Beyond Blockbusters

“A growing numbero of companies have found great opportunity in targeting therapies for smaller patient populations”

As many pharmaceutical companies struggle with their pipelines, success may no longer be defined by blockbuster drugs alone. Rather, some of the most promising pipeline products today are focused on specialty therapeutics, suggesting an alternative pathway for success in the industry. Regardless of how complex or challenging targeted therapies are to develop, there is significant opportunity – and need – for companies to champion medicines for much smaller patient populations.

Recent failures of late-stage pipeline drugs, the increasing number of patent expirations and drug safety issues have underscored the industry’s vulnerability when companies rely too heavily on a few billion dollar products.

Specifically:

• From 2002-2006, the pharmaceutical industry brought to market 43% fewer new chemical-based products than in the last half of the 1990s.
• Drugs with US sales totaling $17 billion lost exclusivity in 2007, with generics replacing brand-named drugs in key therapeutic areas, and this number is expected to grow to a loss of $67 billion in sales for pharmaceutical companies between now and 2012.

• The US.market sales growth for pharmaceutical and biotech products slowed to 3.8% in 2007 (from 8.3% in 2006), according to IMS Health.
• As safety controversies brew around a number of drugs, the FDA is issuing more refuse-to-file letters (11 were issued in 2007 alone), black box warnings, precautionary labeling changes and requiring larger studies of new drugs than ever before.

While companies have historically shifted their attention away from medicines that serve specialized patient groups to focus on larger populations, ‘me-too’s’ or only slightly differentiated products, a growing number of companies have found great opportunity in targeting therapies for smaller patient populations with unmet medical needs. With more than 25 million Americans and millions more throughout the world suffering from more than one of 6,000 rare conditions, there is considerable opportunity for the industry to look at how and why therapies targeting specialized patient groups are not only good for patients but also good for business.

The following learnings may help pharmaceutical executives better identify areas of unmet medical need, which specialty drugs to pursue, and how to overcome challenges to successfully bring more targeted therapies to market.

Uncover unmet needs

The initial step to shifting from large-scale to specialized markets is to identify critical voids where populations of severely-ill patients lack access to effective drugs, and then focus on medicines with the potential to help these groups. Companies can do this by cultivating strong advocacy relationships with patient and professional groups, as well as by listening to physicians about the needs in their practices.

For example, more than five years ago, several leading child epilepsy thought leaders urged us to acquire a highly promising drug for infantile spasms (IS) that was available in more than 50 countries around the world (although the clinical work needed to obtain approval in the US had never been completed).

We listened to the physicians and advocacy groups who were eager to have an approved treatment for this rare and devastating type of childhood epilepsy. In 2004, the company acquired the North American rights to a novel epilepsy drug and began working with pediatric neurologists to complete the development program for US approval.

Harness innovation

An alternative, technology-intensive approach to drug development gaining momentum today is the push towards finding targeted treatments for niche indications based on a person’s genetic makeup (often called personalized medicine). The ultimate goal of this approach is to find the right drug for the right person at the right time, thus increasing the impact of treatment and decreasing potential side effects.

Further advances in molecular medicine will likely increase the prevalence of tailor-made therapies, many of which will then qualify for orphan exclusivity. This will enable companies to benefit from the economic incentives that come with developing therapies for rare conditions. Most importantly, more adequate treatments for serious, debilitating conditions for which there have been no effective treatment options will be available for the patients who need them.

Add low-risk projects

When considering which development programs to take on, companies can substantially minimize their risk by pinpointing which medicines have the greatest potential – and also the shortest regulatory pathways – for ultimately achieving approval. Two strategies for finding lower-risk investments are:

1. Looking abroad for therapies with proven track records. Specialty therapies on the market in other countries typically have substantial clinical data available to demonstrate their efficacy and safety. Given that the likelihood of an unexpected safety issue emerging is lower, these therapies often make the strongest targets for development in the United States. These programs also have a more straightforward regulatory pathway, maximizing the potential for a successful outcome.

2. Seeking new indications for already-approved products. There are often significant opportunities to explore new uses for drugs currently within a company’s portfolio. We regularly investigate how its proven commercial products might satisfy other unmet medical needs and help even more people. For example, an approved therapy for acute porphyrias is being explored for treating other hematologic and oncologic disorders.

Develop marketing creativity

An inherent benefit of pursuing a more targeted approach to drug development is the built-in demand for these products. Companies working with smaller patient populations often find their medicines are extremely well-received, taking on blockbuster significance for the patients who have had few, if any, other treatment options available. Yet, the challenge is identifying physicians who treat these patients.

Companies can improve their chances of successfully commercializing medicines for smaller patient groups by ensuring their marketing teams use a personalized approach: pinpointing the right physicians who, in turn, identify the patients in need of a specialized therapy. For example, where traditional prescription data doesn’t uncover physicians already treating a specialized/rare disease, we’ve found that we can supplement traditional data sources with medical claims and/or hospital discharge data. This provides valuable insights into symptoms being treated, as well as the physicians and institutions already diagnosing specific rare diseases, thus enabling us to target our outreach efforts directly to those most in need of our therapies.

Consider outsourcing

A final consideration for companies looking to develop smaller-volume therapies is determining the most effective way to manufacture products while maintaining a consistent supply for patients whose lives depend on it.

For small-volume products, having flexibility in production capabilities is essential. Working with contract manufacturing organizations (CMOs) allows companies to apply their technical expertise to the process of manufacturing a broad range of complex formulations, while benefiting from the specialized processing equipment different CMOs offer. In this way, the special needs of each product are more likely to be met in each production cycle.

The benefits of using a CMO are similar to the qualities of any good sports team. They enable pharmaceutical companies to have just the right number of players, equipment, training, and enough games in the season (i.e., manufacturing runs) to develop the medications they need at the volumes their patients require.

Taking a less-traveled path in the pharmaceutical industry brings its own unique set of challenges; however the benefits of not following the beaten path – and giving patients suffering from rare, life-threatening illnesses access to effective medicines that might not otherwise be available to them – are numerous.

One of the best ways for the industry to continue moving forward and transcending current pipeline struggles, sluggish R&D efforts and regulatory concerns is to consider how we all might approach drug development in a different way. Having successfully built and grown our business over the last seven years by not pursuing the next blockbuster, we’ve found that taking a look at disease states and patient groups lacking any effective treatment options, and seeing where and how we can serve them, is highly promising, as well as deeply fulfilling.

Mike Burke is Chief Commercial Officer at OVATION Pharmaceuticals, where he oversees the sales, marketing and business development groups who work to support and grow OVATION’s currently approved therapies and who identify new therapies to acquire, develop and bring to market. Prior to joining OVATION in 2001, Mike Burke spent nine years at Abbott Laboratories’ Pharmaceutical Products Division.