Identifying Unmet Medical Need

An unmet medical need refers to a condition for which no satisfactory treatment exists, or where the available therapies are inadequate. The FDA defines it as a condition whose diagnosis or treatment is not sufficiently addressed by existing therapies. For researchers, this is not just a theoretical concept; it directly impacts how we approach drug development.

To understand unmet medical need, we must start with the end in mind, working backward from the problem the patient faces to develop a solution.

The most obvious unmet need occurs in serious diseases for which there is no effective treatment, such as metastatic pancreatic adenocarcinoma. For these diseases developing a therapy that is curative or alters disease progression could meet an urgent unmet medical need.

Sometimes existing therapies only partially address the problem. For example, in Parkinson's disease, current therapies can improve symptoms and quality of life but do not halt the progression of the disease. This represents a clear unmet need: the demand for a therapy that can slow or prevent disease progression.

Even when treatments exist, unwanted side effects can create their own unmet medical need. For example, chemotherapy regimens for Hodgkin’s lymphoma are quite effective in achieving a cure, but can cause delayed, potentially life-threatening cardiac toxicity. There is a clear need for highly effective drugs that do not have this unintended side effect.

Sometimes a subset of patients may carry genetic variants that cause them to fail to respond to a medication or experience increased side effects. In these cases, new dosing regimens or new therapies may be required to improve efficacy or reduce adverse reactions.

Another important consideration when defining unmet medical need is the severity of the disease. For example, patients with Huntington's disease may be more willing to tolerate significant side effects for a therapy that could delay or prevent disease progression, whereas patients with allergic rhinitis would be less likely to accept a treatment that causes side effects worse than the symptoms themselves.

When assessing unmet medical need, it’s not just about efficacy and safety. Factors such as patient preference, convenience, and cost also play a role. For instance, intravenous treatments that require administration at infusion centers can be both inconvenient and costly for patients. A therapy that offers oral administration with less frequent dosing might dramatically improve patient adherence and quality of life.

An example is azithromycin, an oral antibiotic dosed once per day for five days that has replaced erythromycin, which required four doses daily over a span of seven to ten days. The shift to a more convenient regimen has led to better patient adherence and overall outcomes.

The need for affordable treatments is also growing. As biological drugs become prohibitively expensive, ensuring that treatments are accessible and affordable for diverse populations becomes a critical aspect of identifying unmet medical need.

In some cases, the unmet medical need lies in reaching underserved populations. COVID-19 vaccines that required ultra-cold storage and handling were difficult to distribute to low-resource settings, highlighting a significant barrier to global access. A vaccine requiring only a single dose would result in wider patient uptake and greater efficiency for the health care system.

Understanding unmet medical needs isn’t just about the patient experience. We must also consider healthcare providers, payers, regulators, and society. Even the most promising therapeutic solution is only viable if it aligns with the expectations of these stakeholders.

Once we’ve clearly identified the unmet need, the next step is to evaluate whether our potential therapeutic approach will provide a suitable solution. This involves speaking with physician experts, patient advocacy groups, and regulatory agencies to refine our understanding of what makes an acceptable therapy.

Some of the key questions we need to answer include:

• Will the new drug improve patient outcomes significantly?
• Will it save money for the healthcare system?
• Is the risk-to-benefit ratio acceptable for the intended patient population?
• Will the delivery method be acceptable to both patients and providers?

These questions help ensure that the drug development process remains focused and driven by the actual needs of the patient population.

Understanding the unmet medical need is not just the first step in drug development; it forms the foundation for every scientific, regulatory, and strategic decision that follows. It ensures that our efforts are aligned with the real-world needs of patients and the healthcare system, ultimately driving the creation of meaningful, impactful therapies.