Begin with the end in mind

Pharmaceutical leaders need to understand the factors holding assets back from reaching their full potential and keep a sense of perspective on drugs throughout their life cycle

April 2021

Dr. Paul Harney, President

Introduction

We should begin every journey knowing the destination. Roadworks, traffic jams and scenic detours may force a change of route, but the destination itself must not change.

Distractions from the intended end in mind may be a fact of life, but for any company to succeed in the long term, it is essential to avoid letting the road take you where it may. This is particularly true in pharma, a market in which assets take a long time to achieve viability and even longer to accrue value. The journey from concept to profitability will include several checkpoints, such as clinical trials, accessibility, prescriptions, fulfillment and persistence; meanwhile, there are several factors that will inhibit this journey.

As depicted in this graphic, there are factors at every stage of the journey that will prevent that blue line of profitability from achieving its full potential: Drugs that are unsafe for certain populations, fail to achieve efficacy expectations or end up with a limited label are only the first three. Within pharmaceutical companies, leadership must consider the whole life cycle and delivery of a product; however, that asset may change hands within the company at various stages as it moves from development to commercialization. Each team or individual may have a relatively narrow or limited-term perspective of the asset and its ultimate use; for example, the clinical team’s primary goals are typically the submission and approval of an asset, whereas the marketing team will be more focused on making the asset the first drug a prescribing physician considers.

Mere Approval

Merely reaching approval – although it may represent a great achievement – is not adequate. As , said during the Generics Pharma Manufacturing Annual Meeting in 2014, an event marking the 30th anniversary of the Hatach-Waxman Act of 1984, approval is the minimum standard for a drug and, although necessary, in itself does not meet the quality bars set by the ultimate customers: Patients and physicians.

In effect, Woodcock was saying that, to be a success, a product must surpass the assumptions that a customer will make of any pharmaceutical product – in other words, that it has crossed a minimum standard for safety and efficacy. Approved products still need to fulfill an unmet need or somehow be superior to, or otherwise more attractive than, competitor assets. It is not the role of the laboratory to establish market viability.

We see this borne out when clinical trial data are compared with real-world results; clinical trials are conducted under highly controlled conditions, and that control disappears in the marketplace. There is a myriad of reasons why drugs cannot achieve the results they did in trial conditions.

Janet Woodcock’s words are a warning to any company that takes a “build it and they will come” mentality toward a new product. There is an important gap between clearing the hurdles to market and achieving the full potential of a product in the market itself. Bridging this gap is key to achieving the goal of a product: Better patient outcomes.

Rather than pushing from the beginning of a drug’s development and working toward submission, the challenge for the savvy pharmaceutical company is to start with a focus on patients and work backward toward product design.

Fundamentally, it means acknowledging that drugs only reach their full efficacy in the real world when they are backed up by non-drug support mechanisms. If drug companies do not act to deliver on this care, especially in the case of generics, the assets will struggle to succeed. The ultimate goal is to shift the real-world evidence closer to the clinical trial results by adding value.

Efficacy in the Real World

There are several programs that have been developed to add exactly this kind of value beyond the efficacy of a drug.

A holistic patient assessment from the NHS West London Cancer Network maps out nine factors beyond the drug regime that influence patient outcomes. Despite the NHS’ “free at the point of use” model and its focus on cancer care, many of these factors hold true irrespective of the healthcare provider or disease under consideration.

Physical needs

Symptom assessment and use of validated tools, side effects, etc.

Emotional needs

Psychological assessment including an understanding of the patient’s expectations and the patient’s desire for a depth of information regarding low moods, fears and anxieties, strengths, coping mechanisms, etc.

Personal needs

Inclusion of allowances for cultural backgrounds, ethnic groups, sexuality, etc.

Social care needs

Social care assessment, welfare rights screening assessment, assessment for carers.

Information and communication needs

Identification of key workers, liaison with primary health or receiving health care workers, retrieval of patient-held records if appropriate, etc.

Control and autonomy

Assessment of one’s mental capacity to make decisions regarding patient choices, treatment options and plans, preferred places of care, etc.

Out of office

Identification of appropriate services according to treatment intentions, preferred priorities for care and transferring information to out-of-hours services.

Living with your disease

Ongoing care needs, such as rehabilitation support, referral to other agencies, end-of-life care planning if appropriate.

After-care

Bereavement needs, including funeral arrangements, bereavement risk assessment, future support and follow-up.

Looking over these factors, you can see that each has the potential to shift the needle on patient experience; taken together, they constitute a powerful, patient-centered approach to care. You will also see that, while these factors fall within the remit of the healthcare provider (in this case, the NHS), there is no reason to exclude other agencies from involvement. If you wish to make your drug more successful, this model is rich in ideas for added value that a drug company could offer to patients. Getting this delivery right will close the gap between clinical and real-world data, starting with ensuring that the drugs prescribed by physicians are taken with better compliance by the patient.

The noticeable absence from the NHS West London Cancer Network’s holistic patient assessment is “finance”. This is, in part, due to the NHS’ model of care, but it also speaks to how pharmaceutical companies overbalance to focus on financial access to a drug; from the patients’ perspectives, however, things look very different.

There is, therefore, a disconnect between the care a patient expects to receive and what companies can deliver. Placing the customer at the front of the model will help to achieve a better alignment between the two.

Rosetta Stone

We have dubbed this graphic “The Rosetta Stone”: It serves as a helpful reference point to remind us that various stakeholders approach the end in mind from completely different perspectives and often using very different language.

The problem is that there are two different ends in mind: approval and prescription. Goals, claims, messages and customers’ needs are different languages within biopharmaceutical companies, and real or perceived “firewalls” limit real communication. Companies must map out these different yet linked languages to understand the needs of various customers and stakeholders to enhance their target product profiles. Without understanding the motivation for each stakeholder, pharmaceutical companies will find they are disappointing at least one group of people, which will negatively impact uptake and profitability.

Conclusion

Starting with the end in mind is important, but wishing it so does not make it happen. To reach that destination, biopharmaceutical companies need to understand not only the optimum journey but also the nature of the obstacles in their way.

Goals are frustrated by different stakeholders with competing objectives and desires, who are often working at counter-purpose. The most successful drugs are those that fulfill these desires effectively across the board and reduce gaps and friction between internal functions and objectives. After all, we each got into the healthcare arena to help patients, and they have a higher bar than our regulatory partners; let’s keep them in mind at all points of development and commercialization.

Prescient has a long history of helping companies achieve this goal. Using planning and strategy, our approach combines real-world care delivery, clinical data, industry knowledge and competitive intelligence, so our clients are better able to navigate their way to a destination, even as circumstances force a change of plan.