To answer the primary and secondary research questions, qualitative data were collected from key informants using a semi-structured questionnaire and interview format (Appendix 5). The choice of key informants and interview questions were intended to encompass the full range of information relevant to pharma R&D including patient privacy, intellectual property and patent law, regulatory oversight, science, healthcare financing, information technology, policy, politics and marketing.
The key informant interview methodology is well suited for exploratory research such as a new and hypothetical OSRD paradigm. Moreover, the primary endpoints, pharma R&D quality and efficiency, at this time are ill-defined under the current R&D model, therefore providing no credible baseline on which to build a more quantitative research methodology.
Finally, it would be impractical to rapidly pilot-test OSRD by applying it to some pharmaceuticals in development, and then comparing the efficiency and quality of OSRD versus pharmaceuticals developed under the traditional paradigm.
In other words, there are currently no quantitative means by which to
determine, for example, ‘an OSRD process would be XX% more or less expensive per approved drug,’ or ‘require YY more or less years to move from discovery to
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approval.’ The qualitative key informant approach is therefore ideally suited to investigate a hypothetical pharma R&D process such as OSRD to assess its potential feasibility and acceptability.
Identifying Key Informants
Key informants were contacted representing three major stakeholder groups: academia, industry, and regulatory authorities, as shown in Figure 4. Academics were sought in order to gather feedback from
researchers that were more likely to be open to innovations that challenge the
current drug R&D process. Industry representatives were interviewed as it was initially assumed that they would be the most invested in the status quo, the current drug development process, and therefore the most resistant to change. Moreover, pragmatically, it is unlikely that the pharma R&D process can be modified without buy-in from industry, and therefore it was important to gauge pharma’s appetite for change. Finally, regulators were interviewed because changes to the R&D process could have an impact on the future approvability of drug candidates, and therefore regulatory support for any major innovation is critical.
Senior scientists, executives, and regulators were chosen from organizations such as the following:
Figure 4: Stakeholder map for pharma R&D
41 Academia
Cleveland Clinic Coordinating Center for Clinical Research, Duke Clinical Research Institute, Harvard Clinical Research Institute, etc.
Various healthcare economics, financing, patient advocacy groups, and/or policy organizations (e.g. Open-Science In Drug Discovery, Clinical Trials Transformation Initiative)
Industry
Pharmaceutical, device and/or diagnostic development companies, both “Large Pharma” and smaller “Biotechs”
Contract Research Organizations, to which pharmaceutical companies typically outsource R&D (e.g. Quintiles, PPD, PRA)
Regulatory
US FDA, EMA
Note that the above list of organizations is illustrative in order to protect the confidentiality of the key informants who actually participated in the interviews. The initial target number was 3-4 senior representatives from each stakeholder group or until saturation was reached. Saturation in this context refers to the situation where little or no new insights or information is gained from each additional key informant.53
42 Recruiting Key Informants
The process for recruiting potential key informants included the following steps:
1. Potential participants were contacted via e-mail or letter (Appendix 1) to ascertain interest in participating in research regarding general innovations to the current paradigm for pharma R&D.
2. Potential participants were provided with a brief description of the proposed hypothetical OSRD model (Appendix 2).
3. Potential participants were given an Informed Consent Form (Appendix 3) prior to seeking verbal consent and encouraged to contact the researcher with any questions or concerns regarding participation. Verbal consent was obtained immediately prior to conducting an interview.
4. Thirty-to-60 minute interviews were scheduled and conducted in-person or over the telephone.
IRB Review
As the key informants were not a vulnerable population, the information
sought in the interviews was not particularly sensitive, and the likelihood of breach of confidentiality was low, the UNC Biomedical Institutional Review Board (IRB)
granted an exemption on 4 June 2012 for the key informant interviews and approved a request for a verbal consent process (Appendix 6).
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Confidentiality Issues
In order to protect the confidentiality of the key informants, information about their roles and experience was reported in the aggregate. Many participants had multiple degrees, but only one degree was reported. Masculine pronouns (he, his) were used regardless of the sex of the participant. Finally, when referring to
someone’s role such as CEO, Senior Regulator, or Academician, the active tense is used, suggesting that they were in the stated role during the time of the interview even though they may not have been (e.g. retired). The title or role reported represents the highest or the longest role in duration in their careers at the time of the interview.
Also, throughout the paper attributed quotes are used, for example in the Review of the Literature and also the Discussion chapters. Note that none of the attributed quotes came from people who were also key informants for the research.
Data Collection Procedures
Interviews were recorded when permitted by the respondents and notes taken by the interviewer in all cases. In some cases, the key informants referenced or provided additional materials to support their comments. Immediately after the interview, the interviewer clarified and/or amended the interview notes (Appendix 4).
All recorded interviews were transcribed. To protect key informant
confidentiality, all transcripts (Microsoft Word documents) were password protected, the key informant’s identifying information (title, employer, etc.) were deleted, and
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their names were replaced with a letter/number code. The key for identifying which code corresponded to which interview was maintained in a password-protected Microsoft Excel file.
Data Management and Analysis Plan
The transcribed interviews were coded and analyzed manually, using the Coding Manual developed for this research and presented in Appendix 7. The
quotations reported here are predominately verbatim, with minimal editing in order to remove information that might compromise the confidentiality of the participant. When such edits occur to directly quoted responses, the edits are identified by
brackets such as [edited text inserted by researcher] and/or ellipses […] to represent deleted material. Furthermore, the quotations imbedded in the text of the dissertation at times were edited to improve flow and clarity while seeking to avoid any corruption of the key informant’s intent. However, the transcribed quotations in Appendices 8 - 10 are excerpted from the interviews but generally not edited for flow or clarity.