Sudip S Parikh PhD

	The Drug Information Association (DIA) has released initial results of its 

Study of Patient-Centric Initiatives (PCI) in Drug Development

, conducted in collaboration with researchers from the Tufts Center for the Study of Drug Development (Tufts CSDD). Initial insight from this study points to a groundswell in the desire to move toward true patient-centricity, but large variation in the approaches to implementation. These divergent approaches to implementation will require metrics and validation to identify best practices. Here, we highlight some key insights from the study.
	
	

Drug development and clinical trials have historically focused on the tested molecules and researchers doing the testing


	
	Traditional approaches to clinical trials by researchers have typically been focused on the collection and assembly of data necessary to gain approval of the study drug/molecule, rather than being patient-centric.
	
	In the traditional trial model, the “voice of the patient” has been secondary to approval-focused elements, or was included where it was easiest to do, i.e., where it had the lowest activation energy to include patient input.
	
	In the past, clinical trial focus on the product rather than the patient happened because researchers executed the trial with the end goal of simply getting the product approved. There is considerable inertia in this model; metrics and incentives in the mix have historically been product-approval focused. The clinical trial team knows from experience what works, and they logically build a trial structure and data assembly approach similar to studies that have successfully led to past approvals.  
	
	

Patient-centricity has become a mantra for industry, regulators and patients


	
	Desire for patient-centricity was a clear imperative from results of the DIA-Tufts study. According to the survey, “Almost all stakeholders agree that engaging patients in the drug development process is valuable, but they do not necessarily agree on why or how.”
	
	While stakeholders seem to agree that drug development should be patient-centric, the current reality is that companies have strict budgets, timelines and hard metrics (e.g., study uptake, progress and cost) that drive collection and assembly of clinical data to get a product approved. Costs without validated impact on outcomes are not readily supported.
	
	


	
	The study results point to a groundswell of support for patient-centered drug development in industry and academia. Also, it was found that half the pharmaceutical and biotech companies surveyed have already launched, and a similar percentage are piloting or planning to launch PCIs. Though the results are preliminary, it is clear these companies expect significant benefits from patient-centricity, including:

		Reduction in the number of protocol amendments

		Lower cost and increased ease of study conduct

		A greater number of patient-relevant endpoints

	The DIA-Tufts study found other important expected benefits of patient-centricity, including improvement in patient activation measures (PAM scores) and better internal and external trial reach. Preliminary insight from the study shows patient-centricity and business ROI need not be mutually exclusive. Indeed, as payers and providers increase their demand for products that address key aspects of a patient’s disease state, true patient centricity and effective PCIs will be essential to business ROI.
	
	

But, importantly, the study found that there is not yet clear consensus on how to implement PCIs


	
	While the DIA-Tufts study found broad agreement on the value of PCIs, the question of “How” is still being grappled with by industry. Still elusive is understanding what models for patient engagement-or what points of involvement in the process-deliver the best results for the patient and for the company.
	
	The study found that barriers to PCI implementation include difficulty in getting internal buy-in and the lack of authority to implement initiatives. But once the value proposition of PCIs are clearer, these barriers are likely to lessen as companies see the real business advantages of patient-centricity mentioned above. To shift toward patient-centricity, clinical trial teams will need to have the authority to focus trial design on the patient experience, with the full support of their organization. At present, disconnects remain between the goals of executive leadership and the incentives driving drug development projects.
	
	This disconnect has been overcome in other industries. For example, improvements to “safety culture” at chemical companies-where in the leading safety-oriented companies, the entire accountability chain is constantly thinking about safety metrics. When this primacy of safety was woven into the accountability of every managerial level, it became a foundational driving principle and deeply ingrained in the culture of the entire organization.
	
	


	
	The study identified, and goes into deep detail, on three low cost and high benefit PCIs that are relatively easy to implement: Advocacy group support and involvement (e.g., HIV/AIDS, Duchenne muscular dystrophy and others), patient advisory panels, and social media and online engagement PCIs.
	
	


	
	Companies that are planning on implementing PCIs will have many emerging technology tools to help them shift toward patient-centricity. Large data analysis, precision medicine, combination products, and the development of tandem diagnostics that prove a product’s value to a patient are all signs of a massive new wave of convergence with the patient squarely in center of focus. This convergence is occurring because at the highest level, companies need actionable data about each patient and the relative value of the study drug/molecule in order to positively influence the aspects of the disease state the patient experiences.  
	
	An example of how a PCI might work is where a clinical team may wish to incorporate the voice of the patient (VOP) during the design of the protocol, and add budget for VOP capture with the goal of better adherence and speed-of-trial execution. This approach starts with the patient and structures the trial around elements that actually matter to the patient and clearly quantify improvement of his or her disease state.  
	
	The business reality is that it is unlikely that a company will develop a drug that effectively and completely treats the disease state for 300 million people. Convergence-and the use of PCIs using next-generation technology-will allow study drugs/molecules to be designed with a sharp focus on the full continuum of disease state elements for smaller patient groups. To succeed in trials designed for smaller patient groups, the need to focus protocol design on data elements that measurably improve the patient’s experience is essential. This convergence is changing the way drugs are developed in the industry and in basic research in academia.
	
	


	
	The Study of Patient-Centric Initiatives in Drug Development clearly found a pent-up demand for rapid movement toward patient-centricity. Later this year, DIA and collaborators will be presenting a deeper look at these results in a full peer-reviewed article that examines the issues and benefits related to patient-centric clinical trials, with the focus on answering the “how to implement the right PCIs to make the most impact” question.
	
	

, is Senior Vice President & Managing Director, DIA Americas

 at the 2017 DIA Annual Meeting, June 18-22, in Chicago.

Articles

Initial findings from a DIA study of patient-centric initiatives in drug development reveal pharma’s desire to move toward true patient-centricity, but approaches to implementation are varied.

Sudip S Parikh PhD

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