Are We on the Right Track?

Choices.

Have you ever found yourself in an unfamiliar city trying to get from point A to point B as quickly as possible?  Taxi? Bus? Subway? Traffic scares you, so the subway it is.

You stare at the nicely designed kaleidoscope of a map and try to make sense of which trains are going where, when, and how quickly. Do I need a local? Can I take the express?

It’s a big decision.

You find the nearest station and head down the stairs to wait for the train. If you pick the wrong one, you may be late. Or if the express doesn’t get you close enough to your destination, you may need to walk farther than you want.

But what if you picked the wrong station. You may not reach your destination at all. Once you are on those tracks, the train can only go one route. Options are suddenly very limited.

Maybe you should ask someone who lives here first.

Understanding the Problem

The purpose of this series is to identify and characterize some of the barriers to effective collaboration between patients and the innovative pharmaceutical industry.

There are several factors that make it difficult to achieve the type of deep patient involvement in the drug development process and the shaping of the future of medicine. In this series, we will explore five of these barriers and look for common elements that may provide a course of action.

• Patients Don’t Speak the Language of Drug Development
• Professional Experts Can Be Intimidating
• Patients and Researchers See the Journey Differently
• Many Stakeholders, Many Stakes
• Biopharma May Be Missing What is Most Important to Patients

In this installment, we will compare the relative benefits of using patient input for tactical and strategic decisions. We will explore why biopharma companies may not gain the full advantage that the patients’ voices may offer.

Faster, Better, Cheaper – Pick Two

The literature about patient-centric drug development points to two potential benefits or objectives that drive the discussion; operational efficiency and improved outcome.

• Operational efficiency refers to the cost, time, and effort required to complete a study.
• Improved outcome refers to the ultimate output of the efforts; the results, the delivery of better products.

Getting there quickly

Operational efficiency is critically important to the biopharmaceutical companies from a strictly business standpoint. They strive to accelerate patient recruitment into clinical studies and to ensure that those patients fully complete the protocols. This saves time and money.  Sponsors of clinical studies also want study subjects to have a positive experience as a participant. A lot of work has gone into reducing the burden of participating in a clinical trial and there certainly is much to be learned from patients’ experiences in clinical studies.[1] Companies are seeking the luxury express train option.

Operational efficiency is important to patients as well. Clearly, if the clinical studies can be completed sooner, then one would expect the treatment to be available sooner. If the costs of studies can be reduced, there is a case for lowering the overall cost of medications. On a more personal level, a well-run study should be more convenient and less of a hassle for patients.  However, operational efficiency probably is not the primary concern for patients if the train doesn’t stop at their station.Patients really want better treatments.

Arriving at the right station

Improving the overall outcome of drug development is everybody’s goal. Right?

• For the scientist (and regulators), an improved outcome means the answers are clear and decisions can be made about the safety and efficacy of a given investigational product.
• For the marketer, an improved outcome means that the results support all the claims they want to be able to make about the product.
• For the government payer or private insurer, better outcomes mean that the treatment is cost-effective – that the difference is worth the price. Here the term “outcomes” takes on a pharmacoeconomic meaning.
• For the patient, an improved outcome means that the medicine actually addresses how they function and feel.

Yes, everyone generally agrees improving the outcome of drug development is important, even if the specifics differ. This is where bringing the voice of the patient can radically change the successful development of truly innovative medicines. This is the big win for patient involvement and biopharma may be missing it. They may be taking the fastest train to the wrong destination.

Biopharma may be missing the big win because it is so much harder to manage and they have little experience letting patients drive the discussion. Maybe, it is not up to the companies, but rather patients to take the initiative. The key will be to equip patients so that they are able to step up to this challenge.

Laying the tracks for a future paradigm

The general consensus is that to really accomplish patient-involvement, -centricity, or -leadership it is important to get patients involved at the very beginning of a drug development program. It is believed that the sooner the patient is involved, the more they will be able to shape the research, and the better the outcomes will reflect patient needs.[2] This is much more difficult to achieve and measure than tinkering with recruitment and retention rates. Practically speaking, how will patients actually be involved? How will we monitor whether this involvement has a positive impact?

There are emerging regulatory efforts to further this agenda and answer these questions.

For example, Title III Section 3002 of the 21st Century Cures Act requires FDA to issue guidance regarding the methods and approaches used in capturing and measuring patients’ experiences and perspectives. The US FDA is working on developing guidance for Patient-Focused Drug Development and published a framework for this work in May 2017[3]. The framework consists of eight (8) topic areas summarized here:

1. Methodological approaches to collect patient experience data, including methods to collect meaningful patient input throughout the drug development process and considerations for collecting, reporting, managing, and analyzing the data.
2. Methodological approaches to develop and identify what is most important to patients
3. Methodological approaches to identify and develop methods to measure impacts on those variables.
4. Methods, standards, and technologies to collect and analyze clinical outcome assessments for purposes of regulatory decision making
5. Guidance on submitting draft guidance related to patient experience data
6. Format and content for submission of patient experience data
7. How the Secretary (of HHS) intends to respond to submissions of patient experience data
8. How the Secretary (of HHS) anticipates using patient experience data and input to inform regulatory decisions.

The Plan also outlines a timeline for the issuance of each part of the framework over the next 5 years. The first four points are particularly relevant to patients and must be shaped by patients or Patient Advocate Groups. While the remaining four are important, they are focused on mechanisms to implement and administer the guideline.

There is a huge opportunity for patients to get involved in the shaping of this guidance. All US Patient Advocate Groups should be building this work into their strategic plans for the next 5 years. Part of this strategy should be to provide drug development education to their constituents so that those patients are better prepared to effectively contribute.

In addition, Patient Advocacy Groups, working with industry partners, are working to define opportunities for potential patient involvement throughout the drug development cycle.  For example, the Parkinson’s Disease Foundation has developed a simple framework for patient involvement at 10 different steps in drug development.[4]

Just like railroads had to do in the 19th century, standards need to be developed across different therapeutic areas, companies, PAGs, and even countries. By agreeing on a common framework for involving patients, these groups can work to prepare patients to take part in the areas that make the most sense to them. An educational system that can be leveraged across these boundaries will reduce the time and cost it takes to get patients equipped, and improve the chances that they will get the researchers on the right track.

What do you think?

What does this framework need to include?

Who needs to be involved?

How might you help?