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The dilemma of creating drugs that please patients and regulators

The dilemma of creating drugs that please patients and regulatorsSometimes, in biopharmaceuticals, it feels like we’re staring in the rearview mirror.  At the recent annual Biotech Showcase meeting in San Francisco, scores of speakers and panelists with important news to share during the three-day event found themselves instead of addressing investors’ worries about negative public perceptions and dangers of a pricing backlash—topics that held particular urgency in the first half of 2017.

I was struck by this phenomenon when I sat on a panel titled “The Future of Biotechnology: Are we at a tipping point?” where one might expect to gaze forward and probe the most important themes for 2018. Over and over, the conversation veered away from clinical and commercial strategies that will benefit patients in the next 12 months. Most of the big issues for the panel were last year’s topics: namely, biopharma’s image problem and how this could negatively affect federal and state policies.

I’m not saying that pricing debacles are secondary, or that our reputation challenges are behind us.  But there are many other important topics to consider. We need to do a better job communicating complex scientific breakthroughs to the public. And, just as important, we must figure out how to raise success rates when launching newly-approved medicines, so patients can actually benefit from the underlying medical breakthroughs.

Success at launch

Here, the industry track record is discouraging—and not because of pricing conundrums, anti-pharma sentiment, or unfavorable federal or state policies. For a variety of reasons including commercial missteps, two out of three drugs launched between 2003–2009 failed to meet revenue forecasts. Of the promising new drugs approved in 2011 and analyzed in a report in Nature Reviews/Drug Discovery, nearly half failed to achieve $200 million in sales. I haven’t heard any recent data suggesting this problem has gone away.

The fact is, a drug’s success in the market matters a great deal to patients battling diseases the medicine might ameliorate. Helping companies achieve success is also a priority for my company, Syneos Health™. We’re the first clinical research organization (CRO) that also serves biopharma clients as a contract commercial organization (CCO), with capabilities ranging from regulatory and reimbursement consulting to product branding, advertising, and public relations, as well sales force and nurse outsourcing.

Many drug launches fail because the market is already crowded with look-alike products. In some ways, this is a commercial problem. By now, managers tasked with selecting the most promising molecule should have a better understanding that payers, physicians, and patients all value new drugs that work better than existing treatments.

The problem isn’t confined to “me-too” medicines. Novel drugs also fail because payers don’t understand the products, or word never reaches patients who need them—a missed opportunity that can’t be blamed on the organization’s research team.

Bringing commercial to clinical

The solution may be simpler than many people realize. I believe companies can increase chances of a successful launch by bringing insights from market access and other commercial functions directly into phase II and phase III clinical research. This way, the treatment that eventually wins FDA approval is more likely to match what payers, providers and patients say they want.

Sadly, our industry is still quite a long way from this point. Last year, a study in the peer-reviewed journal BMC Medicine critiqued the biopharma industry’s heavy reliance on surrogate endpoints such as progression-free survival in cancer clinical trials. Surrogates are useful in a regulatory review. However, too often, the industry defaults to surrogates without running postmarketing surveillance studies to correlate the results with the quality of life and overall survival.

Commercial teams understand these correlations matter for payers and physicians. And they can help research colleagues assure that the necessary bases are covered when a drug reaches the market.

In fact, there are many insights commercial groups can glean that will enrich clinical research and position drugs for success. By analyzing insurance claims and prescription data, we can predict quite a lot about which endpoints matter to payers and prescribers in different therapeutic areas.  While drugs are in early stages of development, we can also survey these same stakeholders, as well as patient organizations whose opinions they value, to produce an even deeper perspective on how different stakeholders define “value.” And, of course, we can shed light on real-world efficacy by beefing up postmarketing surveillance studies within our commercial organizations.

I was pleased to see this subject resonated with a few other members of the Future of Biotech panel. Leslie Stolz, head of Johnson & Johnson Innovation Center/JLABS California, at one point explained to the audience why this type of integration matters to big pharma as well as small biotechs. The challenge is “bringing commercial back in [and] taking what we learn back into clinical trials,” Stolz said. “Johnson & Johnson works really hard to get rid of the silos now that were in between the [research and commercial] organizations and bring everyone back in order to accomplish that.”

As many panelists pointed out, 2017 was a stupendous year for biotech tools and key technologies, from CRISPR gene-editing and sub-$1,000 genome sequencing to the first commercial forays into gene therapy, stem cell therapy, RNA-interference, to the advent of AI-assisted drug discovery.

What we all must remember is that technologies aren’t treatments, and regulatory approvals don’t automatically mean lower morbidity and mortality.  Medical breakthroughs should be more than just interesting science experiments. To truly benefit patients, we must apply the full complement of commercial insights to drug development and make sure effective drugs succeed at launch.

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