How and Why to Involve Patients in Drug Development: Perspectives From the Pharmaceutical Industry, Regulatory Authorities, and Patient Organizations

2019 ◽  
pp. 216847901986429
Author(s):  
Marianne Botoft Hansen ◽  
Lotte Stig Nørgaard ◽  
Christine Erikstrup Hallgreen
Keyword(s):  
Pharmaceutical Industry ◽  
Drug Development ◽  
Patient Involvement ◽  
Drug Approval ◽  
Business Failure ◽  
Structured Interviews ◽  
Regulatory Authorities ◽  
Patient Organizations ◽  
Qualitative Interview Study

Background: Despite increasing interest and focus on patient-centric approaches to drug development, there might still be divergent views between key stakeholders in how to perceive patient involvement and how possibly divergent views influence the role of patients in the drug development process. The objective of this study is to explore how the perception of patient organizations, pharmaceutical companies, and regulatory agencies influence the role of patients in drug development. Method: A qualitative interview study based on 12 semi-structured interviews with representatives from the 3 stakeholders. Interviews were transcribed, and data were analyzed using a social constructivist approach in the form of systematic text condensation. Results: Three main perceptions of patient involvement were identified: “a way to improve quality of life,” “a way to avoid business failure,” and “a way to foster a faster drug approval process.” Transparency, trust, and clarification of expectations and roles were factors perceived as prerequisites for a valuable collaboration. Furthermore, a required cultural mindset change in the pharmaceutical industry, the lack of a common framework, patient organizations having limited resources available, and concerns about what to do with patient responsibility were perceived as the most important barriers for patient involvement. Conclusion: Based on the findings, the pharmaceutical industry, patient organizations, and regulatory authorities were labeled as “pioneer/dominant,” “unaware/quiet,” and “hesitant,” respectively. The 3 behavioural descriptors reflect a limited negotiation of the role patients have in drug development. Thus, the pharmaceutical industry appears to be the largest influencer with regard to patients’ role in drug development.

scholarly journals New Drug and Biologics Approvals in 2019: A Systematic Analysis of Patient Experience Data in FDA Drug Approval Packages and Product Labels

2020 ◽  
Author(s):  
Katrine Schultz-Knudsen ◽  
Ugne Sabaliauskaite ◽  
Johan Hellsten ◽  
Anders Blaedel Lassen ◽  
Anne Vinther Morant
Keyword(s):  
Drug Development ◽  
Patient Experience ◽  
Drug Evaluation ◽  
Patient Empowerment ◽  
Drug Approval ◽  
New Drugs ◽  
Systematic Analysis ◽  
Regulatory Decision ◽  
Patient Reported

Abstract Background The FDA Patient-Focused Drug Development Initiative was launched to ensure the incorporation of the patient voice into drug development and evaluation. Since 2017, the FDA must publish a statement outlining patient experience data (PED) considered in the approval of new drugs. This study investigated the presence and role of PED in drug approval and translation into product label claims. Methods PED reported in approval packages of the 48 drugs approved by FDA’s Center for Drug Evaluation and Research in 2019 was identified and categorized. PED in the form of clinical outcome assessments (COAs) was characterized by endpoint positioning and outcome. The product labels were analyzed for PED-related claims. Results PED was reported as relevant for 39 of 48 (81.3%) drugs approved in 2019. COAs were the predominant PED type; other PED was identified for only 9 (18.8%) drugs, and none included qualitative or patient preference studies. COAs were the only type of PED for which associated claims were identified in the product labels. 27 out of 48 (56.3%) labels contained one or more efficacy claims based on COAs; of these, patient-reported outcomes were the most prevalent with claims identified in 19 labels (39.6%). Conclusion There are ample opportunities for incorporating PED beyond COAs to inform drug development and facilitate availability of medicines tailored to patient needs. A higher level of transparency on the role of PED in regulatory decision-making and a clear path to PED-based label claims could incentivize sponsors and enable patient empowerment in treatment decisions.

scholarly journals What influences the outcome of active disinvestment processes in healthcare? A qualitative interview study on five recent cases of active disinvestment

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Adriënne H. Rotteveel ◽  
Mattijs S. Lambooij ◽  
Joline J. A. van de Rijt ◽  
Job van Exel ◽  
Karel G. M. Moons ◽  
...  
Keyword(s):  
The Netherlands ◽  
Healthcare Providers ◽  
Policy Decision ◽  
Structured Interviews ◽  
Qualitative Interview Study ◽  
Dutch Health ◽  
Financial Interests ◽  
Care Assistance ◽  
Insight Into

Abstract Background Recent attempts of active disinvestment (i.e. withdrawal of reimbursement by means of a policy decision) of reimbursed healthcare interventions in the Netherlands have differed in their outcome: some attempts were successful, with interventions actually being disinvested. Other attempts were terminated at some point, implying unsuccessful disinvestment. This study aimed to obtain insight into recent active disinvestment processes, and to explore what aspects affect their outcome. Methods Semi-structured interviews were conducted from January to December 2018 with stakeholders (e.g. patients, policymakers, physicians) who were involved in the policy process of five cases for which the full or partial withdrawal of reimbursement was considered in the Netherlands between 2007 and 2017: benzodiazepines, medication for Fabry disease, quit smoking programme, psychoanalytic therapy and maternity care assistance. These cases covered both interventions that were eventually disinvested and interventions for which reimbursement was maintained after consideration. Interviews were transcribed verbatim, double coded and analyzed using thematic analysis. Results The 37 interviews showed that support for disinvestment from stakeholders, especially from healthcare providers and policymakers, strongly affected the outcome of the disinvestment process. Furthermore, the institutional role of stakeholders as legitimized by the Dutch health insurance system, their financial interests in maintaining or discontinuing reimbursement, and the possibility to relieve the consequences of disinvestment for current patients affected the outcome of the disinvestment process as well. A poor organization of patient groups may make it difficult for patients to exert pressure, which may contribute to successful disinvestment. No evidence was found of a consistent role of the formal Dutch package criteria (i.e. effectiveness, cost-effectiveness, necessity and feasibility) in active disinvestment processes. Conclusions Contextual factors as well as the possibility to relieve the consequences of disinvestment for current patients are important determinants of the outcome of active disinvestment processes. These results provide insight into active disinvestment processes and their determinants, and provide guidance to policymakers for a potentially more successful approach for future active disinvestment processes.

scholarly journals Ethical considerations in prehospital ambulance based research: qualitative interview study of expert informants

2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Stephanie Armstrong ◽  
Adele Langlois ◽  
Niroshan Siriwardena ◽  
Tom Quinn
Keyword(s):  
Ethics Committees ◽  
Research Field ◽  
Structured Interviews ◽  
Ethical Considerations ◽  
Qualitative Interview Study ◽  
Regulatory Bodies ◽  
Fit For Purpose ◽  
The Uk ◽  
Time Limitations

Abstract Background Prehospital ambulance based research has unique ethical considerations due to urgency, time limitations and the locations involved. We sought to explore these issues through interviews with experts in this research field. Methods We undertook semi-structured interviews with expert informants, primarily based in the UK, seeking their views and experiences of ethics in ambulance based clinical research. Participants were questioned regarding their experiences of ambulance based research, their opinions on current regulations and guidelines, and views about their general ethical considerations. Participants were chosen because they were actively involved in, or in their expert capacity (e.g. law) expressed an interest in, ambulance based research. Results Fourteen participants were interviewed including principal investigators, researchers, ethicists and medical lawyers. Five major themes were identified: Capacity, Consent, Clinical Considerations, Consultation and Regulation. Questions regarding consent and capacity were foremost in the discussions as all participants highlighted these as areas for concern. The challenges and use of multiple consent models reflected the complexity of research in this environment. The clinical theme referred to the role of paramedics in research and how research involving ambulance services is increasingly informing improvements to patient care and outcomes and reducing the burden on hospital services. Most felt that, although current regulations were fit for purpose, more specific guidance on implementing these in the ambulance setting would be beneficial. This related closely to the theme of consultation, which examined the key role of ethics committees and other regulatory bodies, as well as public engagement. Conclusions By interviewing experts in research or ethics in this setting we were able to identify key concerns and highlight areas for future development such as improved guidance.

scholarly journals Pharmacogenetics in drug regulation: promise, potential and pitfalls

2005 ◽  
Vol 360 (1460) ◽  
pp. 1617-1638 ◽  
Author(s):  
Rashmi R Shah
Keyword(s):  
Drug Development ◽  
Dose Response ◽  
Genetic Factors ◽  
Response Relationship ◽  
Dose Response Relationship ◽  
Prospective Data ◽  
Regulatory Authorities ◽  
Regulatory Guidelines ◽  
Prescribing Information

Pharmacogenetic factors operate at pharmacokinetic as well as pharmacodynamic levels—the two components of the dose–response curve of a drug. Polymorphisms in drug metabolizing enzymes, transporters and/or pharmacological targets of drugs may profoundly influence the dose–response relationship between individuals. For some drugs, although retrospective data from case studies suggests that these polymorphisms are frequently associated with adverse drug reactions or failure of efficacy, the clinical utility of such data remains unproven. There is, therefore, an urgent need for prospective data to determine whether pre-treatment genotyping can improve therapy. Various regulatory guidelines already recommend exploration of the role of genetic factors when investigating a drug for its pharmacokinetics, pharmacodynamics, dose–response relationship and drug interaction potential. Arising from the global heterogeneity in the frequency of variant alleles, regulatory guidelines also require the sponsors to provide additional information, usually pharmacogenetic bridging data, to determine whether data from one ethnic population can be extrapolated to another. At present, sponsors explore pharmacogenetic influences in early clinical pharmacokinetic studies but rarely do they carry the findings forward when designing dose–response studies or pivotal studies. When appropriate, regulatory authorities include genotype-specific recommendations in the prescribing information. Sometimes, this may include the need to adjust a dose in some genotypes under specific circumstances. Detailed references to pharmacogenetics in prescribing information and pharmacogenetically based prescribing in routine therapeutics will require robust prospective data from well-designed studies. With greater integration of pharmacogenetics in drug development, regulatory authorities expect to receive more detailed genetic data. This is likely to complicate the drug evaluation process as well as result in complex prescribing information. Genotype-specific dosing regimens will have to be more precise and marketing strategies more prudent. However, not all variations in drug responses are related to pharmacogenetic polymorphisms. Drug response can be modulated by a number of non-genetic factors, especially co-medications and presence of concurrent diseases. Inappropriate prescribing frequently compounds the complexity introduced by these two important non-genetic factors. Unless prescribers adhere to the prescribing information, much of the benefits of pharmacogenetics will be squandered. Discovering highly predictive genotype–phenotype associations during drug development and demonstrating their clinical validity and utility in well-designed prospective clinical trials will no doubt better define the role of pharmacogenetics in future clinical practice. In the meantime, prescribing should comply with the information provided while pharmacogenetic research is deservedly supported by all concerned but without unrealistic expectations.

scholarly journals Downstream and into deep biology: Evolving business models in 'top tier' genomics companies

2006 ◽  
Vol 12 (2) ◽  
Author(s):  
Harry Rothman ◽  
Alison Kraft
Keyword(s):  
Drug Discovery ◽  
Pharmaceutical Industry ◽  
Drug Development ◽  
Business Models ◽  
Major Effect ◽  
Business Strategies ◽  
Fully Integrated ◽  
Biotechnology Firms ◽  
The Past

Genomics companies are changing their business models and some have moved beyond drug discovery into drug development. The authors' analysis of genomics companies' business models yields further insights into the widening role of genomics firms within drug innovation and on the evolving dynamics between the genomics sector and the wider pharmaceutical industry. Business models within the sector have included that of the FIPCO (Fully Integrated Pharmaceutical Company), technology and information platforms, and, more recently, a new 'dual' business model that combines established platform capabilities with drug development. The study identifies a cohort of 22 leading genomics companies and takes as its focus those companies following the dual and platform business strategies. The paper describes how, over the past five years, leading genomics companies have, typically, refocused their interests downstream within drug innovation, a move that brings new commercial opportunities but also threats. New and evolving business models are enabling these companies to leverage their commercial positions and capture value in the later stages of drug development. These shifts are characterised and the possibility that this 'downstream' trend could exert a major effect on the future relations between genomics companies and pharma/large biotechnology firms, and on drug innovation, is explored.

scholarly journals Innovators’ views on involving users and patients in surgical device development: a qualitative interview study

BMJ Open ◽  
2021 ◽  
Vol 11 (8) ◽  
pp. e050801
Author(s):  
Kas Woudstra ◽  
Marcia Tummers ◽  
Maroeska M Rovers ◽  
Rob Reuzel
Keyword(s):  
Patient Involvement ◽  
End Users ◽  
Strategic Choices ◽  
End User ◽  
Structured Interviews ◽  
Device Development ◽  
Surgical Devices ◽  
Qualitative Interview Study ◽  
Surgical Device ◽  
Innovation Practices

ObjectivesInvolving end-users and patients in the development of surgical devices, even when patients are not end-users, is deemed important in policy and in academia since it could improve strategic choices in research and development (R&D). Nonetheless, research into innovators’ views on end-user and patient involvement is rare. This study explores what end-users and patients are being involved by innovators during development, what methods for involvement are being used and what topics are being discussed with these end-users and patients.DesignA qualitative study featuring semi-structured interviews with innovators of surgical devices. Interviews were recorded and a thematic analysis was performed on verbatim transcripts.Participants15 interviews were conducted with 19 innovators of 14 surgical devices.SettingInnovation practices of surgical devices in the Netherlands and Belgium.ResultsEnd-users were engaged in R&D with formal methods and in unsystematic ways. These users all work in the clinical domain, for example, as surgeons or nurses. The innovators engaged users to analyse problems for which a device could be a solution, define functionalities, make design choices, analyse usability, ensure safety and improve aesthetics. Patients were rarely involved. Innovators stated that patients are not considered to be end-users, that physicians can represent patient interests and that involving patients is unethical as false expectations could be raised.ConclusionInnovators involve end-users with methods and unsystematic ways in the development of surgical devices. Despite governmental calls for patient involvement in the development of medical devices and surgical devices, innovators do not generally involve patients.

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