Pharmaceuticals Precision Medicine Regulation

How to regulate precision medicines

Currently, pharmaceutical treatments that are used in the U.S. need to gain an approval from the FDA.  The FDA’s approval is contingent on a demonstration of efficacy and safety in a randomized controlled trial (RCT).  However, precision medicine makes the standard FDA approval problematic.

As described in Breckenridge et al. (2016), in the precision medicine paradigm, patient response predicted by a specific patient gene or genes.  Thus, while RCTs approach has the advantages of strong internal validity and the establishment of causal inference, RCTs may not be sufficiently powered for treatments targeting rarer genes.

Alternatives include using real-world data such as data from patient registries and electronic medical records.  Alternatively, researchers can used a an adaptive clinical trial design based on Bayesian rather than frequentist statistics.

Patient reported outcomes could even be included in precision medicine trial designs.

Rather than considering patients as a large heterogeneous group, precision medicine provides the possibility of working with smaller, more homogeneous and better-informed patient populations with potentially unique urgencies and needs that are clearly expressed. Direct involvement of patients in regulatory activities has brought to the fore the need to assess symptomatic results using patient-reported outcomes (PROs) even in the development of drugs for the treatment of diseases such as cancer that have clear and highly relevant clinician-reported outcomes.

Another potential problem with precision medicines is their high price tags. If drugs are priced to value, precision medicines could generate significant value for a smaller subset of patients.  Thus, precision medicine is likely to increase the cost per user price to sufficiently reimburse the innovator, but the effect on overall drug spending is unclear as these precision medicines will be used on a smaller patient population.

Precision medicines may be better adapted to “learn and confirm” regulatory approval processes such as the FDA’s Breakthrough Therapy and EU’s PRIME designation.

Finally, patient risk tolerances will likely play a role in whether certain precision medicines are viable in the market.

When medicines have to be taken long before patients are confronted with the morbidity and mortality of their condition, their tolerance for the uncertainties about benefits and harms of new products may be limited, requiring new approaches to long-term monitoring of benefits and risks.

In short, precision medicines offer the opportunity to significantly improve patient outcomes, but regulatory and reimbursement challenges loom.

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