|
Abstract: Novel medical treatments that skip or mask genetic mutations are capable of solving previously incurable ailments. Genetic mutations are individually rare but collectively common, affecting 30 million individuals in the US alone. Genetic interventions provide treatments that save lives, particularly those of children. These interventions are developed for as few as one patient, earning the moniker “N-of-1 precision medicine.”
Such ultra-individualized treatments pose challenges for the existing system of 1) premarket regulation, 2) pharmaceutical incentives, and 3) tort compensation. First, the goals of N-of-1 precision medicine create legitimate concerns over whether precision medicine constitutes drug development, over which the FDA has authority, or the “practice of medicine,” over which it does not. Moreover, the onerous and slow premarket approval process conflicts with the emergency circumstances in which N of-1 treatments are currently used. Second, N-of-1 precision medicine treatments target too few individuals to justify the cost of drug development under the traditional patent system. Finally, patients seeking tort compensation for injuries caused by such treatments face significant hurdles for both products liability and medical malpractice claims.
On top of these challenges, the structure of N-of-1 precision medicine creates a further complication. Each N-of-1 precision treatment uses shared modalities to deliver individualized treatments; this means that information created in one treatment’s development can benefit the development of another treatment. This unique feature both creates the potential for information-sharing that can reduce development costs and simultaneously undermines incentives to do so. Addressing this shared modality feature holds the key to regulating N-of-1 precision medicine.
Given the promise of N-of-1 precision medicine treatments and their uneasy fit within the existing framework for population-based drugs, this Article proposes a new paradigm. Drawing from the platform economics literature, the Article reframes the interconnected nature of N-of-1 precision therapies as a positive network externality, which can be well-managed in a multi-sided platform system. Onerous ex-ante premarket approval would be replaced by standards-based good practice review of pre-registration designs, similar to the regulatory structure currently governing laboratories. Rather than relying on the patent system to provide incentives to create, laboratories would be paid for sharing data from pre-registered studies. Data sharing potentially reduces costs of development and helps insurance markets price the risk of covering such treatments. Finally, the gaps left by products liability and medical malpractice claims would be filled by monitoring the pre-registered designs. N-of-1 precision medicine can cure illnesses that previously constituted death sentences, extending lifespans and improving quality of life. This potentiality, however, will never scale without the legal infrastructure to facilitate development and ensure quality care.
|