DIA’s “What Lies Ahead” Document

Feb 12, 2014
Bart Cobert

Pharmacovigilance, Drug Safety and Regulatory Affairs Author & Expert

The Drug Information Association has just released an interesting document entitled “What Lies Ahead 2014: Trends to Watch: Medical Product Development in North America” http://telerx.bz/4k

It is a short review focusing on 10 trends (actually 11 as two trends are tied for tenth place) identified by thought leaders dealing with drug products from industry, academe, government and patient groups.  This is not a drug safety document and touches on drug safety only peripherally.  I’d like to take these topics (not being a DIA-selected “thought leader” for this project) and comment on these trends in regard to drug safety.  This is a well-written document that is worth the read.

1. Evolution of Patient/Consumer Engagement.  The point here is that patients are now inputting into the design of clinical trials, outcomes, and benefit/risk decisions in a formal way.  In the world of safety, there is clearly patient input since many if not most adverse events (AEs) come from patients, particularly in the post-marketing setting.  Interactions with patients certainly play a role in drug safety (DS) and pharmacovigilance (PV).  This is most seen in the OTC area where most AEs come in from consumers and patients and the company will often engage in dialogue on the phone to try to convince the patient to continue to use the product, to get marketing information or to promote other OTC products.  Feedback here is invaluable for the company.  Such interactions can also identify product quality issues, packaging defects or errors, product name confusion etc.  The challenge for industry is to better utilize patient information to enhance safety.

 

2. Learning how to Utilize Big Data. This trend is definitely a major one in DS/PV.  The use of databases, particularly big databases and electronic health records, will play a major role in pro-active identification of safety data where a large amount of patient exposure is needed to pick up rare SAEs.  Much money and work is now being spent on setting up the mechanisms for collecting or using massive databases (e.g. see OMOP http://omop.fnih.org/ The Observational Medical Outcomes Partnership amongst others).

 

3. Regulatory Agency Support of Innovation.  Related to number 2 above.  The FDA, NIH, the EMA and other governmental or quasi-governmental/NGO organizations are working actively in the field of DS and PV.  Much of the benefit-risk concept (REMS, RMPs) has come out of the agencies via CIOMS and ICH.  It seems, unfortunately, that industry has not played a leading role in PV innovation and academe even less so.  There seems to be little interest in medical school pharmacology departments or the schools of public health and nursing to look at drug safety.  One would expect pharmacy schools to be developing this area but not much is coming from that quarter either. (Hint, hint).

 

4. Importance of Collaboration. As in many other areas, it seems that the “low hanging fruit” has largely been picked.  The great efforts that individuals made in the past seem less frequent now as projects are becoming bigger and bigger (even massive) requiring teams, matrix organization, outside subject matter experts etc.  This means collaboration, good will, common goals, the ability to work in teams, lowering one’s ego are all needed for large projects to succeed.  This is true in drug safety and is being seen in OMOP and other projects.  As noted above, though, many players who could contribute enormously are absent.

 

5. Personalized Medicine/Tailored Therapies and Companion Diagnostics. Personalized drug therapy based on pharmacogenomics and other sophisticated analyses of an individual patient seems to be the most exciting concept to hit pharmacotherapeutics recently.  Perhaps one day we really will be able to predict how a patient will respond to a particular therapy based not only on how other patients responded (e.g. in clinical trials) but also on how a particular patient handles the drug.  One could imagine being able to predict adverse events, drug interactions, efficacy and even dose and therapy duration.  We are a very long way from this but at least the possibility is no longer science fiction but science prediction.

 

6. Clinical Trial Data Transparency.  This is a tricky area not for scientific reasons but rather for commercial, competitive, political and ethical reasons.  Many feel all data should be “free” and by free one means open and transparent.  Some also take “free” to mean without costs.  Most scientists and information technology people feel data should be free in order to benefit humanity.  However, when it is their own data, feelings sometimes change.   Nevertheless, there is a consensus forming that clinical trial data should be openly available early in addition to post-marketing safety data which is more open than trial data.  Clintrials.gov is an effort in this direction but does not have full safety data yet.  Doubtless this will come one day but not for some time.

 

7. Implementation of Risk-Based Monitoring in Clinical Trials.  This is an operational area that is being discussed and implemented whereby there will not be 100% validation of trial data but rather “smart”, risk-based monitoring and data review.  From a safety point of view, this may be worrisome.  Many times SAEs are found only when a skilled monitor visits a site and finds “hidden” or missed SAEs in patient diaries, in CRFs that were not picked up when they occurred and other obvious and, sometimes, less obvious areas.  This occurs for a variety of reasons and would be expected to get worse if there is less monitoring.  Nonetheless, this is the wave of the future.

 

8. Focus on Unmet Medical Needs.  By this, one means study of diseases where there are not effective or “safe” treatments now such as Alzheimer’s and Parkinson’s as well as cancer.  It would also include diseases of developing countries as well as rare diseases.  Clearly this is a good thing and suggests fewer me-too drugs and more innovative products.  If only we knew how to do this better.  The impact on pharmacovigilance seems a bit distant right now.

 

9. Data Standardization and Interoperability.  This is a “big deal”.  This is critical clearly for big data and effective data transmission.  This will be a major advance in drug safety and PV when it occurs – and it clearly will occur though it will take some time.  The methodology exists to standardize data.  The problems are largely political and economic.  Expensive and time consuming changes in IT systems will have to be done inconveniencing everyone.  We can easily transmit money anywhere in the world (bank wires) and we can also get cash from ATMs anywhere in the world.  But we still cannot easily transmit digitally an ECG from one side of town to the other.  When this is put in place and when we use “big data” drug safety and PV will make major advances.

 

10. Continued Importance of Global Markets.  Certainly true but the impact on drug safety seems unclear at the moment.  Always nice to get more cases and a bigger sample size to understand different rates and occurrences of AEs.  But not clear this is a big trend for PV.

 

11. An Explosion of Mobile Health Applications.  Definitely will play a role in AE reporting though how and how much remains to be seen.  If done well, we may indeed be able to get early or even real-time monitoring of AEs.  This is an exciting area and has much promise as the world moves to smart phones and tablets.

 

Comments

Missing from this list, in my view, is the impact of social media.  There are some early social media websites dedicated to AEs and drug safety.  Their value, beyond a forum for patients and health care professionals to vent, is unclear.  One could easily imagine use of, say, Facebook to assist in an urgent investigation of a particular SAE mobilizing this “database” of over a billion users.

So the list above is fascinating and has different implications for drug safety and PV from the rest of the pharma world.

This is my take.

What are your thoughts and comments?