a well-defined patient population, clear endpoints for clinical trials and measurable biomarkers.
Industry is increasingly recognizing that investigators at academic research centers know this path to clinic best. This has brought a shift in how industry interacts with academia, and new and equitable partnership structures are poised to facilitate joint therapeutic development like never before.
Pharma companies have a strong incentive to change. …
It’s widely acknowledged that the pharmaceutical industry is in crisis. Pharma companies are seeing drugs coming off patent, competition from generics, diminished R&D success and increased cost pressures, all of which are contributing to the downfall of the blockbuster drug era. Various solutions have been proposed, including creating shared knowledge repositories to learn from failures and pairing treatments with companion biomarkers.
These solutions and others have merit. But I’d propose more emphasis on what I consider a major failing of the industry: the inability to effectively commercialize naturally derived compounds possessing therapeutic benefit.
Pharmaceutical companies have typically avoided naturally derived compounds because it is difficult to keep competitors out of the territory. Legislation such as the Dietary Supplement Health and Education Act prohibits market exclusivity for natural compounds with known chemical compositions. …