Unmet Medical Need: Abnormal Circulating Thyroid Hormone Concentration


1. Disease Summary:

Abnormal circulating thyroid hormone concentrations encompass both hyperthyroidism (excess thyroid hormones) and hypothyroidism (deficiency of thyroid hormones). These conditions can significantly impact various bodily functions, leading to a range of symptoms and complications. Hyperthyroidism is characterized by symptoms such as weight loss, anxiety, palpitations, and heat intolerance, while hypothyroidism can cause fatigue, weight gain, cold intolerance, and depression. Both conditions can lead to severe health complications if left untreated, including cardiovascular issues, osteoporosis, and in the case of hyperthyroidism, adverse pregnancy outcomes.

2. Global Prevalence and Disease Burden:

  • Hyperthyroidism: Affects approximately 0.2% to 1.4% of the global population, with subclinical hyperthyroidism affecting about 0.7% to 1.4% (Lee SY, Pearce EN, 2023, PMID: 37847271). The most common cause is Graves' disease, which has a prevalence of 2% in women and 0.5% in men.
  • Hypothyroidism: Overt hypothyroidism affects about 1% to 2% of the population, while subclinical hypothyroidism can affect up to 10% of adults (Chaker L, Razvi S, Bensenor IM, 2022, PMID: 35589725). The burden of hypothyroidism is significant, as untreated cases can lead to cardiovascular diseases, cognitive impairment, and decreased quality of life.

3. Unmet Medical Need:

Despite the availability of treatments, several unmet medical needs persist in managing abnormal thyroid hormone concentrations:
  • Personalized Treatment: Many patients report a desire for more individualized treatment plans that consider their unique symptoms and responses to therapy. Current treatment protocols often apply a one-size-fits-all approach, which may not address the specific needs of each patient (Source: medRxiv).
  • Persistent Symptoms: A significant proportion of patients treated with levothyroxine for hypothyroidism continue to experience symptoms despite normalized thyroid function tests. This indicates a need for better understanding and management of residual symptoms (Taylor PN et al., 2024, PMID: 39368843).
  • Psychosocial Support: Patients with thyroid disorders often report psychological distress, anxiety, and depression, highlighting the need for integrated care that includes mental health support (Source: medRxiv).
  • Access to Care: There are disparities in access to specialized care for thyroid disorders, particularly in rural or underserved areas. Patients may not receive timely diagnosis or treatment, leading to worsened health outcomes.
  • Education and Awareness: There is a lack of awareness among patients regarding the importance of regular monitoring and management of thyroid hormone levels, which can lead to complications if not addressed.

4. Current Treatment Options:

  • Hypothyroidism: The primary treatment is levothyroxine, a synthetic form of the thyroid hormone thyroxine (T4). It is effective in normalizing hormone levels and alleviating symptoms for most patients. However, about 10% of patients report persistent symptoms despite treatment (Taylor PN et al., 2024, PMID: 39368843). Combination therapy with liothyronine (T3) is sometimes used, but its efficacy remains debated.
  • Hyperthyroidism: Treatment options include antithyroid medications (e.g., methimazole), radioactive iodine therapy, and surgery. Antithyroid medications are typically used for 12 to 18 months, but long-term management can be challenging, and some patients may experience relapse (Lee SY, Pearce EN, 2023, PMID: 37847271).

5. Current Clinical Trials:

Ongoing clinical trials are exploring various aspects of thyroid hormone management, including:
  • The efficacy of combination therapies for hypothyroidism.
  • New formulations of levothyroxine that may improve absorption and patient outcomes.
  • Investigations into the genetic factors influencing individual responses to thyroid hormone treatments.

6. Additional Context:

The economic impact of thyroid disorders is substantial, with costs associated with diagnosis, treatment, and management of complications. The burden on healthcare systems includes direct costs (medications, hospitalizations) and indirect costs (loss of productivity, decreased quality of life). Addressing the unmet medical needs in this area could lead to improved patient outcomes and reduced healthcare costs.
In summary, while treatments for abnormal thyroid hormone concentrations exist, significant unmet needs remain, particularly in personalized care, symptom management, and psychosocial support. Addressing these needs is crucial for improving the quality of life for patients with thyroid disorders.