BioPharma

Oncology, rare diseases and too little genomic diversity: 3 themes from #JPM17

From too little genomic diversity to an overemphasis on rare diseases and oncology, here are three trends from the 35th J.P. Morgan Healthcare Conference that will continue to play out this year.

Meet Overhead view of two business persons in the lobby

As always, the J.P. Morgan Healthcare Conference played host to wider debates about the health and direction of the life sciences in 2017.

Drug pricing was part of the discussion (Trump made his splash mid-week). Gender diversity came to the fore in 2016 and made headways at this year’s event.

Beyond the conference agenda and the breaking news, however, three underground themes were playing out at Bioweek.

Genomic diversity
In back-to-back sessions, the issue of ethnic diversity in genomic research came to the fore. The original human genome was caucasian and that set the tone for the millions of genomes that have been sequenced since.

For a multitude of reasons, nearly all large genome-wide association studies (GWAS) have followed white populations. A seminal 2009 study found 96 percent of GWAS participants were of European descent.

Pacific Biosciences President and CEO Michael Hunkapiller raised the issue as he spoke about the overall progress of the genomics space and the path to understanding more about structural variants — long stretches of DNA that have been altered, as opposed to single nucleotide polymorphisms (SNPs).

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At the diagnostics panel, executive director of the Center for Clinical Effectiveness at Blue Cross Blue Shield Association, Suzanne Belinson, highlighted diversity as part of the challenge healthcare companies now face interpreting and bringing meaning to the information generated through sequencing and diagnostics.

To me the most exciting activities that have happened over the last year are some of the collaborations around data. It requires a tremendous amount of data to solve the puzzles that we are all trying to solve. It takes a tremendous amount of data from a diverse population. The majority of the data that we have now to answer some of our genetic questions come from a very homogenous population and if we want to actually solve these problems for the diverse population in which we live, it requires a large and diverse set of data. I’m really excited about some of the organizations and health systems that are coming together to collect that data.

Over-incentivizing rare disease research?
The Orphan Drug Act was passed in 1983 to make the economics of rare disease research more favorable for drug companies to pursue.

It has had a major impact. According to a 2014 Evaluate Pharma report, just 38 rare diseases had approved therapies when the act was passed. By 2014, the number had jumped to 373.

That’s an undeniably positive shift and there are many orphan diseases that still need treatments. But at what point do we factor in the global burden of disease?

Several attendees that I spoke with were grappling with the issue, including Michael Narachi of San Diego, California-based Orexigen. Against all odds, Orexigen managed to get a weight loss drug approved as a small- to mid-sized biotech. The scale and cost of clinical trials in major conditions typically require the involvement of Big Pharma.

Narachi said many of his colleagues in the industry are now actively targeting niche subpopulations within a disease. It increases the odds of getting an approval and paves the way for more streamlined clinical trials. The downside for everyone is that the company then has to charge six-figure sums for the therapy to offset the small market size. And a limited number of people benefit.

The age of the blockbuster drug is over. Will there be some intermediate solution found? Or will personalized medicine continue to drive expensive therapies for targeted groups?

The J.P. Morgan oncology conference
Oncology increasingly dominates biopharma and healthcare discussions.

Annual spending on oncology drugs surged 11.5 percent to $107 billion in 2015, according to a report by the IMS Institute for Healthcare Informatics. The report identified 500 companies actively pursuing a combined 600 late-stage investigational oncology drugs, suggesting the growth will continue in the coming years.

The oncology emphasis carried through to the agenda at J.P. Morgan, drawing crowds to CAR-T presentations and those by major pharma companies such as Merck, which is playing a large-scale chess game getting approvals for its anti-PD-1 checkpoint inhibitor. Combination cancer therapies were another major presentation theme.

On Monday, Takeda announced it had acquired Ariad Pharmaceuticals for $5.2 billion — the deal of the week. Ariad specializes in treatments for rare cancers and leukemias.

Meanwhile, Vice President Joe Biden effectively froze downtown San Francisco as he made his was to the StartUp Health Festival and the main conference to discuss his cancer moonshot.

These were all pre-planned. What was more surprising was the emphasis given to oncology in other open discussions. The Diagnostics Outlook Policy Panel on Tuesday, for example, almost exclusively focused on cancer applications.

Oncology wasn’t mentioned in the abstract for the panel — it was about new policies and laboratory developed tests. But the five experts discussed these topics in relation to cancer diagnostics, interpretation and care.

Granted, two of the five speakers were from companies in the oncology space; Elad Gil from Color Genomics, a 30-gene cancer diagnostics company, and Jeff Huber from GRAIL, the Illumina spin-out that wants to make blood tests for early-stage cancers routine.

As far as the composition of the panel swayed the discourse — that in itself is telling.

Perhaps the emphasis has something to do with the relationship between genomics and cancer research. On Monday IBM Watson Health and Illumina announced a new partnership for the Watson supercomputer to rapidly and reliably interpret results from Illumina’s new cancer panel, the TruSight Tumor 170 panel.

In an interview with MedCity News, Watson Health Vice President Steve Harvey said the technology could theoretically be applied to any disease area with a genetic basis. Watson Health and Illumina chose cancer as an area they believed they could make the greatest impact. It is, after all, fundamentally driven by genetic mutations.

Photo: Piranka, Getty Images