We recently wrote about rare disease drug development and commercialization, and why such franchises succeed better within small companies or perhaps as stand-alone, independently operating units of larger firms. When the patient population targeted by a particular therapy is small, success depends on a highly patient-centric approach to both drug development and commercialization. One must identify and engage the right patients and facilitate their individual access to therapy. One must build relationships of trust, not only with the patients and those involved in their care, but with key thought leaders in the field. And one must work closely and flexibly with both regulators and payers, to help ensure that the drugs are reviewed and approved quickly and reimbursed appropriately. As a result, we believe that successful rare disease firms have a distinctly patient-centric culture and outlook on drug development. Large pharmaceutical firms, with their more rigid and standardized corporate processes and slower decision making practices, may find alignment with such models more difficult and not cost-effective for such small market opportunities.
And yet, we believe such patient-centric approaches to drug development and commercialization offer lessons for the future — especially in some cancers and other diseases where the use of companion diagnostics to tailor therapy to small numbers of individual patients with a particular gene mutation or other defining biomarker is fragmenting the market into much smaller, orphan-like indications. Engaging with patients can help drug developers better select indications and endpoints for clinical trials, as well as aid with patient recruitment. Commercial teams may also benefit from a patient-centric approach to these diseases; as more data aggregates around a specific patient population the company can learn more about the market and provide patient-specific offerings. Finally, with patients becoming more engaged in their own treatment (in oncology for example), such an approach can also take into account what really matters to the patient for their own therapy — be it safety, efficacy, budget or treatment that has as little impact as possible on their family.