Orphan drugs clinical research grants

The Orphan Drug Act authorized grants for clinical research on potential orphan products, and one of its more recent amendments (Public Law 100-290) extended this authority to preclini-cal studies. These grants represent a direct subsidy for orphan drug R D.31 The Office of Orphan Products Development administers the program in a manner parallel to other Public Health Service grants. 

The clinical research tax credit (discussed in chapter 8), protocol assistance, and clinical research grants theoretically lower the cost of orphan drug R D the market exclusivity provision increases the expected revenues to such R D. In practice, the protocol assistance has had little effect, especially in recent years, and the tax credit and grants program represent, overall, a relatively small commitment of Federal funds to orphan products. This commitment may be critical for certain drugs, however, so it should not be discounted. 

The code will be broken and data will be analyzed when the 12-mo follow-up has been completed for all patients. At that time, DHA therapy will be offered to all patients who desire to receive it. Clinical and laboratory studies will be monitored by an independent data and safety monitoring committee. This committee will also perform an interim analysis when 30 patients have completed assessment and has permission to terminate the study if there are significant favorable findings or unanticipated adverse outcomes. A determination of statistical power indicates that this sample size is sufficient to detect a major effect (0.862 standard deviations). With a more modest effect of 25%, however, the statistical power diminishes to 60%. The study has been approved by the Institutional Review Board at the Johns Hopkins Medical Institutions and is supported by a research grant from the Office of Orphan Products Development at the Food and Drug Administration. Fifty-two patients are enrolled in the study at this time. The code has not yet been broken. No adverse effects attributable to the medication have been observed. 

The Orphan Drug Act of 1983 (Public Law 97-414) provided a 50-percent tax credit for qualifying clinical R D on investigational drugs that have been granted orphan status by the FDA. The credit is available only for qualifying clinical research, not for animal or laboratory research and not for supervisory or other kinds of R D expenditures typically disallowed by the Internal Revenue Service. Also, when the credit is applied, the expenses cannot be deducted, so the net cost of a dollar of qualifying research under this credit is effectively 0.50. Companies without current taxable income cannot save the credit for use in future years, however, so startup research-based firms may not have access to this credit. 

This work has been supported by grants FD-R-000179 and FD-U-000505 from the US Food and Drug Administration s Orphan Products Office, and by the General Clinical Research Center of the University of Michigan Hospitals, supported by the National Institutes of Health (grant number M01-RR00042). 

---