Where Hope Looms, So Do Hurdles

The extreme cost of product development coupled with stringent regulations, and the need to maximize ROI, often discourages healthcare companies from developing products for rare diseases. Nonetheless, the FDA designated nearly 300 drugs and approved 48 novel and repurposed rare disease therapies last year. The FDA’s breakthrough designation helped fuel certain areas, especially where biomarkers have allowed targeting of specific subpopulations of patients within larger populations. So while there is hope, and progress is being made, hurdles remain.

Mapping out the clinical development program as early as possible is one such challenge—requiring early and frequent communication with the FDA. Large random controlled trials (RCTs) and full clinical pharmacology packages are not feasible in developing orphan drugs. Identifying important safety signals with small populations remains an issue. One approach monitors safety from off-target pharmacological MOA as opposed to whole-organ observations. Clinical trial protocols that allow for smaller patient populations are important too, as are genetic biomarkers, which can accelerate and improve trials. But aligning with regulators on the appropriate biomarkers and study endpoints is critical.

Lack of knowledge and training represents another important challenge to commercialization. For many rare diseases, available information is inadequate. Health professionals often lack skills needed to diagnose and treat these diseases. As such, intense educational support is needed for prescribers, patients and payers.

Payers Forced to Reassess

Despite the fact that orphan drugs can cost hundreds of thousands of dollars per year for a single individual, patients have faced little reimbursement pressure. However, in recent years, rapid growth in the number of premium-priced orphan drug launches, price increases, and new indications for existing products, along with intense pressure to control healthcare costs have “forced” payers to reassess their reimbursement strategies. The affordability of these treatments has become a major issue for public and private payers, and is a strong driver of tension between all the various stakeholders. The importance of communicating the value to all stakeholders is more critical than ever. The earlier you bring payers into the discussion, the better.

Finally, manufacturers have responded by developing effective programs to facilitate access to the drugs. Even when these important drugs are available, patients may be unable to access treatment because of high out-of-pocket costs. Patient hubs are important in this space because they provide essential information services for patients (clinical information, side effects, adherence, financial support resources) and providers (supply/purchasing information, benefits verifications, tips for administration, billing, patient counseling).

Enhanced communication and collaboration among researchers, patients, providers and manufacturers will allow us to overcome many of these hurdles—starting with a blueprint of the clinical development plan, a cohesive value story, and a 360-degree patient access program.

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