Cancer is often challenging, however, diagnosis of rare cancer poses extra difficulties. Of all the diagnosed cancers, approximately 22% of the cases are rare cancers. According to the US National Cancer Institute, a cancer rare cancers as those with an incidence of fewer than 15 cases per 100 000 per year.
Rare cancers when taken into consideration together are more common than any common cancer prevalent in the world. However, these cases often manage to sneak out owing to disproportionate prevalence.
So, what makes any cancer rare? The condition varies. Some experts say if the diagnosis rate is fewer than 2 in 100,000 people each year, while for others, cancer is rare if it affects fewer than 6 in 100,000 each year. This implies that 1 in 5 people diagnosed with cancer has a rare type. DelveInisght has recently published comprehensive insights into Rare cancers putting forward changing dynamics of the Rare cancer market. Insights into some of the Rare cancer market are listed below:
Soft Tissue Sarcoma Market
Soft Tissue Sarcomas (STS) are a rare and heterogeneous group of tumors that make up less than 1% of all adult cancers. These tumors originate from connective tissues including muscles, tendons, blood vessels, fatty and fibrous tissues. Tumor beginning at lower extremities accounts for the majority of cases. The head and neck, as well as the trunk and retroperitoneum, are other possible sites. According to DelveInsight, the total STS incident population (including GIST Cases in the US) in the 7MM (the US, EU5 (the UK, Germany, Italy, Spain, and France) and Japan) was 41,787 in 2020.
Surgery, chemotherapy, radiation therapy, targeted therapy, and antiangiogenesis medications, or a mixture of these treatments, are primary options in the Soft tissue sarcoma market. The mainstay of the STS treatment market is cytotoxic chemotherapy. Doxorubicin, ifosfamide, gemcitabine, taxanes, and a number of other active drugs are also used; they can be used as single agents or in combination regimens. The combination of doxorubicin and ifosfamide is the most widely used therapy. The US FDA-approved drugs comprise Votrient (pazopanib), Yondelis (trabectedin), Lartruvo (olaratumab), Vitrakvi (larotrectinib), Rozlytrek (Entrectinib), and others.
However, the histology-driven approach remains the gold-standard for the treatment of advanced unresectable or metastatic STSs. Novel medical interventions with improved efficacy and fewer side effects are desperately required.
Several organizations are competing in the market focusing on combination approaches that target several molecular pathways and mechanisms, with an emphasis on antagonistic antibodies, TK inhibitors, and inhibitors of downstream PI3K, MAPK, and mTOR molecules. DelveInisght expects an increase in the Soft tissue sarcoma market size during the study period, 2017–2030 at a CAGR of 6.47% in the 7MM.
Key Soft Tissue Sarcoma Pipeline Therapies
- Selinexor (KPT-330): Karyopharm Therapeutics
- Ripretinib (DCC-2618): Deciphera Pharmaceuticals
- GSK3377794: GlaxoSmithKline
- BA3021: BioAtla
- Tazemetostat: Epizyme
Synovial sarcoma (SyS) Market
Synovial sarcoma (SyS) is a rare form of soft tissue sarcoma accounting for 5–10% of all soft tissue sarcomas. It may arise from a number of soft tissues, including muscle or ligaments. Soft-tissue tumors account for 5–10% of all cancers. As per DelveInsight’s epidemiological analysis, SyS incidence in the 7MM was estimated to be 1,610 in 2020, which is expected to further rise in the coming decade.
The primary goal of Synovial sarcoma treatment is safe surgical resection of the entire tumor with clear margins. Radiotherapy can also be used to totally obliterate residual cancer cells, either before or after surgery. Chemotherapy (usually Doxorubicin and/or Ifosfamide) is indicated for advanced or metastatic disease.
Analysts at DelveInsight Rare cancer market forecast analysis, the Synovial sarcoma market is expected to show positive growth, majorly due to increased prevalence and expected launch of novel therapies. In addition, the discovery of multiple biomarkers and comprehensive epidemiological studies in the field are expected to boost the market size by a modest CAGR of 19.14% in the 7 MM during the forecast period 2021-30.
Key Synovial Sarcoma Pipeline Therapies
- AL 3818: Advenchen Laboratories
- ADP-A2M4: Adaptimmune Therapeutics
- GSK3377794: GlaxoSmithKline
Cholangiocarcinoma (CCA) Market
Cholangiocarcinoma (CCA) is a heterogeneous malignant cancer with epithelial cell origin of the biliary duct. It can originate from any section of the bile duct ranging from terminal ductules (canals of Hering) to the ampulla of Vater, as well as at the peribiliary glands (intramural and extramural) and sometimes from the gallbladder.
DelveInsight’s Rare cancer epidemiological analysis demonstrated that the total CCA incidence was recorded to be approximately 73,442 in 2020 in the 7MM, which is expected to increase further during the study period 2018-30.
The Cholangiocarcinoma treatment market is dominated by surgery with adjuvant chemotherapy and chemoradiation therapy. For advanced and metastatic Cholangiocarcinoma cases, systemic chemotherapy is the only option. Surgical treatments are the only potentially curative therapeutic options for all the subtypes of CCA. No established second-line systemic therapy that can be given following the progression after first-line treatment although chemotherapeutic agents either in monotherapy or in combination are used. Besides lack of standard treatment, the majority of CCA patients are diagnosed with late-stage disease, and nearly more than one-fourth of patients considered resectable are found to be unresectable during explorative laparotomy. The CCA market also witnesses higher cases of recurrence of tumor.
Improvements in molecular understanding have led to the development of targeted therapies. Last year, Incyte corporation received approval for its pemigatinib as the first targeted therapy for adults with previously treated, unresectable locally advanced, or metastatic cholangiocarcinoma.
To combat the unmet needs, several pharmaceutical companies and biotech players are investigating novel therapeutic agents revolving around a personalized approach, targeting FGFR gene fusions and somatic mutations in IDH 1/2 in iCCA, PRKACA, or PRKACB gene fusions in pCCA, and ELF3 mutations in dCCA/ampullary carcinoma.
DelveInsight estimates modest growth of the Cholangiocarcinoma market size in the next decade for the forecast period 2021-30 at a CAGR of 12.71% in the 7MM. The driving factors include a better understanding of the molecular pathway of cancer through whole-genome analysis, identified multiple actionable mutations, development of targeted agents with promising results in early clinical trials, increasing incidence, better funding, and a newly-shifted focus towards the Rare cancer market domain.
Key Cholangiocarcinoma Pipeline Therapies
- Ivosidenib: Agios Pharmaceuticals
- Durvalumab: AstraZeneca
- M7824: Merck/EMD Serono
- Infigratinib: QED Therapeutics
- Nucana: Acelarin
- Telotristat ethyl: Lexicon Pharmaceutical
Glioblastoma Multiforme (GBM) Market
A rare tumor found mostly in adults, Glioblastoma multiforme (GBM), is a glioma of grade IV and, according to the classification of WHO 2007, is the most common and lethal type of primary malignancies. DelveInsight has estimated a total of 29,440 GBM incident cases in the 7MM in the year 2020, which is suspected to moderately increase over the next decade.
Surgery is the most common procedure followed to treat GBM patients, preceded by radiation and chemotherapy. The aim of surgery is to achieve “maximal safe resection,” which means removing as much of the tumor as possible without causing irreversible neurological damage. However, since removing all cancerous cells is difficult, any remaining cancer cells are treated with additional therapies such as chemotherapy or radiation therapy.
The clinical course for GBM is chemotherapy with the drug temozolomide. In addition, for recurrent glioblastomas, bevacizumab is usually used as second-line therapy. When both drugs are administered at the same time, Lomustine tends to help enhance the functionality of bevacizumab.
Despite the availability of multidisciplinary therapies, patients with GBM have a median average survival of just 14.6 months. This may be attributable to the high degree of intra-tumor heterogeneity, which means that radical tumor resection isn’t always curative. Other unmet needs include poor prognosis, restricted treatment choices, medication resistance, failure of therapies to cross the BBB (blood-brain-barrier), and a high mortality rate.
Nonetheless, advances in medical research have laid the groundwork in the Rare cancer market for a greater understanding of the disease and, as a result, the emergence of a possible cure. The anticipated implementation of potential therapies in the forecast period 2021-30 is expected to bolster the Glioblastoma Multiforme market growth. DelveInsight anticipates a positive growth of the GBM market size at a CAGR of 12.9% during the forecast period 2021-30, in the 7MM.
Key Glioblastoma Multiforme Pipeline Therapies
- Ofranergene obadenovec: VBL Therapeutics
- Trans Sodium Crocetinate: Diffusion Pharmaceuticals
- Regorafenib: Bayer
- Durvalumab: Astrazeneca
- DNX-2401: DNAtrix
- ONC201: Oncoceutics
- Selinexor (KPT-330): Karyopharm Therapeutics
- Paxalisib (GDC-0084): Kazia Therapeutics
- AV-GBM-1: Aivita Biomedical
- VBI-1901: VBI Vaccines
- MDNA55 : Medicenna Therapeutics
- VAL-083 (Dianhydrogalactitol): DelMar Pharmaceuticals
- pp65-shLAMP DC with GM-CSF (ITI-1000): Immunomic Therapeutics
- Ad-RTS-hIL-12 + Veledimex: Ziopharm
Multiple Myeloma Market
Multiple myeloma is a plasma cell cancer. It’s a hematological malignancy with a wide variety of symptoms. It’s a bone marrow cancer that causes bone fractures (osteolysis) due to an increase in osteoclastic activity and a decrease in osteoblastic activity.
Despite its rarity, it is the second most frequently diagnosed blood cancer in the United States, after non-Hodgkin lymphoma. Males have a higher predisposition than females. According to DelveInsight, the estimated number of Multiple Myeloma incident cases in the 8MM (7MM + China) in 2020 were recorded to be 91,520.
Chemotherapies in combination with proteasome inhibitors, monoclonal antibodies, corticosteroids, and bone marrow transplantation are currently used to treat Multiple myeloma. Melphalan, Vincristine (Oncovin), Cyclophosphamide (Cytoxan), Etoposide (VP-16), and Doxorubicin (Adriamycin) are some of the chemotherapy medications used to treat multiple myeloma. Corticosteroids such as dexamethasone and prednisone are also widely used to treat multiple myeloma.
However, heterogeneity of the tumor, inadequate knowledge of etiology, associated side effects of the available therapies, and cost-burden are some of the serious impediments in the growth of the MM market.
Nevertheless, the anticipated addition of novel therapeutics and increased R&D has contributed to the market growth. DelveInsight’s Rare cancer market anlysis estimates a surge in the Multiple myeloma market size at a CAGR of 2.1% during the forecast period 2021-30 in the 8MM. The launch of BCMA-targeting therapies along with increasing MM incidence is expected to garner much revenue and attention of pharmaceutical stakeholders to explore the domain. Moreover, the expected approvals of the two Chimerican antigen receptor therapies (CAR-Ts), idecabtagene vicleucel (Ide-cel; Bristol Myers Squibb) and ciltacabtagene autoleucel (cilta-cel; Janssen) for relapsed/refractory multiple myeloma is going to significantly contribute to the revenue addition to the Multiple myeloma market.
Key Multiple Myeloma Pipeline Therapies
- Idecabtage ne vicleucel: Bristol-Myers Squibb/Bluebird bio
- Venetoclax: AbbVie and Genentech
- JNJ-68284528: Janssen and Legend Biotech
- Melflufen: Oncopeptides AB
- Keytruda: Merck Sharp & Dohme
- Cetrelimab: Janssen
- REGN5458: Regeneron Pharmaceuticals
- Iberdomide: Bristol-Myers Squibb/Celgene
- NY-ESO-1 C259 T Cells: Adaptimmune Therapeutics /GlaxoSmithKline /Merck
- Braftovi: Pfizer (Array Biopharma)
- Felzartamab (MOR202): MorphoSys AG/I-Mab Biopharma
- JCARH125: Juno Therapeutics (BMS)
- Chidamide (Epidaza): Chipscreen Biosciences
The Rare cancer market represents several unmet medical needs ranging from inadequate knowledge, late diagnosis, limited healthcare access, lack of expertise, the paucity of effective treatment, to cost factors. The rarity of the cancers makes it difficult to study and conduct a clinical trial and as a consequence, the attention of pharma stakeholders is driven further away from rare cancers. Not only rare cancers, but all the rare diseases are also often isolated due to these reasons. Therefore, the Rare disease market needs continuous support and efforts from the scientific community, stakeholders, and private pharmaceutical companies to speed up the process of drug development for rare cancers.