After years of steady progress, the global biopharmaceutical industry is approaching its zenith. Regulatory rules are modernized. Investors are ready and willing. Industry’s pipeline is ever growing. And patients? They’re eager for more, and more advanced, treatment options.
There’s just one final piece of the puzzle: access. It’s pivotal, yet often overlooked by investors and manufacturers. Amid the intricacies, the business, of developing and launching a new product, manufacturers can forget a central truth. A good drug that patients can’t access is good for nobody. Patients can’t get treated, manufacturers can’t recoup costs, and investors can’t realize ROI.
Stakeholders, including healthcare providers and patient advocates in the United States know this fact well. From breakthrough cardiovascular drugs to unprecedented rare-disease treatments, advocates have honed their skills in using the access message to inform policymaking. So here I offer four lessons learned that may guide biopharmaceutical interests across the globe.
Effective access initiatives are proactive, sometimes launching before access barriers have even taken shape.
In the United States, for example, advocates may engage with the Food and Drug Administration during the approval process. Letters of support, or testimony at FDA advisory committee meetings, for instance, can establish the framework for the access message.
Regulators’ primary focus in the approval process is, of course, establishing the safety and efficacy of a given therapy. But hearing from motivated advocates can inform both their pace and their process.
Demonstrate the need
What could be more important than the quality, efficacy and side effect profile of a new therapy? The burden of the condition it treats. Yet even the savviest of manufacturers may zero in on perfecting their product, rather than on illuminating the problem it’s intended to address.
Advocates can paint this picture by drawing out factors like societal impact—the condition’s costs to the healthcare system, or how a disease affects employers due to absenteeism and lost productivity. Advocates can also delineate disease burden—how the condition disables patients or reduces their quality of life. Establishing these factors helps convey to policymakers the importance of patient access.
EBD Group, partnered with CBI, produces Rare Disease Innovation and Partnering Summit, June 13–14, where key stakeholders connect and propel curative progress, orphan product success and patient advocacy. Learn more about how you can attend here.
Find your story and storytellers
Demonstrating the potential impact of a new therapy does not mean overwhelming policymakers with charts and scientific data. It’s about telling a story. A powerful narrative weaves together the personhood of the patient, the impact of his or her condition, and the value of treatment—creating a poignant vignette. The story can shape public perception, and it can drive policy.
But the weight of this story depends largely upon who tells it. I would tell biopharmaceutical manufacturers this: The voice of patients and healthcare providers conveys authenticity. It invites compassion. And, as we have seen in the United States, it even topples access barriers.
Use data to keep the conversation moving
After advocates have established their narrative, data can play a role—so long as it’s targeted and contextualized. That may mean polling patients to confirm the quality-of-life impact of their disease or the barriers they face in accessing the medicine their physician prescribes. It may mean analyzing health system data to gauge how many patients are accessing the medicine they need.
This information can create a feedback loop with regulators and policymakers, keeping the access message fresh and reinforcing the importance of timely treatment.
Investors and manufacturers already know that investment, robust research and development, and a strong regulatory structure can impact success in the life sciences industry. But patient access—that can ultimately determine whether a medicine performs in the market as desired, and has the intended benefit to patients.
So I urge biopharmaceutical manufacturers to engage early—with policymakers, with healthcare providers and with patient advocates. Only then can you appreciate the total value a new drug offers. Only then can you be prepared to articulate that value to decision makers at every step of the process.