NICE INSIGHT OVERVIEW: Supply Chain

Patient centricity may initially have been a buzzword, but today pharmaceutical companies have begun active patient engagement programs that touch on all aspects of their discovery, development and manufacturing activities. That includes members of the supply chain, from CROs and CDMOs to raw material suppliers and distributors.

What is Patient Centricity Today?

Patient centricity has been defined in a number of ways. The first definition, developed through a collaborative effort among AstraZeneca, patients and caregivers, was proposed in 2017: “Putting the patient first in an open and sustained engagement of the patient to respectfully and compassionately achieve the best experience and outcome for that person and their family.”¹ The group identified five values important to patients seeking to take control of their own health: inclusiveness; sharing goals that are patient- and family-centered; empowering patients; showing respect, compassion and openness; and partnering. A set of 10 guiding principles were also proposed that outline steps to achieving meaningful engagement with patients.¹

Patients want pharma companies to focus on how they deliver their products in order to make what they deliver “meaningful and impactful,” according to Guy Yeoman, Vice President of Patient Centricity at AstraZeneca.¹ “We need to partner with patients using a common language and guided by agreed principles and that patients have to be part of the process from the outset. We need to listen to patients, work with them to co-create solutions and clearly communicate in a mutually acceptable way and time,” he noted.²

Unpacking the Drivers

Why has patient centricity become so important to the pharmaceutical industry today?
A confluence of factors is making the adoption of patient-focused drug development essential.

Rapidly advancing technologies are a leading driver. Patients have access to information about their diseases and are becoming not only more knowledgeable but more active in the management of those diseases. Social media facilitates communication among patient populations, advocacy groups and pharma companies developing new medicines. They not only expect but demand that their voices are heard. At the same time, big data, advanced analytics, artificial intelligence and other technologies are enabling the evaluation of data sets comprising results generated both in clinical trials and in post-marketing studies that can be used to improve drug design.

Regulatory agencies have taken steps to incorporate the voice of the patient into the development and approval processes. Both the U.S. Food and Drug Administration (FDA)3 and the European Medicines Agency (EMA)⁴ are working to incorporate patient opinions into development. The FDA, for example, holds disease area–specific meeting with individual patients and patient groups. The FDA and the EMA also formed the FDA/EMA Patient Engagement Cluster in order to share best practices for patient selection and training for drugs being developed to treat cancer and orphan diseases.

Patient input has already influenced the FDA’s decisions on several drugs, including a new melanoma therapy approved in 2015.⁵ In May 2018, the agency published a new policy on what constitutes an outsourcing facility for drug compounding versus facilities used for the compounding of patient-specific prescriptions. Part of the reasoning given by the agency was the need for healthcare providers and patients to know the standards under which the compounded drugs they purchase are made.⁶

There is also recognition that, if the pharmaceutical industry truly seeks to develop medications to improve and extend patient lives, then patients should dictate what and how those drugs should be delivered, rather than pharma companies assuming what is best for them. There is a paradigm shift occurring, from a focus on profit itself to one that considers how that profit is made and used.⁷ There is also a recognition that the industry needs to build trust with providers and patients before it can become truly patient-centric and create additional societal value.⁸

Clear Benefits

Indeed, the general consensus is that patient-centric drug development is valuable — a key finding of the Drug Information Association’s (DIA) 2017 Study of Patient-Centric Initiatives (PCIs) in Drug Development, which was conducted in collaboration with researchers from the Tufts Center for the Study of Drug Development (Tufts CSDD).⁸ For clinical trials in particular, expected benefits identified by the companies that participated in the study include faster trial subject recruitment, improved subject retention rates, reduction in the number of protocol amendments, lower cost and increased ease of study conduct and a greater number of patient-relevant endpoints. Overall, patient engagement leads to better outcomes and ensures that the right problems are being addressed.

Another important finding of the study was that many different approaches to patient-centric drug development are currently being implemented, with little consensus on which methods are most effective. Newly implemented programs generally evolve over time to develop the resources and understanding needed to be effective. Specific PCIs described in the study as low cost but high benefit include advocacy group support and involvement, patient advisory panels and social media and online engagement.⁹

It is also important to remember, according to the Pharmaceutical Research and Manufacturer’s Association (PhRMA), that developing “a science-based and systematic approach to gathering patient input robust enough to support FDA’s regulatory decision making is necessary to realize the goal of a patient-centered approach to drug development and regulatory review.”⁵

If done right, patient-centric drug development will not only improve clinical trial design but also promote the best choices when it comes to formulation and delivery. The latter can lead to improved outcomes through better medication adherence — a key problem in the industry today.¹⁰ The design of drugs for specific patient populations, such as children and the elderly, takes into consideration ease of swallowing and dosage frequency through extended-release forms. Combination products can help reduce the number of medications that patients need to manage. Single-dose packaging eliminates the need to measure or count and is more convenient for people who need to take medications on the go.¹¹ The list of options is long indeed, and growing.

Industry Initiatives

For pharmaceutical companies to become truly patient-centric, transformation is needed from the top down. All aspects of the business — strategy, structure and processes — must be revised to enable transparency and a patient-driven development process.⁷ Most pharmaceutical companies have at least taken initial steps toward adopting a patient-centric approach.

A few examples include:

• AbbVie developed a YouTube channel — Doctor’s Channel — for Israeli patients to access simplified, medically approved information for over 114 diseases.⁸
• AstraZeneca implemented a multi-year PaCe (patient centricity) pilot program to embed patient-centric principles into its operations, which led to the routine
  incorporation of patient insight into protocol development, among other changes. It has also developed a “complete patient value model” with Medical Affairs teams as the “gatekeeper” of patient centricity.⁸
• GSK has committed to promoting “transparency of clinical research” and “was the first company to grant access to anonymized patient data[.]” The company’s “patient-level data access site has become a multisponsored portal.”⁷
• Sanofi, through its partnership with patient-centric technology firm Science 37, is achieving higher enrollment in clinical trials more quickly while using fewer resources.⁸
• Shire increased the rate at which patients who fill out start forms actually receive treatment by placing “a nurse at the center of its patient-support efforts to coordinate the process.”⁸
• Takeda has focused on increasing the empathy of its employees for patients with IBD to introduce patient centricity throughout the organization.⁸

Many companies are also learning from organizations that have experience collaborating with patients, including the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the Medical Device Innovation Consortium (MDIC), the European Patients’ Academy on Therapeutic Innovation (EUPATI) and the International Federation of Pharmaceutical Manufacturers & Associations (IFPMA).⁷

The Patient-Centric Supply Chain

In the traditional view of the pharmaceutical industry supply chain, the patient is at the end of the chain, after discovery, development, manufacturing, distribution and sales. With the new focus on patient centricity — combined with the advent of personalized medicine, next-generation therapies that require manipulation of individual patient samples in the drug manufacturing process and outcome-based reimbursement — this view of the supply chain must be redefined.

For one thing, the aging of the global population is increasing the need for delivery of medicines to patients where they are located at the time they need them and in forms that are easy and convenient for them to use. Visibility and transparency across the supply chain will be essential if the pharmaceutical industry is to deliver patient-centric medications in a cost-effective manner that meets the real needs of patients.¹²

Globalization of the supply chain has created numerous challenges for the industry, but it also presents opportunities to achieve that end-to-end visibility. Merck, for instance, is moving from the traditional step-by-step approach to development, manufacturing, commercialization and distribution to a concurrent planning model that allows for greater responsiveness.¹² Advances in cloud-based management solutions are also facilitating the move from linear management of larger projects to the adaptive management of a series of smaller projects. Digital technologies are also enabling the monitoring of cold-chain shipments, direct-to-patient delivery of medicines and in-home collection of biologic samples.¹³

More Work to Do

Becoming a truly patient-centric industry requires significant effort and a complete change in the way pharmaceutical companies operate. Patients are all different; they have different needs that depend not only on their specific diseases but also on their individual backgrounds, personalities, coping mechanisms and many other characteristics. In addition, patients may have expectations that differ significantly from those of their healthcare providers.⁷ Navigating these complex waters is not an easy task.

Pharma companies and their contract services providers (research organizations, manufacturers and logistics firms) are taking the first steps — leveraging patient advisory groups, working with collaborative organizations and developing internal programs designed to incorporate patient insights into their operations.

As these initiatives mature, collaboration expands, insights are shared and standards are developed, the benefits will be realized for all members of the supply chain. Unmet patient needs will be better defined, leading to new drugs that better address them. Clinical trials will be shorter and provide better data. The result: more drugs that provide more value and enable enhanced medication adherence for achievement of the ultimate goal: better patient outcomes in terms of improved health and extended lifetimes.

References

1. First collaborative definition of patient centricity, co-created by AstraZeneca with patients and carers, is published in BMJ Innovations. AstraZeneca, 31 Mar. 2017. Web.
2. Yeoman, Guy. “Patient centricity – a collaborative definition.” Patient Focused Medicines Development. 20 Apr. 2017. Web.
3. Patient Engagement Advisory Committee. U.S. Food and Drug Administration. 25 Jul. 2017. Web.
4. Incorporating patients‘ views during evaluation of benefit-risk by the EMA Scientific Committees. European Medicines Agency (EMA). 23 Oct. 2014. Web.
5. Patient-Focused Drug Development. PhRMA. n.d. Web.
6. FDA in Brief: FDA issues new policy on what constitutes an outsourcing facility, a key step in implementing the agency’s comprehensive framework for compounding. U.S. Food and Drug Administration. 10 May 2018. Web.
7. du Plessis, Danie, John-Kenneth Sake, Katarina Halling, Jackie Morgan, Anna Georgieva and Neil Bertelsen. “Patient Centricity and Pharmaceutical Companies: Is It Feasible?” Therapeutic Innovation & Regulatory Science (2017). 51: 460–467.
8. Ricci, Marco. “What’s the value of patient centricity?” pharmaphorum. 14 Nov. 2017. Web.
9. Parikh, Sudip S. “Study Insights: Patient Engagement in Drug Development,” Applied Clinical Trials. 19 Apr. 2017. Web.
10. Siew, Adeline. “Patient Centricity Takes Center Stage.” Pharmaceutical Technology (2017). 41:16–23.
11. Kudla, David, Rao Tatpudy. “The People’s Choice: Premeasured Single Dosage Forms.” Pharma’s Almanac. 5 Jun. 2017. Web.
12. Durbha, Madhav. “Patient-centric networks at the heart of pharmaceutical supply chain management: Lessons from LogiPharma Europe 2018.” Kinaxis 21st Century Supply Chain blog. 18 Apr. 2018. Web.
13. Wheeler, Wes. “Supporting Patient-Centric Clinical Trials from Point-of-Origin through the Last Mile.” Pharma’s Almanac. 29 May 2018. Web.