Achieving regulatory approval alone no longer determines a drug’s or therapy’s commercial success or even guarantees its market launch. Today, each product must be evaluated from a value perspective by payers and health authorities to be placed on the formulary, factored into reimbursement rates, and put into treatment plans before it is available for healthcare providers to prescribe. In this blog, I’ll discuss recent trends for using health economics and outcomes research (HEOR) and real-world evidence (RWE) to evaluate the value of medicines and health technologies.
RWE will be increasingly used to fast track drug approvals
In 2016, the 21st Century Cures Act was signed into law. One of its mandates was for the US FDA to develop a framework to evaluate how data from sources other than clinical trials can be used to support drug approvals. RWE is increasingly being used to fast track drug products especially in rare diseases. Thus, a drug may be able to gain marketing authorization on the strength of phase 2b results instead of having to wait until phase 3 clinical trials are completed. Therefore, sponsors won’t have to conduct huge and hugely expensive late stage clinical trials. They can collect real world data to show that the drug works just as well in our daily lives as it does in trials. In addition, this approach means that patients receive critical medications sooner and more cost-effectively.
Using RWE to support indication expansion
In addition to using RWE to fast track drug approvals, it will also be leveraged for indication expansion: a regulatory approval for a drug for an indication other than what it was originally approved for. Typically, pharma companies achieve this by conducting additional clinical trials that demonstrate efficacy in the new indication. But, conducting additional studies is both expensive and time consuming, not to mention operationally challenging for very rare diseases.
Another strategy is to first secure marketing authorization in one indication, either the highest price indication, or the one with the highest unmet need (these are often the same). Then expand to patients with other targeted indications using real world data. This approach allows companies to reduce the required number of large phase three clinical trials, which is hugely cost saving.
By capturing real world data, you get a better sense of the experience the patients are having on a medication. This allows assessment of the actual medication compliance as opposed to the compliance observed in a carefully controlled clinical study. Thus, the drug company gets a much better understanding of the likely response and patient outcomes under typical conditions.
Technology is making drug development become more patient-centric
Sometimes what’s important to patients is different than what we imagine their priorities to be. There’s a trend towards making drug products and medical technologies more patient-centric. One way to do this is capturing the patients’ feedback. So, patients are being increasingly asked to use smart devices to assess quality outcome measures (QOMs): when they take their medicine, how they’re responding to medicines, etc.
In addition, pharma companies are involving patients earlier in the drug development process to understand how they are managing their diseases, particularly for rare disease patients. For example, imagine a hemophilia patient who has inject herself with medication every day. Maybe she has to spend a lot of time each day finding a vein. Thus, this cumbersome treatment ends up impacting her medical condition as well as her social life. By improving the method and frequency of administration of the drugs that patient uses— perhaps making the medication longer-acting – we can improve her experience.
Capturing the patient’s experience is key to demonstrating the ‘real’ value of your product where it matters the most!
Medication’s value to society: From “nice to have” to “must have”
Health care and social care are separate budgets in the majority of health economies. Thus, the impact of medications on society hasn’t been a major concern in the past. However, some health systems, such as the U.K., are beginning to combine health and social care budgets. And, it’s likely to become more of a factor in the drug approval process in the next decade.
Pharma companies need to consider the patient and social impact of their investigational drug programs. This way, by the time their product hits the market, they’ve generated the evidence for a cost-effectiveness story around health and value to society. Does your drug help patients maximize the time at school or work? Does it decrease the burden on care givers?
Consider as an example a new drug for chronic obstructive pulmonary disease (COPD) that’s not differentiated from the competition. What if its manufacturer provided a system where patients can get advice online or by phone and receive reminders about when to take their meds? This value-adding system might be the differentiator that convinces the health system to reimburse this new product rather than a competitor.
Health technologies are leveraged to tackle the problem of medication non-adherence
Medication non-adherence has been estimated to cost the US healthcare system between $100-300 billion annually.1,2 Improving medication adherence saves health systems money and supports better patient outcomes in many disease states. Health technologies are increasingly being used to help patients take their medications. For instance, the patient can record on a phone or a device every time they take their medicine. Health care providers are also using text messages to remind their patients to take their medications.
The result of improved adherence on health outcomes can include fewer doctor visits, as well as a reduction in the need for poly-pharmacy and rescue medicines. And, it supports the commercial success of drug products because improved adherence leads to better outcomes. Getting volunteers in a clinical study to take their medication is relatively easy, but payers want to know that it’s going to work the same in the real world.
Innovative contracting and risk-sharing agreements
Risk-sharing agreements between the pharma industry and governments are becoming increasingly popular. They stipulate that if products don’t perform well in the real world, then the pharma company has to pay back some money to that country’s health system. So, it’s in the company’s interest to develop practices that help ensure patient adherence and to capture the real world data to prove this.
In conclusion, by embracing advanced Market Access and HEOR strategies early in product development, drug companies are more likely to achieve regulatory and commercial success while better serving patients in need.
1. Iuga AO, McGuire MJ. Adherence and health care costs. Risk Manag Healthc Policy 2014;7:35–44.
2. Viswanathan M, Golin CE, Jones CD, et al. Interventions to improve adherence to self-administered medications for chronic diseases in the United States: a systematic review. Ann Intern Med 2012;157:785–95.