Last month, we discussed patient-reported outcomes and some potential implications of the final FDA guidance on using patient-reported outcomes to validate claims in medical product labeling. I read an interesting blog article today about Foundation Medicine’s plan to genome-sequence every cancer tumor. The blog’s author, Trista Morrison, stated:

If they can pull it off, this could totally change not only the way cancer is treated, but the way cancer drugs are developed and approved.

Foundation Medicine’s goal is to create a way for a cancer patient to compare their normal genome to their cancer genome, then see all the mutations. Ultimately, this comparative information could provide a personalized guideline for treating the cancer - essentially personalized medicine based on a person’s DNA.

It is easy to see how this will require a more personalized approach to clinical trials. If such an inventory of medicine is to be created to target each type of tumor a person has based on that individual’s own DNA, how will such medicines get tested? The traditional clinical trial format relies on the participation of hundreds or thousands of patients who respond to treatment in similar enough ways to prove a treatment can do what it’s promoted to do. That likely will not be the case with this new way of treating cancer.

The future clinical trial format will need to be more “personalized.” Rather than relying on treatment data for a large pool of patients, patient-reported outcomes will play a more central role. Patients will need to carefully keep track of their symptoms and their treatment in order to produce a sufficient amount of information about the efficacy of medicines on a person by person basis. Investigators and clinical research coordinators will even find that it could be easier to recruit participants for clinical trials if they can screen patients using eDiaries or journals.

In January, the Food and Drug Administration published its final guidance on the use of patient-reported outcomes (PRO) instruments to support claims in approved medical product labeling. Although the guidance does not address the use of PRO instruments beyond this scope, it does lend itself to some interesting connections and observations about using PRO tools for other purposes. For instance, Ntrypoint Media has been talking to life sciences firms over the past year about capturing patient-reported outcomes before, during, and after clinical trials to accelerate recruitment and maximize retention at a lower cost.

The FDA defines a PRO 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.

The FDA says sponsors should begin PRO instrument development and evaluation early in their medical product development life cycle, prior to clinical trial enrollment, and involve patients in the process who represent members of the intend population for the trial. If an online research community is used to capture patient-reported outcomes and general feedback on an ongoing basis, this requirement would be inherently satisfied. Also, actual recruitment can occur faster if the very target population for the trial is a captive audience who helped developed the PRO instrument from the start.

There are special considerations for electronic PRO tools. We are all seeing more and more of our information transfer to digital media and repositories. This can make recording PROs easier. Nevertheless, offline protocols still must be adhered to even if an ePRO instrument is incorporated. For example, direct control over source data should be exercised by the clinical investigator, not the sponsor or the contract research organization. An ePRO tool in the form of an online research community can simplify the process of screening patients for recruitment and give the clinical investigator the appropriate level of control.

The FDA’s published guidance demonstrates that the government is taking electronic health records (EHR) and personal health records (PHR) seriously. We can likely expect the FDA to issue similar guidance on how to use PRO for other purposes. Using PRO to identify and screen clinical trial participants is an obvious place to focus. This approach can support claims of the presence of hard-to-diagnose conditions, the environmental causal relationship on health conditions, and the likelihood of patient compliance throughout the trial.

There is no shortage of articles written about how pharmaceutical, biotechnology and medical device makers supposedly fear the Internet and social media. That’s why I am glad to see social media playing an increasing role in patient outreach as evidenced by sites like UC and Crohn’s, Novel Patient and PatientsLikeMe. Recent research published on the Pew Internet & American Life Project website found that 64% of Americans search the Internet for health information and defined these internet users as “e-patients.” It goes on to say that this group is more likely than other internet users to engage in social media.

So why is the life sciences industry still treading slowly if the Internet is more or less ripe for engaging e-patients with social media? Likely, it has little to do with fear in the sense that one is afraid of the dark, and more to do with strict regulations that govern their clinical research activities. In general, social media doesn’t fit the clinical research mold because of information security, regulatory compliance and no consideration for Good Clinical Practice (GCP).

• Encryption at all levels of communication, from login to logout, should be applied to protect information shared through social media and using online communities
• Specific data protection, role-based access, intended use, auditing and disaster recovery principles must be followed for HIPAA compliance
• A key component of GCP is protection for patients through Informed Consent

A basic review of most, if not all, social media would show they have a lot of work to do in these areas and the life sciences industry has good cause to be guarded in using tools like Twitter, Facebook and YouTube. However, I came across an article published in May of this year offering 10 suggestions for using Twitter for clinical trial patient recruitment. It offers good suggestions to get the word out about clinical trials, but I would say not as good as an online  research community. The community has to be designed with the considerations described above first and foremost, but also allow clinical investigators to stay engaged with patients before, during and after specific studies are done. The feelWell Online Online Research Community is an example of this approach that bridges the gap between social media and clinical research. Researchers can learn more here. It helps speed up patient recruitment, increase pre-screening intelligence and retain patients throughout the clinical trial and for future studies.

It is time life sciences firms take a more serious look at social media in the form of an Online Research Community like feelWell Online.