App allows clinicians to discuss off-label treatments, but information is intended to be hypothesis-generating, not for regulatory use.

Process for sharing health care economic and other information has been simplified for manufacturers, and payers are seeing more economic modeling data earlier as a result. However, there is still uncertainty as to the scope of who may communicate the information, and to whom, under the agency’s 2018 final guidance, experts said.

One regenerative medicine for an ultra-rare disease may have a small impact, but payers will be flooded with dozens of new cell and gene therapies over the next few years. Enzyvant, Novartis and Spark share how they are positioning their complex products.