Perhaps counter-intuitively, rare diseases can present attractive business opportunities for pharmaceutical companies. As discussed previously on Vector, they generally offer:
1) a population of patients with a high, unmet need, greatly lowering the bar to FDA approval
2) a closely networked disease community, greatly lowering the bar to creating disease registries and mounting clinical trials
3) well-studied disease pathways.
Recoiling from expensive failures of would-be blockbuster drugs, companies like Pfizer, Novartis, GlaxoSmithKline, Sanofi, Shire and Roche are embracing rare diseases, despite their small market sizes, because of their much clearer path to clinic. But in the current risk-averse industry environment, some projects are stalling. Industry may need more incentive to jump in—and Cydan Development is basing its business model on providing it. Full story »
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 »
In Part 1 last week, Vector took a look at digital health apps, telemedicine, genomics, phenomics and new behavioral diagnostics as transformative trends in pediatrics. This week, we complete our list. These posts will also appear as an article in the fall issue of Children’s Hospitals Today magazine.
6. New pharma research and development (R&D) models
Academic medical centers have always worked with the pharmaceutical industry but never so closely as now. In the old model, industry drove therapeutic development. A company might fund an academic project or supply reagents, but the relationship generally ended with the project (and publication of a paper).
Now, with drug pipelines drying up and R&D costs rising, Big Pharma is under pressure to change. New industry-academia collaborations are forging creative partnerships, altering how both parties do business. The new models are allowing hospital researchers to do what they’ve never done before: take the lead in R&D. Full story »
Recombinant DNA technology might turn bacteria into factories for producing siRNAs. (zoetnet/Flickr)
If you are a scientist and you want to turn off a gene, one option that’s been gaining traction is RNA interference (or RNAi). In this molecular process—first discovered in plants and only 12 years ago detected in mammals—bits of RNA called small interfering RNAs (siRNAs) cancel out a gene’s messenger RNA, effectively muffling that gene.
Labs can order custom-made, chemically synthesized siRNAs for just about any DNA sequence they want to silence. The tricky part is deciding what the right sequence is—especially when that gene is part of a virus, where genes can mutate pretty quickly.
However, a biotechnology approach to producing siRNAs could make it relatively easy for just about any lab that can master recombinant DNA technologies to make a number of siRNAs against multiple sequences within the same target gene: a potential bonus for companies seeking to make drugs that rely on RNAi. Full story »
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 Timesreported 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:
Diseases like malaria strike children harder than adults, but clinical trials for these diseases rarely include or focus on children. Why? (WHO/P. Virot)
We’re pretty focused on the safety of the things around us. Our drinking water gets checked for chemicals, bacteria and other things that could make us sick. Kids’ car seats are tested to make sure they’ll keep children safe in an accident.
But there’s one surprising arena where this focus on safety and testing often falls short: the medications we give our children. Not just in the United States, but globally.
There are lots of reasons why fewer drugs get tested for safety and efficacy in children than in adults. It’s time-consuming, expensive and, frankly, risky. The ethics of testing new medications in children are pretty thorny.
And, overall, the market for pediatric drugs is much, much smaller than that for drugs for adults, since children fortunately don’t get sick as often as us grown-ups.
But for some diseases like asthma and diarrheal diseases, children bear a greater burden than adults—one that’s not matched by the amount of research done on drugs for kids. Full story »
Nearly 50 years ago researchers isolated rapamycin from the soils of Easter Island. Like aspirin, it’s being used in a broad range of human diseases. (Ndecam/Flickr)
I’ve heard it said that if aspirin had to go through today’s FDA approval process, it would never be approved for over-the-counter use because it just does so many things. Lately, it’s been hard to cover biomedical research at Children’s without stumbling on another drug that’s also FDA-approved and also seems to have multiple uses: rapamycin.
It’s a drug that targets a pathway fundamental to nearly every cell in the body, yet is seemingly good for nearly everything. But how can one drug touch on so many cells and tissues and organs and still be both effective and safe? Full story »
“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.
Can we get this family to function? (Photo: eyeliam/Flickr)
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. Full story »
Since BioPharm is in Boston this year, we’re also heavily involved as presenters. Bruce Zetter of Children’s Vascular Biology program will speak about cancer biomarkers on a Wednesday panel titled Lessons Learned in Personalized Medicine in Oncology. Leonard Zon, director of Children’s Stem Cell Program, will describe the development of stem-cell-boosting drugs as part of a Friday panel titled Stem Cells and Drug Discovery: Confronting the Translational Imperative.
And Erik Halvorsen, director of Children’s TIDO, is a panelist in Wednesday’s session titled: Pharma’s Interest in Collaborating Early: Flavor of the Month or Viable Business Model?
Halvorsen elaborates on this theme in an interview for the online life sciences journal Partnering News. Full story »
Perhaps counter-intuitively, rare diseases can present attractive business opportunities for pharmaceutical companies. As discussed previously on Vector, they generally offer:
