According to PharmaField, the Scottish Medicines Consortium (SMC) recently approved and accepted 14 new medicines to be used by NHSScotland. Many of these medications, such as Xospata, are used to treat patients with rare diseases or conditions. Below, we will unpack what medicines were approved and what conditions they treat.

SMC Acceptance

The Scottish Medicine Consortium (SMC) describes themselves as:

a committee of clinicians, pharmacists, NHS board representatives, the pharmaceutical industry and the public. Members of the committee consider a broad range of evidence in order to decide which medicines should be accepted for use by NHSScotland.

Recently, the SMC approved and accepted the following medications.

Cannabidiol (Epidyolex)

Lennox-Gastaut syndrome (LGS) is a rare, severe form of epilepsy that begins in childhood. It is responsible for recurrent and severe seizures, developmental delays, and behavioral problems. Now, researchers approved cannabidiol to treat LGS. Researchers analyzed the efficacy and impact of Cannabidiol through the PACE process, designed to develop medicines for rare conditions. Clinicians do note that Cannabidiol is most effective in reducing seizure severity and frequency when used in conjunction with clobazam.

Additionally, Cannabidiol and clobazam can be used together to treat Dravet syndrome. Dravet syndrome is a rare genetic brain dysfunction that begins within a healthy child’s first year of life. Patients with Dravet syndrome often experience prolonged and severe seizures.

Esketamine (Spravato)

The SMC accepted esketamine (Spravato) for adults patients with treatment-adverse major depression. Patients using Spravato have most likely tried at least two, if not more, prior treatment. A clinician intranasally administers Spravato. It can be used in conjunction with additional antidepressants.

Pembrolizumab (Keytruda)

In a PACE discussion, clinicians and patient groups explored Pembrolizumab (Keytruda) for patients with either metastasized or inoperable head and neck squamous cell carcinoma (HNSCC), or renal cell carcinoma. Both conditions cause severe pain, fatigue, and other symptoms that interrupt quality of life. The prognosis for patients with HNSCC is not positive, with a usual survival rate of less than one year following diagnosis. Renal cell carcinoma, an aggressive and easily spreading kidney cancer, is incurable and also has a low survival rate. Clinicians believe that Keytruda could assist patients with improving quality of life and survival rate.

Gilteritinib (Xospata)

PACE also contributed to Gilteritinib (Xospata) acceptance. The drug treats patients with acute myeloid leukemia (AML) with FLT3 gene mutations. AML is a cancer of the blood and bone marrow. Patients with these specific mutations are more likely to experience relapse. Xospata also offers a more accessible orally-administered solution than chemotherapy. Researchers believe Xospata can reduce the need for stem cell transplants and increase survival rate.

Iluvien

Iluvien, also known as a fluocinolone acetonide intravitreal implant, is injected into the eye and lasts for a 3-year period. The implant is designed to prevent the recurrence of non-infectious uveitis. This condition causes uveal inflammation, eye pain and sensitivity, and disrupted vision.

Alternate Treatments with SMC Acceptance

The SMC accepted the following medicines in an expedited manner. This decision focuses on providing patients with medication and treatment even during the pandemic:

• Pertuzumab for breast cancer
• Brolucizumab (Beovu) for wet macular degeneration, a severe eye disease causing dramatic changes in vision
• Lokelma for hyperkalemia, or excess potassium in the bloodstream
• Caplacizumab (Cablivi) for acquired thrombotic thrombocytopenic purpura (aTTP), a disorder causing issues with blood clotting

Finally, the SMC accepted Ondexxya, Polivy, and Holoclar for the treatment of various conditions. However, these are subject to further testing and evaluation.