The pharmaceutical industry is in the midst of an unprecedented change in its business model. Large pharmaceutical companies, while retaining remnants of their research divisions and some of their local manufacturing facilities, will soon cut to their core business of drug development. Emerging companies will fill the void, providing innovation and potential therapies for the development pipeline. And manufacturing will be contracted to third parties or conducted overseas where it is less expensive.
The current variants of this model-generic drug companies, contract research organizations, drug delivery specialty companies, and virtual pharmaceutical companies-will eventually fall into line, but not until large pharmaceutical companies acknowledge the new model and build a strong interface among themselves, emerging companies that provide innovative new therapies, and the regulatory bodies, such as the Food and Drug Administration in the United States.
M&A Activity Hampers Innovation A debate held at the American Association of Pharmaceutical Scientists (AAPS) annual meeting last November probed whether the last decade of mergers and acquisitions within the pharmaceutical industry had hampered innovation. The most compelling arguments concluded that it has. In fact, reduction in innovation is blamed for the scarcity of new chemical entities in development pipelines. As a result, major pharmaceutical companies are looking to emerging biotechs to supply compounds or technology platforms to license, acquire, or shepherd via strategic partnerships in an effort to bolster pipelines and allay shareholder concerns.
Exclusivity losses in one or more major markets will claim a number of blockbusters in the coming year, eroding innovators’ profits as the generics industry undersells them. Risperdal, Topamax, Lamictal, Depakote, and Fosamax are among those on the chopping block for 2008. And although there are 25 to 30 new products awaiting approval in 2008, 80 percent are specialty pharmaceuticals, according to IMS Health. It is hard to imagine there will be new blockbusters to replace those lost to generics. And considering the current rate of FDA approvals, it is difficult to predict how many of the new medications will even make it to market this year.
Global Market Growth The global pharmaceutical market is expected to grow 5 to 6 percent in 2008, to $745 billion, according to IMS Health. For the first time, the U.S. will comprise only one-third of the global pharmaceutical market as patients in China, Brazil, Mexico, South Korea, India, Turkey and Russia gain access to better healthcare. These emerging markets are driving new growth and opportunities for pharmaceutical companies, but the widening demographic argues more for specialty pharmaceuticals than for blockbusters.
Industry analysts report rising R&D costs and reduced numbers of new drugs approved each year. The reduction in drug approvals is blamed in part on the chronically understaffed FDA, in part on the lack of innovative drugs in the pipelines of major pharmaceutical companies, and in part on the inexperience and lack of funding that hampers smaller companies’ efforts to meet all of the regulatory hurdles in a timely fashion. In fact, many licensing deals are turning out to be more expensive than anticipated as licensees are finding it necessary to re-optimize their lead candidates.
Consider Regulatory Needs Early Now is the time for both large and small pharmaceutical companies to include relevant regulatory bodies in their development plans very early on. In the U.S., the FDA has been asking companies to engage it with greater transparency regarding development plans and study results. Although this might seem risky, obtaining early agreement on the path forward is likely to save time and money and avoid the dreaded “Approvable” letter. It might even prevent the lawsuits and public scrutiny of a Vioxx debacle.
In fact, pharmaceutical companies have a perfect opportunity to establish a more open relationship with the FDA by working with the agency to develop more predictive toxicity assays that eliminate problem compounds early in the development process. In an interesting article appearing in the November issue of Pharmaceutical Executive titled “FDA’s Approvable Problem,” senior editor Walter Armstrong discusses the controversy between the industry, the FDA, and public opinion. He notes the lack of public understanding that even though a drug has been approved, toxicity issues still can arise later. People don’t seem to understand all that talk about risk/benefit that is certainly a part of the approval process.
Communicate Risks Better This is something that will probably never go away. We cannot know everything about a drug before it is approved, but the industry needs to do a better job of communicating this to the public. It must make continual, concerted efforts to establish better methods for evaluating drugs before they are approved. That’s a “win” for everybody. The effort can be communicated to the public to show the industry’s concern and can help restore FDA credibility. In addition, information shared with emerging companies will ensure that these studies are implemented early in the development process before licensing.
As evidence of the new pharmaceutical business model in the making, the Financial Times published several articles last December on GSK’s new head of R&D, Moncef Slaoui, who inherited the much-heralded Centres of Excellence for Drug Discovery that CEO J.P. Garnier and then-head of R&D Tachi Yamada created in 2001. The centers were designed to separate the research organization into specialty units simulating autonomous biotechs; the units would identify quality lead compounds which they then handed off to the international development machine. After six years of experimentation, Slaoui commented: “It’s one thing to create an organigram. It’s another to change values…We’ve done well on the structure but more is required on behavior.” His resolution for the short term is to retain about four of the centers and work to re-focus them on the science and innovation, but at the same time to look for licensing opportunities to fill as much as 50 percent of the pipeline.