The Most Expensive Medicines in the World
The Most Expensive Medicines in the World
Expensive medicines are often innovative therapies with high costs due to the extensive research, development, and manufacturing involved, as well as their effectiveness in treating rare or complex diseases. Here’s a list of the 7 most expensive drugs, along with information on each, including their applications, mechanisms of action, market impact, and unique features.
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Zolgensma
Diseases: Spinal Muscular Atrophy (SMA)
Mechanism: Gene therapy that repairs the defective SMN1 gene.
Market Change: Introduced a revolutionary approach to treating genetic diseases with a single gene therapy.
Differential: The first gene therapy cure for SMA.
Laboratory: Novartis
Advances Post-Medicine: Increased availability of gene therapies.
Patient Life Improvement: Cure of SMA in children leads to a near-normal life.
Cure: Yes
Life Improvement: Significant -
Luxturna
Diseases: Inherited Retinal Degeneration
Mechanism: Gene therapy that inserts a functional copy of the RPE65 gene into the retina.
Market Change: Opened the use of gene therapies in ophthalmological diseases.
Differential: The first gene therapy for an inherited eye disease.
Laboratory: Spark Therapeutics
Advances Post-Medicine: Accelerated research in therapies for rare eye diseases.
Patient Life Improvement: Significant improvement in vision and quality of life.
Cure: No
Life Improvement: High -
Soliris (Eculizumab)
Diseases: Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
Mechanism: Inhibits the C5 protein in the complement system, preventing red blood cell destruction.
Market Change: Changed the approach to treating rare blood diseases.
Differential: The first effective therapy for complement-related diseases.
Laboratory: Alexion Pharmaceuticals
Advances Post-Medicine: Development of more effective complement therapies.
Patient Life Improvement: Reduces severe complications and increases survival.
Cure: No
Life Improvement: Significant -
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Kymriah
Diseases: Acute Lymphoblastic Leukemia (ALL), Lymphoma
Mechanism: CAR-T cell therapy that reprograms the patient’s T cells to attack cancer cells.
Market Change: Paved the way for genetically modified T-cell therapies.
Differential: Effective treatment for certain types of leukemia and lymphoma.
Laboratory: Novartis
Advances Post-Medicine: Advances in T-cell therapies for other types of cancer.
Patient Life Improvement: Significant increase in remission and cure rates in some patients.
Cure: Yes (in some cases)
Life Improvement: High -
Hemlibra
Diseases: Hemophilia A
Mechanism: Replaces coagulation factor VIII to prevent bleeding.
Market Change: Became an effective and easier-to-administer alternative for hemophilia A patients.
Differential: More accessible therapy with fewer complications than previous treatments.
Laboratory: Genentech
Advances Post-Medicine: Development of more effective therapies for bleeding disorders.
Patient Life Improvement: Reduces bleeding episodes and improves quality of life.
Cure: No
Life Improvement: High -
Adakveo
Diseases: Sickle Cell Anemia
Mechanism: Reduces the adhesion of sickle cells in blood vessels, preventing crises.
Market Change: Provided a new therapeutic option for a rare and debilitating disease.
Differential: Alleviates painful crises in sickle cell patients.
Laboratory: Novartis
Advances Post-Medicine: Expanded treatment options for hematological diseases.
Patient Life Improvement: Improved quality of life and reduced hospitalizations.
Cure: No
Life Improvement: Significant -
Blincyto
Diseases: Acute Lymphoblastic Leukemia (ALL)
Mechanism: Bispecific antibody that links T cells and leukemia cells, activating the destruction of malignant cells.
Market Change: Innovation in the treatment of leukemias and lymphomas.
Differential: Uses the patient’s own immune response to treat cancer.
Laboratory: Amgen
Advances Post-Medicine: Increased use of bispecific antibodies in other oncological conditions.
Patient Life Improvement: Improved remission rates and survival.
Cure: No
Life Improvement: High
Table: Cure and Life Improvement
| Medicine | Disease | Cure | Life Improvement |
|---|---|---|---|
| Zolgensma | Spinal Muscular Atrophy (SMA) | Yes | Significant |
| Luxturna | Inherited Retinal Degeneration | No | High |
| Soliris | Paroxysmal Nocturnal Hemoglobinuria | No | Significant |
| Kymriah | Acute Lymphoblastic Leukemia | Yes (in some cases) | High |
| Hemlibra | Hemophilia A | No | High |
| Adakveo | Sickle Cell Anemia | No | Significant |
| Blincyto | Acute Lymphoblastic Leukemia | No | High |
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Zolgensma,
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Luxturna,
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Soliris,
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Kymriah,
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Hemlibra,
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Adakveo,
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Blincyto,
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Opdivo,
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Keytruda,
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Darzalex,
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Repatha,
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Spinraza,
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Nusinersen,
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Stelara,
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Nivolumab,
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Emicizumab,
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Rybelsus,
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Tecentriq,
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Gleevec,
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Cyramza,
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Imbruvica,
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Xarelto,
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Vitrakvi,
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Alectinib,
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Biktarvy,
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Ocrevus,
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Kineret,
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Enbrel,
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Dupixent,
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Trodelvy,
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Regeneron,
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Vyndaqel,
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Imfinzi,
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Vosevi,
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Kymriah,
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Darzalex Faspro,
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Yescarta,
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Blincyto,
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Avsola,
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Tanzeum,
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Treanda,
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Nivolumab,
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Tivdak,
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Farxiga,
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Keytruda,
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Repatha,
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Eculizumab,
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Gazyva,
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Cotellic,
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Tecfidera,
