The journey of a medication from bench to bedside has always been long and expensive, but in the past decade, R&D expenditure has reached dizzying new heights—in fact, some estimates put the cost of developing a new drug as high as $1.3 billion.1 That's why it's vital for each product to prove it meets the needs of regulatory authorities, payers, doctors, and patients alike—something easier said than done.
Where it was once sufficient for a new pharmaceutical product to demonstrate value in terms of clinical advantage over existing products, high-profile withdrawals due to safety; better-informed patients; and tighter payer budgets mean that more is asked of new products, creating additional hurdles to be addressed post approval. For example, companies need to answer questions such as:
- Is this medication safe when used long term?
- Does it retain its efficacy over an extended period of use?
- Does it improve quality of life?
- Does it offer treatment satisfaction?
- How does the value of this medication compare to that of others?
- Which patient populations will it benefit most?
- How can we optimize outcomes?
Late phase studies can help answer these questions, demonstrating a product's long-term safety and extending its lifecycle by collecting data in support of patient reported outcomes (PROs). These are defined by the US Department of Health and Human Services as "any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else." And with the finalization of an FDA guidance document in 2009 (providing sponsors with a better understanding of the agency's expectations with regards to the use of PRO data in support of labelling claims), PROs are becoming an increasingly important part of marketing authorization submissions.
However, conducting such studies and capturing this data can be challenging. For example, considering the intense regulatory and payer scrutiny late phase studies face, they have relatively tight budgets compared to earlier phase trials. Additionally, their global scale and scope can create operational challenges due to inconsistent processes, communication issues, and planning difficulties.
The art of keeping patients
Particular challenges faced by late phase studies is patient retention due to long trial durations, the burden patients face and the fact that the study drug is not free (among other things). In some cases, these factors can produce dropout rates of up to 50% along with poor PRO quality. And with each patient in a Phase IV trial costing up to $12,000, it's clear why retention is vital to ensuring a study comes in both on time and on budget.2
Patient retention is all about motivation—motivating patients to continue with a study, motivating them to complete study-mandated tasks, and motivating them to report quality-of-life data. But motivation is a complex consideration that is dependant on many factors. In the case of a clinical study, for example, these include whether the patient feels they are respected as an important part of the study and how burdensome they find completing any study-related tasks. After all, clinical studies are not a patient's primary focus in life—their family, work, and hobbies are. That's why PRO data collection should fit around their lives, not the other way around.
We must also remember that we need to cater to our patients' needs and use the channels of communication they feel most at home with. The Internet, for example, has never been more fully utilized in our everyday lives—in 2009, 61% of Internet users went online to research a health topic.3 It should therefore come as no surprise that compliance with paper-based PRO collection is commonly less than 30%, whereas compliance with ePRO is more than 89%.4
There are several different tools used for ePRO data collection, including smartphones, tablet devices, and interactive voice-response systems. Another particularly important tool is the Internet—specifically, web-based portals (e.g., CRF Health's newly launched TrialMax Web™) that enable patients to enter ePRO data via their home computer and/or smartphone, quickly and easily. Such customizable portals combine both industry and patient insights to aid retention, ensure compliance, and generate ePRO data of the highest scientific integrity by fitting into patients' lives seamlessly and taking the burden out of data entry.
While motivation is key to encouraging patient retention, information is vital to ensuring engagement. That's why these portals do more than just collect ePRO data—they also communicate vital study information. For example, a portal can house study information that participants can use to educate their families on the trial's key points, while study events timelines allow patients to better incorporate the study schedule into their lives.
However, all retention methods are not suitable for every study, which is why customization is key to these portals' success, allowing them to be tailored to suit every study need and patient population.
Rachael King, CRF Health's CEO, said "web-based portals will change the face of ePRO data collection. Such portals will not only increase patient retention and protocol compliance, they'll also help to make the clinical study journey a little better for patients, too."
Rauha Tulkki-Wilke is VP, Product and Service Management, CRF Health, www.crfhealth.com/.
1. Tufts Center for the Study of Drug Development. "Drug Developers Are Aggressively Changing the Way They Do R&D," January 2011, http://csdd.tufts.edu/news/complete_story/pr_outlook_2011/.
2. Cutting Edge Information, "US Phase IV Budgets Top $12,000 Per Patient," September 2011, http://www.cuttingedgeinfo.com/2011/phase-iv-budgets-per-patient/.
3. Pew Internet and American Life Project. "61% of American Adults Look Online for Health Information," June 2009, http://www.pewinternet.org/Press-Releases/2009/The-Social-Life-of-Health-Information.aspx.
4. R. King, "Embracing Electronic PRO," Applied Clinical Trials, EDC & Information Technology supplement, 18 (3) 6-8 (2009).