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Product and Pipeline Assessment of the Global Orphan Drugs Market

  • February 2014
  • -
  • Frost & Sullivan
  • -
  • 189 pages

Attractive Market Lures Hundreds of Innovators, Large and Small

Executive Summary

•The US National Institute of Health (NIH) currently recognizes more than xx orphan diseases, approximately xx of which have approved orphan designated therapies.
•The US Food and Drug Administration (FDA) has approved xx orphan drugs since 1983, with at least an additionalxx granted orphan drug designations. Japan ranks second, with xx approved orphan drugs, and Europe ranks third, withxx approved orphan drugs.
•Rare cancers is the therapeutic area with the highest level of drug development activity and the most well penetrated, with xx drugs approved in the United States for xx rare cancer designations. Other disease areas of high drug development activity include blood/lymphatic system diseases, infectious/parasitic diseases, neurologic diseases, metabolic diseases, and immunological/inflammatory diseases.
•Advancements in biomedical research are finally yielding breakthrough therapeutics with true disease-modifying properties rather than palliative care or symptom management, such as Alexion’s Soliris and Vertex Pharmaceuticals’ Kalydeco for cystic fibrosis.
•Pipeline drugs targeting orphan diseases use diverse approaches such as small molecules, antisense, gene therapy, monoclonal antibodies (mAb), bi-specific antibodies, peptide therapies, and stem cell and other cell therapies to correct the underlying pathology of disease. Many are targeted therapies designed to address a specific genetic defect for a personalized medicine approach, and many have strong potential to slow or stop disease progression rather than simply manage the disease symptoms.
•The pipeline is generating hope for some of the most serious and debilitating or life-threatening rare diseases in need of better therapies, such as pancreatic cancer with xx candidates in Phase 3 and an additional xx in earlier stages of development, Pompe disease with xxpromising candidates in late-stage development, and amyotrophic lateral sclerosis with xxcandidates in late-stage development.
•This research service provides an overview and analysis of global orphan drugs development, with an emphasis on the US market.
•Unless otherwise noted, marketed and pipeline products described are orphan designated. There may be additional compounds in development for the covered indications that are not orphan designated. While some non-orphan drugs have been included when deemed appropriate, this research service focuses on orphan-designated drugs.
•All attempts have been made to include a comprehensive and timely pipeline assessment of select disease areas with a high degree of activity and relevance; however, because of the dynamic nature of orphan designation, some compounds that have been newly designated may not be included.
•Data and supporting information were collected from published sources, including the following:
oUS FDA Orphan Drug Product Designation database
oUS Department of Health and Human Services National Institutes of Health Office of Rare Diseases
oEuropean Commission Community Register of Medicinal Products
oEuropean Medicines Agency (EMA) European Public Assessment Reports (EPAR)
oJapanese Ministry of Health, Labour, and Welfare (MHLW)
oDisease organization Web sites
oCompany publications, including annual reports, US Securities and Exchange Commission (SEC) filings, and press releases
oPublished articles in scientific journals

Market Background

Drug development for rare diseases once primarily consisted of a side project for a drug already in development for a more profitable indication. If that side project could be conducted without too much of a financial burden, drug developers were able to provide a handful of patients with a desperately needed treatment while also ensuring return on investment through the more profitable indication of a larger patient population. However, these breakthroughs were too few and far between and left far too many patients underserved. Therefore, government legislation such as the Orphan Drug Act was instituted to provide the needed financial incentives to pharmaceutical companies to step up drug development for orphan diseases.

The Orphan Drug Act, signed into law in 1983 by the US government, was the first of such legislation and laid the groundwork for similar government initiatives in other regions. In 1993, the Japanese government adopted a similar version of orphan drug legislation, followed by Australia in 1997 and Europe in 2000. Although these regulations provide for special considerations for drugs for rare diseases, each differs slightly in its definition of a rare disease as well as the particular incentives conferred.
Development and commercialization of orphan drugs have evolved into an attractive market for both multinational pharmaceuticals as well as small-to-midsize companies because of the before-mentioned regulatory provisions as well as generous reimbursement allowances. In addition, the extra assistance provided by the regulatory agencies in the case of orphan designation helps enable smaller, less experienced companies to navigate successfully the complicated drug development and approval process.

Depending on the drug’s target and mechanism of action (MOA), orphan drugs can potentially be useful to treat multiple diseases, both orphan and non orphan. For example, Novartis AG’s Gleevec was first approved and launched for chronic myeloid leukemia in 2001. It has since also been approved for gastrointestinal stromal tumors (GIST) in 2002, and acute lymphocytic leukemia, soft tissue sarcoma, and myelodysplastic syndrome in 2006.
For drugs designated for serious diseases with no other therapeutic alternatives, or if a new drug offers substantial clinical benefits over available therapies, payers are willing to reimburse at much higher rates compared to conventional therapies.

A case in point is Alexion Pharmaceuticals, Inc.’s Soliris (eculizumab), with the highest price tag of any drug worldwide at $xxa year per patient. Soliris is a breakthrough drug that safely and effectively treats essentially every patient with paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder of the complement system that causes anemia. Because of the severity of the disease and the effectiveness of the drug, payers are willing to pay the high price tag, thereby ensuring a good return on investment for Alexion. Soliris is also approved for atypical hemolytic uremic syndrome and is being developed for neuromyelitis optica, a rare but serious neurological condition similar to multiple sclerosis (MS). Because of its MOA involving the complement system, a mechanism potentially conserved across numerous disease conditions involving the immune system, its potential is also being explored in dozens of investigator-initiated trials for other conditions, including kidney transplantation, macular degeneration, CD59 deficiency, dense deposit disease and C3 nephropathy, and cold agglutinin disease.

Initial market penetration as an orphan drug is also often a successful strategy for drugs with potential utility in much larger patient populations. Initial approval for an orphan indication allows for faster time to market, which is then proceeded by eventual label expansion to include a broader indication(s). For example, Novartis AG’s Afinitor, a mammalian target of rapamycin (mTOR) inhibitor, was first approved for tuberous sclerosis and pancreatic neuroendocrine tumors, followed by approval for the broader indications of kidney and breast cancers.

However, a caveat to this strategy is that while an orphan drug can justify a high price tag for serious and rare diseases, payers may not be willing to pay the same price for non-orphan indications for which several other treatment options may exist. Also, the extra exclusivity afforded to orphan drugs does not apply to non-orphan indications.
Many popular drugs were approved for diseases that were previously considered orphan indications but are no longer rare. For example, Avonex was approved for MS when that disease was still considered rare. Avonex has xx additional orphan approvals: juvenile rheumatoid arthritis, pulmonary fibrosis, and primary brain tumors. Remicade, a popular anti-inflammatory drug used in multiple indications, was approved for Crohn’s disease when that condition was considered rare.

Table Of Contents

Product and Pipeline Assessment of the Global Orphan Drugs Market
Executive Summary
Methodology and Scope
Market Background
Global Overview
US and European Markets
Pipeline Analyses for Select Rare Diseases—Blood and Lymphatic System Diseases
Pipeline Analyses for Select Rare Diseases—Cardiovascular Diseases
Pipeline Analyses for Select Rare Diseases—Lung Diseases
Pipeline Analyses for Select Rare Diseases—Congenital and Genetic Diseases
Pipeline Analyses for Select Rare Diseases—Connective Tissue/Skin Diseases
Pipeline Analyses for Select Rare Diseases-Digestive/Nutritional Diseases
Pipeline Analyses for Select Rare Diseases—Endocrine Diseases
Pipeline Analyses for Select Rare Diseases—Exposure/toxicity
Pipeline Analyses for Select Rare Diseases—Eye Diseases
Pipeline Analyses for Select Rare Diseases—Immunological/Inflammatory Diseases
Pipeline Analyses for Select Rare Diseases—Infectious/Parasitic Diseases
Pipeline Analyses for Select Rare Diseases—Metabolic Diseases
Pipeline Analyses for Select Rare Diseases—Musculoskeletal Diseases
Pipeline Analyses for Select Rare Diseases—Neurologic Diseases
Pipeline Analyses for Select Rare Diseases—Rare Cancers
Company Analysis
Conclusions
Appendix
The Frost and Sullivan Story

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