The conflict of interest policy of the National Academies of Sciences, Engineering, and Medicine (http://www.nationalacademies.org/coi) prohibits the appointment of an individual to a committee authoring a Consensus Study Report if the individual has a conflict of interest that is relevant to the task to be performed. An exception to this prohibition is permitted if the National Academies determines that the conflict is unavoidable and the conflict is publicly disclosed. A determination of a conflict of interest for an individual is not an assessment of that individual’s actual behavior or character or ability to act objectively despite the conflicting interest.

Dr. Steven Galson has a conflict of interest in relation to his service on the Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union based on his membership on the board of directors of BioCryst Pharmaceuticals and his ownership of stocks of BioCryst Pharmaceuticals and Amgen, Inc., both of which develop products for rare diseases.

The National Academies has concluded that in order for the committee to accomplish the tasks for which it was established, its membership must include at least one person who has relevant broad expertise and experience in FDA drug regulatory policy, knowledge of what drives FDA decision making for approval of drug products, and an understanding of how FDA and the European Medicines Agency can collaborate.

As described in his biographical summary, due to his past leadership roles at FDA and at Amgen Inc., Dr. Galson has extensive expertise and experience in FDA drug regulatory policy from a variety of perspectives.

The National Academies has determined that the experience and expertise of Dr. Galson is needed for the committee to accomplish the task for which it has been established. The National Academies could not find another available individual with the equivalent experience and expertise who does not have a conflict of interest. Therefore, the National Academies has concluded that the conflict is unavoidable.

The National Academies believes that Dr. Galson can serve effectively as a member of the committee, and the committee can produce an objective report, taking into account the composition of the committee, the work to be performed, and the procedures to be followed in completing the study.

Dr. Anaeze Offodile II has a conflict of interest in relation to service on the Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union based on his role as chief strategy officer at Memorial Sloan Kettering (MSK) Cancer Center, a nonprofit oncology teaching hospital and research institute that derives a portion of its income from licensing of its pre-clinical or early-clinical stage oncology therapies, invented by its scientists to pharmaceutical or biotech companies. For example, over the past 5 years MSK has received revenues, related to rare diseases, under its licenses to Venthera Inc., Takeda Pharmaceuticals, YmAbs Therapeutics, Atara Biotherapeutics, and Theragnostics Ltd.

The National Academies has concluded that for the committee to accomplish the tasks for which it was established, its membership must include at least one person who has relevant expertise and experience in translational research with current understanding of technology transfer for drug development, particularly at the intersection of clinical care and drug development. As described in his biographical summary, due to his current role at MSK and prior role at MD Anderson Cancer Center, Dr. Offodile has extensive expertise and experience in translational research and technology transfer.

The National Academies has determined that the expertise and experience of Dr. Offodile is needed for the committee to accomplish the task for which it has been established. The National Academies could not find another available individual with the equivalent expertise and experience who does not have a conflict of interest. Therefore, the National Academies has concluded that the conflict is unavoidable.

The National Academies believes that Dr. Offodile can serve effectively as a member of the committee, and the committee can produce an objective report, taking into account the composition of the committee, the work to be performed, and the procedures to be followed in completing the study.

The National Academies believes that Dr. Offodile can serve effectively as a member of the committee, and the committee can produce an objective report, taking into account the composition of the committee, the work to be performed, and the procedures to be followed in completing the study.

Dr. Anne Pariser has a conflict of interest in relation to her service on the Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union because she is vice president of medical and regulatory affairs at Alltrna, which is developing a platform technology designed to utilize tRNA as a therapeutic for a variety of genetic diseases.

The National Academies has concluded that in order for the committee to accomplish the tasks for which it was established, its membership must include at least one person who has relevant current expertise and experience in private sector product development for rare diseases

As described in her biographical summary, as vice president of medical and regulatory affairs at Alltrna, as well as through her prior roles at NIH and FDA, Dr. Pariser has expertise and experience in product development, translational research, and drug regulatory policy for rare diseases.

The National Academies has determined that the experience and expertise of Dr. Pariser is needed for the committee to accomplish the task for which it has been established. The National Academies could not find another available individual with the equivalent experience and expertise who does not have a conflict of interest. Therefore, the National Academies has concluded that the conflict is unavoidable.

The National Academies believes that Dr. Pariser can serve effectively as a member of the committee, and the committee can produce an objective report, taking into account the composition of the committee, the work to be performed, and the procedures to be followed in completing the study. In each case, the National Academies determined that the experience and expertise of the individual was needed for the committee to accomplish the task for which it was established. The National Academies could not find other available individuals who had the equivalent experience and expertise and did not have a conflict of interest. Therefore, the National Academies concluded that the conflicts were unavoidable and publicly disclosed them on its website (www.nationalacademies.org).