A round up of news from across the globe.
Realising that the era of blockbuster drugs is coming to an end, Big Pharma has turned its eyes to focus on a new game — orphan drugs. The pharma industry's new fascination with these drugs is highlighted in Thomson Reuters' The Ones To Watch report for October–December 2009, which examines some of the most promising drugs launched or receiving approval, and moving through each of the clinical phases, between October and December 2009.
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"As we see from this quarter's selection, drugs for small patient populations have already reached the market," says the report. "Meanwhile, drugs for urea cycle disorder and multiple myeloma are moving apace through the pipeline, giving hope to patients diseases that are unlikely to ever deliver a blockbuster drug."
The report also added that, according to a January 2010 report by the Tufts Center for the Study of Drug Development, the number of drugs receiving orphan status has doubled since 2000. Meanwhile, research from BCC Research also shows that companies migrating away from blockbusters to orphans are likely to be rewarded— the orphan drugs market is expected to grow from $58.7 billion in 2006 to $81.8 billion in 2011.
Of Thomson Reuters' predicted five most promising drugs launched or receiving approval, three are treatments for rare conditions. Dyax's Kalbitor, a treatment for hereditary angioedema, which affects between 1 in 10000 and 1 in 50000, has been forecast by Thomson Pharma to achieve sales of approximately $150 million in 2013.
Meanwhile, Folotyn, developed by Allos, was granted accelerated approval by the FDA in September 2009 and launched in the US in October, and is the first treatment for relapsed or refractory periphereal Tcell lymphoma (an often aggressive type of nonHodgkins lymphoma). The report also highlighted Istodax, developed by Celgene for the treatment of cutaneous Tcell lymphoma (CTCL). The drug is the second anticancer histone deactylase inhibitor to be approved after Merck's Zolinza (also for CTCL), which, according to the report, suggests "significant potential in this class of compounds".
The other two drugs highlighted were Novartis' Onbrez Breezhaler treatment for chronic obstructory pulmonary disease, which is expected by Thomson Pharma to achieve sales of $397 million in 2013, and Theravance and Astellas' Vibativ — a treatment for complicated skin structure infections caused by Gram positive bacteria that is forecast to reach sales of $128.2 million in 2013.
Thomson Reuters has also identified some of the promising drugs entering the various stages of clinical testing. Again, many treatments for niche conditions are featured including a treatment developed by ERYtech for acute lymphoblastic leukemia, which is entering Phase III trials and has orphan status in the US and the EU. A treatment for thymic cancer, which currently has no standard approved treatment at all, is also being investigated in a Phase II trial by Nerviano Medical Sciences
As well as orphan drugs, Thomson Reuters added that drugs for cancer continue to attract significant attention, with 25% of the quarter's promising drugs being for oncological indications. Interestingly, the report also showed that companies are looking at lifestyle fatcors that may lead to cancer; for instance, Nabi Biopharmaceuticals' NicVAX for Nicotine addiction has entered Phase III clinical trials, while a treatment for alcohol addiction, developed by Alkermes under license from Rensselaer Polytechnic Institute, has entered Phase II trials.
A pharmaceutical company's first line of defence against losses caused by products that fail to reach the market is a highly functioning and effective New Product Planning (NPP) group; however, establishing such a group is not always an easy task, according to a study released by research and consulting firm Best Practices LLC (NC, USA).
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The central challenge for NPP is to ensure that the right new products are developed efficiently with minimum risk and maximum value because a project that is halted in late stage development can cost hundreds of millions of dollars. The unstable financial landscape has also added even greater emphasis on cost reductions and the tightening of processes.
This makes the role of NPP crucial. In its study, Pharmaceutical New Product Planning: Structure and Activities to Drive Growth and Profitability, Best Practices explained: "With pressure on all stakeholders in the pharma industry and few new drugs coming to market in recent years, NPP should take on more and more importance in driving what projects to dedicate resources to."
There are several challenges facing NPP, including growing safety concerns, payer-controlled environments, accelerating costs and low approval rates, but the key challenge comes from within the company itself: interacting with different people across the organisation.
"Strong cross-functional communication and leadership skills are critical to ensure sound decisions are made in an efficient and timely manner," Cameron Tew, executive Director of Research at Best Practices, explained to Pharmaceutical Technology Europe. "For example, commercial can point out when the initial plans for a compound have low potential market value, or research can point out that changing a product's proposed formulation would be too costly or slow to meet project parameters."
Playing this cross-functional role is the main challenge for NPP. NPP adds a commercial aspect to a project, but Tew explained: "Though the commercial teams provide input into development, commercial values can take a backseat in organisations focused on the R&D aspects of product development. In fact, it is not uncommon for R&D and NPP to speak different languages and have different priorities that can prevent the two groups from understanding and fully valuing each other."
For this reason, NPP groups must be skilled negotiators and influencers who can communicate effectively across professional divides. "These diplomatic skills will help them excel in roles that have significant influence, but little authority," said Tew.
This cross-functional approach should begin as early as possible in the preclinical stages. If a decision to stop work on a drug is made at its earliest time, it can save significant amounts of revenue, and wasted R&D time and resources.
As well as cross functionality, another key requirement for effective NPP is centralisation because this allows people who work on different customer segments to share resources, ideas and experiences. "Diverse work groups also ensure that all viewpoints on a project are considered," said Tew. "By bringing all the key departments together in the earliest of stages, companies have the best chance of reaching a decision that is not myopic."
Centralisation helps establish and ensure common process, performance and evaluation standards for projects, programmes and product development, which Tew believes lead to faster, fairer and better prioritisation of projects, resources and capital requests that support corporate strategy and goals.
The full interview with Cameron Tew discussing the role and structure of NPP groups can be read online at: www.pharmtech.com/NNPinterview