Around the globe, too many patients with serious illnesses have exhausted all available commercial therapies for their disease. In the case of a rare disease, no therapies may even exist. For these patients, access to a drug or biologic outside the clinical trial or commercial setting can represent a new, and in many cases, life-saving treatment option.
These patients are more educated and empowered with regard to their health than ever before. Coupled with greater transparency of pharmaceutical and biotechnology company pipelines, patients and their advocates are increasingly seeking access to medicines via alternative routes rather than waiting for them to be registered and launched in their country of origin, as the wait may be too long.
Additionally, physicians and other healthcare providers have greater access to the information about new medicines contained in international medical journals and presented at medical conferences.
This leads to significant challenges for companies developing new medicines. As a new therapy proceeds through each development phase through to global approvals, a very small proportion of the patients with need for access around the world actually obtain access to this new medicine.
Patient access to medicines across the lifecycle from Phase I testing through to discontinuation follows a typical pattern (A). The number of patients gaining access grows as the trial process moves forward and continues to increase following first (B) and subsequent launches (C). Actual patient demand for medicines that address an unmet medical need (D) often exceeds the level of access that is typically delivered. As the new therapy completes each phase of development, the awareness of trial data grows and the demand for access to the new medicine increases. Even following commercial launches in the US and Europe, the demand in other markets will continue to outpace the actual availability of a new medicine.
Why is this the case? A patient may not meet the entrance criteria for a clinical trial or may not be close to a trial site. There may be a delay in availability of a new medicine once approved, or it may never be made available in a patient’s home country. At the end of a product’s lifecycle, discontinuations present a challenge for patients who have come to rely on a medicine.
Managed Access Programs bridge these gaps along the entire lifecycle, allowing patients to access new therapies prior to their approval and launch, or when commercial access is not an option, all in a fully compliant manner.