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Report Finds More Personalized Rx on the Market, but Field Lacks 'Integrator'

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Originally published Nov. 10.

NEW YORK (GenomeWeb) – Consulting firm Diaceutics has released a report that shows pharmaceutical companies' readiness to develop and deliver personalized medicines has improved since 2010, at least according to the numbers.

Between 2010 and 2014, the number of drugs associated with a biomarker, as well as the list of PGx drugs on FDA's website have quadrupled. However, success and failure in the personalized medicine field isn't as straightforward as the top line numbers suggest, Diaceutics CEO Peter Keeling reflected at the recent Convergence conference in Dublin, Ireland.

While guiding many of the top pharma and diagnostics shops through personalized medicine projects, Keeling has been consistent with the message that success isn't achieved by simply developing a great drug or a novel test. It's a more complex and sometimes elusive prospect, he tries to tell Diaceutics' clients, that requires industry players to introduce a treatment strategy in a healthcare ecosystem teeming with different stakeholders and divergent interests.

At the meeting last month, where Diaceutics brought together different healthcare stakeholders working on personalized medicine efforts, Keeling pointed out that during the last four years a number of molecularly targeted drugs had come to market, but companies still hadn't figured out how to advance the field beyond cancer, into complex, chronic diseases, such as Alzheimer's disease and rheumatoid arthritis.

Comparing the healthcare enterprise to a city, Keeling noted that when it came to personalized medicines, "pharma is still building one street at a time" – meaning that while drugmakers are developing more molecularly targeted therapies, the industry isn't collectively focused on trying to change the healthcare infrastructure so such treatments can become the standard of care instead of the one-off example.

"Think of the infrastructure of a city," he said. "It needs water systems, grand boulevards, and little side streets" to function.

Undoubtedly, as Diaceutics' report confirms, there are more personalized medicine options today than there were four years ago. From 2010 to the second quarter of 2014, drug developers have launched seven new drugs with companion tests. Meanwhile, the FDA has revised labeling for 53 marketed treatments with biomarker-associated safety data.

Personalized medicine products comprised 6 percent of the top 12 pharma companies' portfolios in 2010. Fast forward to 2014, these types of products now account for 19 percent of pharma portfolios, according to Diaceutics' report. Four years ago, 54 percent of Phase III/registration trials at these firms had biomarker-linked drugs or therapies that could potentially be prescribed as part of a personalized medicine strategy. As of 2014, that number has grown to 74 percent.

Looking at 12 of the top drug developers – Sanofi, Janssen, Roche, Novartis, Bristol-Myers Squibb, Pfizer, Boehringer Ingelheim, GlaxoSmithKline, Amgen, AstraZeneca, Lilly, and Merck – Diaceutics found that most companies increased their investment in personalized medicine over the four-year period with regard to the number of such therapies in their late-stage or registration pipeline.

Diaceutics then ranked these firms (with at least one molecularly targeted treatment on the market) on their readiness to capitalize on personalized medicine opportunities and categorized them into one of three groups – disrupters, breakaway firms, and followers. Three companies – Roche, Novartis, and Janssen – are now disrupters, or firms that "have demonstrated the ability to competitively reshape therapy areas with personalized medicine," Diaceutics found. Janssen is a new addition to this category since 2010.

BMS, Pfizer, and AstraZeneca have now made investments to be considered "breakaway firms," and are in the process of changing their business models and operating structures to advance personalized therapies. Boehringer, Merck, GSK, Amgen, Sanofi and Lilly are the "followers," that are more likely to "respond to the actions of others" and don't have much internal organization to commercialize personalized drugs, according to Diaceutics.

Among these companies, with the exception of Boehringer and Janssen, which Diaceutics' first report did not include, Amgen was the only firm that had fewer precision drugs in late-stage development in 2014 than it did four years ago, Diaceutics found. Still, as of this year, all companies on the list featured in the report can now boast that 50 percent of their Phase III portfolios are being advanced with a biomarker or personalized medicine strategy.

The growth is encouraging, but Keeling still sees hesitation among drugmakers to invest in the field. "No one wants to leap first," he said. "They're all looking to who's done it before." Certainly, the paths pharma seems to be treading most heavily in the personalized medicine space are those already well paved, such as in HER2-targeted breast cancer, BRAF-mutated melanoma, lung tumors driven by EGFR mutations, and leukemia.

At the American Society of Clinical Oncology's annual meeting, for example, at least three companies presented data on next-generation EGFR inhibitors that have shown in early trials to be more potent and less toxic by hitting mutant EGFR tumor cells with increasing accuracy. These drugs come after first-generation EGFR inhibitors, such as Tarceva (erlotinib) and Iressa (gefitinib), which for a decade now have shown to work well in patients with EGFR mutations.

And in markets where there aren't blockbuster personalized medicine success stories like Tarceva, Herceptin (trastuzumab), or Gleevec (imatinib), biomarkers strategies seem to catch on only after a drug failure. For example, although research data suggested that patients with BRCA mutations could respond well to PARP inhibitors, it wasn't until an interim analysis of a Phase II study involving AstaZeneca's olaparib failed to suggest a survival advantage that the drugmaker publicly started talking about using a companion test to identify best responders. Now, Myriad Genetics, a firm that markets the most widely used BRCA test, boasts companion diagnostic deals with five drugmakers advancing PARP inhibitors and other DNA-damaging agents.

Part of the reason for pharma's hesitation in investing in personalized medicine, according to Keeling, is that the field requires the industry to step out of its comfort zone as developers and sellers of drugs, and to figure out how the healthcare system can be corralled to deliver treatment strategies that involve the participation of researchers, diagnostics firms, labs, regulators, payors, and even pharmacists, pathologists, physicians, and patients. "Delivery of personalized medicine is no longer about technology, it's about the model of delivery," he said. "If the goal is to improve patients' outcomes then there has to be cooperation. [Industry] has to stop thinking the pill or the test and think of the ecosystem."

There are signs that pharma is looking beyond the pill and the test and working through the systemic complexities. There is growing recognition among pharma players, for example, that the one-drug-one-test paradigm is not sustainable, and they are collaborating with platform developers such as Thermo Fisher Scientific and Illumina to develop universal testing platforms that could guide personalized strategies for multiple drugs – even when those drugs are developed by competitors.

There seems to more appreciation among pharma of how important lab networks are to the delivery of personalized treatments. Drugmakers are even getting involved in personalized medicine education as seen with Boehringer's Let's Test campaign in lung cancer, which provides pathologists and doctors information about the importance of testing lung cancer patients early for EGFR and ALK mutations.

Pharma has hired diagnostics experts to advise them with their drug development efforts. Companies that are investing most heavily in the personalized medicine space, such as Roche and Novartis, have acquired molecular diagnostics shops, and other drug developers are following their lead by also building (or rebuilding) internal diagnostic capabilities. And on the diagnostics side, Illumina's purchase of regulatory consulting firm Myraqa in July signaled that the testing industry is also figuring out the complexities of the personalized medicine ecosystem.

Despite these advances, personalized medicine is still weighed down by its reputation for not making the kinds of profits drugmakers are used to. Most of the marketed personalized medicine products are currently cancer treatments, and while there are blockbuster examples in Herceptin and Tarceva, "oncology has reinforced the idea that personalized medicine is a niche business," Keeling said. Meanwhile, there are more than 35 million people worldwide with dementia, and a biomarker-guided drug that can identify which patients are likely to develop cognitive decline due to Alzheimer's will not be a niche product.

Still, from where the field stands currently, the road to a personalized Alzheimer's drug appears daunting, fraught with financial risks, technology hurdles, and scientific challenges, not to mention regulatory uncertainties. Although there is money to be made from investing in personalized medicines in non-oncology indications, pharma hasn't yet embraced the complexity of the healthcare ecosystem in a way that will allow it to do so, according to Keeling. Ultimately, what the field is missing, according to Keeling and many other participants at the Convergence meeting, is an "integrator" – an entity that can bring together the divergent stakeholders and foster collaborations in a way that eases some of these systemic challenges.

Groups like the Multiple Myeloma Research Foundation are already acting in such integrator capacity, by joining academia, industry, and community healthcare providers to expedite development and delivery of molecularly targeted treatment strategies. For a time, before its acquisition by Express Scripts, pharmacy benefit manager Medco was playing such a role, by fostering research collaborations to gather clinical utility evidence backing the implementation of molecular strategies and using pharmacists to identify the opportunities in the Medco system where patients would benefit from personalized approaches.

At the Convergence meeting, participants voted and agreed to identify and gain the commitment of an "integrator" entity to kick start personalized medicine efforts in two disease areas – one in the oncology space and another in a non-oncology indication – and crowdfund the project. For this so-called Integrator Project, Diaceutics has formed a committee that will identify the specific disease areas of focus and reach out to possible integrators for their participation. Keeling so far has been able to secure $30,000 in funding for these projects, and is hoping to raise $200,000 in total to get the efforts off to a start.

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