ENSURING EFFECTIVENESS, SAFETY, AND ACCESS TO ORPHAN DRUG PRODUCTS

To encourage continued awareness of, research on, and development of orphan drug products; further,

To advocate for the use of innovative strategies and incentives to expand the breadth of rare diseases addressed by this program; further,

To encourage postmarketing research to support the safe and effective use of orphan drug products for approved and off-label indications; further,

To advocate that health policymakers, payers, and pharmaceutical manufacturers ensure continuity of care and patient access to orphan drug products; further,

To advocate federal review to evaluate whether orphan drug designation is being used inappropriately to receive FDA approval, extend patents, decrease competition, or limit discounts, thereby reducing patient access.

Rationale

The U.S. Orphan Drug Act of 1983 and similar programs in other countries have greatly expanded the number of therapies available to treat rare diseases through the use of financial and other incentives that encourage drug manufacturers to develop medications for limited patient populations. Despite the overall success of orphan drug programs, concerns have been raised about the breadth of drugs approved through these mechanisms. Although there are more than 7,000 designated orphan diseases in the United States, oncology drugs represent approximately 33 percent of all orphan drug approvals. ASHP believes that there is a significant need to develop a more comprehensive approach to orphan drug development in order to encourage drug manufacturers to expand the breadth of rare conditions treated by these therapies.

Once an orphan drug is approved, it may be used without restrictions, and these therapies are frequently used to treat patients and conditions that were not assessed during pre-approval clinical studies. While this use can spur innovation and lead to advances in the treatment of common diseases, ASHP believes that this use is also associated with the potential for increased patient harm, given the small patient populations and other characteristics common to studies used to support orphan drug approval. Research is necessary to evaluate the safety and effectiveness of these therapies under real-use conditions. In addition to manufacturer-conducted research, ASHP encourages private and public sector research in order to provide sufficient evidence to support off-label use.

ASHP is concerned about the high cost of these therapies, which contributes to increased healthcare costs and potentially decreases patient access, especially among those who are under- or uninsured. Further, some orphan drugs have later been discontinued by the drug manufacturer—an occurrence that often leaves patients with rare conditions without a treatment alternative. It is essential that stakeholders (e.g., health policymakers, payers, and pharmaceutical manufacturers) continue efforts to provide patient access to these therapies, including developing strategies to ensure that the cost of these therapies does not create an unreasonable barrier to patient access.

There are additional challenges regarding patient access to orphan drugs. There is a need for more emphasis on increasing patient access and addressing 340B issues, especially with critical access facilities. Orphan drug development and marketing in the U.S. is concentrated in a few therapeutic areas. Despite the increase in the number of orphan drugs approved by the Food and Drug Administration (FDA), the unmet needs of patients with rare diseases provide evidence that the current incentives are not efficiently stimulating orphan drug development. There is need to balance economic incentives to stimulate the development and marketing of orphan drugs without jeopardizing patients’ access to treatment.

Finally, one study (Sarpatwari A, Beall RF, Abdurrob A et al. Evaluating The Impact Of The Orphan Drug Act’s Seven-Year Market Exclusivity Period. Health Aff. 2018; 37:732–7. doi:10.1377/hlthaff.2017.1179) concluded that the orphan drug incentive of 7-year exclusivity only benefits about 33% of orphan drugs. The remainder have 20-year patent exclusivity that outlives the orphan drug incentive. Despite this discrepancy, the number of orphan drugs still grows every year, which may stem from manufacturers’ unfettered freedom to price these new drugs as they see fit. ASHP encourages federal review of current incentives to evaluate whether orphan drug designation is being used inappropriately to receive FDA approval, extend patents, decrease competition, or limit discounts, which can raise prices and reduce patient access.