Patient Registries: A Smart Investment for Rare Disease Research

In the current health care environment, marketing approval for a new drug is a critical milestone, but it may not guarantee that your product realizes its full potential after launch. This is becoming truer now for rare (orphan) disease indications in which the market sizes are already very small. The challenge: the race for a leadership position is more crowded. Sparked by the commercial successes of companies like Genzyme, Shire, and BioMarin, research in rare disease indications is much more common and several small companies may be vying to be the first to launch a new drug product in the same indication, leading to multiple competing trials in the one, small patient population and a struggle to assemble the data and clinical evidence to support their individual product candidates.

Unfortunately, there is often very little published medical literature about patients with rare diseases and guiding a successful drug development plan and preparing for a new product launch require a much broader approach. The smartest companies also understand that every research participant and every piece of data is critical in building the required clinical evidence to fully support new drug treatments for rare disease indications. Therefore, a growing number of these companies are sponsoring related research and education activities – like patient registries – prior to marketing approval (and now increasingly in collaboration with patient organizations) to help them claim a leadership position among the many rare disease research contributors.

As one example of a potential return on a registry investment, biopharmaceutical companies spend a lot of time engaging with regulatory authorities throughout their products’ clinical development plans to define the clinical endpoints and trial designs that will offer the most efficient paths to demonstrating efficacy and safety and obtaining marketing approval. Data from an early patient registry can uncover important clinical questions or trends and inform the design of the planned randomized clinical trials. In some rare disease programs, a well-designed and managed registry may support discussions with regulators regarding the use of the registry data as complementary and supportive to the randomized clinical trials, as well as provide the framework for anticipated post-approval mandates from the FDA or EMA.

From the commercialization perspective, perhaps one of the larger priorities during the latter phases of clinical development is identifying all potential patients who may benefit from the new drug product after approval and launch – not just those known patients participating in the randomized clinical trials. A patient registry can engage treating physicians and their patients worldwide to better define patient referral and diagnostic pathways and to help develop medical communication plans and product distribution channels. In addition, a patient registry may provide the ability to proactively assess emerging issues that arise from observing routine clinical practice. For example, who will likely be the most frequent prescribers of the new product and for which patients? What clinical evidence will be most important to support reimbursement among the worldwide markets? Will the new product provide long-term value or just add health care costs? This information can help build the product’s value proposition and set a foundation for assessing changes in treatment guidelines and physician behavior following product launch.

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