The 21st Century Cures Act introduced a host of new opportunities for the life sciences industry. The Act is changing how drugs are developed and made available to the US. The Act also put in motion many concepts that, up to this point, have only been discussed, not broadly acted on. In this blog we will address the value of Real World Data (RWD) in clinical trials. The Cures Act can change how clinical research is done with it’s emphasis on patient engagement. Deloitte has written a very straight-forward review of the Cures Act. The review explains the changes, their impact, and industry implications. Here is a link to the Deloitte review –

The thinking around the value of RWD and PROs has evolved to support clinical trials. While this thinking has caught on in drug development it is harder to see the use once the drug is commercialized, although the implications of PROS and RWD can have huge positive implications. With the rise of values-based contracting RWD should be taking hold, but that is a discussion for another post.

While the life sciences industry realizes the value of PROs the Cures Act is accelerating the use. This data we are presenting here can be used to help you, in your own organizations, have support to show your leadership the value of these patient-focused efforts (because unfortunately the industry does not rate these efforts).

Both Genentech and Amgen have incorporated RWD into their clinical trials. Our information is derived from publicly available data – we can only make assumptions about these companies’ efforts based on that.


The Amgen/Novartis Neuroscience Partnership is in a three-way race to market in the new preventative migraine arena with erenumab. The other two competitors are fremanezumab (Teva Pharmaceuticals) and lasmiditan (Lilly). The timing for all three is tight so competition will be fierce – both for patients and for acceptance by the payer market. This is where Amgen made a departure from the routine.

Perhaps due to the provisions and requirements of the 21st Century Cures Act, perhaps simply realizing the value of PROs Patient Reported Outcomes, Amgen/Novartis developed and validated a digital PRO collection tool to use during the clinical trials; an app. At the 2017 Headache meeting Amgen submitted a poster summarizing some results from STRIVE and included patient experience data. If this data is included in the label, as we anticipate, Amgen with have RWD to persuade payers of the value of this high cost class of medicines (the latest estimate is $8500/year), specifically erenumab. Payers are more likely to pay for a drug that has data to prove benefits to patients, improvement in quality of life. (link to NEJM abstract of STRIVE study results)

Genentech has filed for its latest hemophilia drug, Hemlibra. It is clear the company collected patient experience data to include in the label. See the table below.

However, as much as the provision to collect patient experience data in clinical trials was established in 2016, the FDA forms still do not have the appropriate space to collect this information. This was a source of frustration for Genentech. Maybe this is due to the thinking on RWD has grown beyond “Did you include this data or not?”…finally. The data Genentech collected went beyond that to reporting more extensive RWD – Genentech has commented on this, click on this link to get access to the comments – . (link to Genentech article in Pink Sheet)


It is good to finally see RWD/PROs collected and included for filing. Clinical trials are sterile experiences. The use of RWD can take the labeling well beyond “safe and effective”. The data can allow the labeling to show how a patient’s life can change while taking this drug. In a world where we are concerned with adherence rates in every therapeutic area, including those were death is an obvious possibility, this RWD could be the motivation to support greater adherence, give the patient a reason why…a value proposition, if you will.