In an earlier blog, I reviewed the positive aspects of extending the trend of crowdsourcing to clinical trials; these included drawing public input regarding clinical trial design and incorporation of relevant quality of life (QoL) measures, as well as expedited recruitment and sustained retention. However, there are always two sides to every coin. In the interest of providing a balanced perspective, I will review some of the drawbacks of this increasingly popular movement.

First and perhaps most importantly is the difficulty with standardization. How do we compare trials for the same disease developed using input from different groups of patients? Using an example from the earlier blog, Transparency Life Sciences used their collaborative platform in the design of a Phase II study assessing the utility of the antihypertensive drug lisinopril as adjunctive therapy in multiple sclerosis, which included a novel efficacy parameter suggested by patients.1 If another company used a similar platform to design a trial for MS patients, and that group suggested a different efficacy parameter, how do we compare the results of these two trials? This not only increases the challenges for interpretation by clinicians, but also for the regulatory bodies reviewing and approving drugs for use in the market.

An extension of that problem is the assessment of relevance of these new markers by the regulators and payor organizations. While there is a strong impetus from patients for trials to examine additional QoL measurements, a disconnect exists between the patients and the regulators. Government bodies controlling patient access to new drugs need to see improved efficacy results such as reduced tumor measurement, progression-free survival or extended overall survival, to prove the worth of a new drug. While QoL measurements provide a reflection of the patient experience, they do not accurately signal the status of the disease.

Within the already burdensome regulatory system, one could imagine that increased patient input will bring about more rules and higher standards, sponsors must adhere to when incorporating patient or “non-expert” feedback to protocol design. However this debate is decided, patients are increasingly becoming drivers for change and governments and the pharmaceuticals industry need to catch up.

References

  1. http://www.transparencyls.com/lisinopril-multiple-sclerosis