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Drug Discovery in Rare Diseases


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Drug Discovery in Rare Diseases

  1. 1. Drug Discovery in Rare DiseasesReport Details:Published:August 2012No. of Pages: 108Price: Single User License – US$3800In comparison with major diseases, the targeting of rare diseases poses many differentchallenges, necessitating consideration of bespoke R&D strategies for drug discovery efforts to besuccessful. This report examines the role that low disease prevalence plays in determining themost suitable R&D path.Features and benefits•Understand the growing interest in developing new treatments for rare diseases, and why low patient numbers do not preclude commercial viability.•Review the regulatory environment governing the development of orphan drugs in different countries.•Identify the key challenges that are presented by low patient prevalences.•Assess how the research strategy chosen can be influenced by the disease prevalence.•Compare the scenarios in which repurposing of existing drugs offers advantages over the development of novel drugs, and vice versa.HighlightsApproximately 7,000 rare diseases have been identified, but only a very small proportion of theseare currently well treated. Orphans represent a greater proportion of all new BLAs than they do ofNMEs submitted as NDAs. Most orphan approvals are not first approvals of new drugs but arenew orphan indications for previously approved drugs.It is possible to obtain orphan drug designation for conditions with a total prevalence greater thanthat defined by legislation, but only if medically justifiable subsets can be defined with a lower(overall) prevalence. Pediatric subsets are most commonly used.In the development of new treatments for rare diseases it is less critical to seek to optimize thepharmacokinetic properties of candidates than is the case for common chronic diseases, withparenteral delivery or frequent oral dosing being much more acceptable provided that efficacy isachieved.Your key questions answered•What impact on R&D strategy does disease prevalence have on moving from rare through very rare to ultra rare diseases?•Can more than one drug be a commercial success for treating rare indications, and how do drug regulators view the question of drug similarity?
  2. 2. •What are the best ways of identifying patients for recruitment into clinical trials for drugs designed to treat rare diseases?•What are the requirements for a clinical candidate to treat a rare disease?•What factors determine whether a small-molecule or biologic strategy is most suitable when targeting a rare disease?Get your copy of this report @ points covered in Table of Contents of this report includeTable of ContentsEXECUTIVE SUMMARYKey findingsThe growing interest in rare diseasesSummaryIntroductionWhat is a rare disease?Why target rare diseases?Characterization of rare diseasesSummaryOverviewRare diseasesVery rare diseasesUltra rare diseasesUseful resourcesOrphan drug statusSummaryIntroductionLegislative distinctionsUSEuropeJapanOther marketsTax benefitsPatient population subsettingSimilaritySummary of key considerationsChoosing rare diseases to targetSummaryIntroductionKey issuesCommercial potential
  3. 3. PrevalenceGeographic distributionDisease understandingAvailable expertsCurrent treatmentsWhat is similar?ConclusionPreclinical developmentSummaryIntroductionScreen or repurpose?RepurposedDirected approachesTaking advantage of orphan drug statusOther issuesSmall molecule or biological?Biological test modelsRequirements of a clinical candidateConclusionsClinical issuesSummaryIntroductionClinical trial designAccess to patientsGeographic distributionIdentifying patientsConclusionsCommercial considerationsSummaryIntroductionIdentifying commercially promising opportunitiesPrevalenceCurrent treatmentsCompetitive positionCase studies(Untitled sub-section)Gaucher diseaseConclusionsConclusionsSummaryIntroductionChecklist to consider
  4. 4. Corporate strategiesConclusionsAppendixScopeMethodologyGlossary/abbreviationsBibliography/referencesList of TablesTable: Timeline of international orphan drug legislation, 1983–2008Table: Comparison of orphan drug legislation in the US, Europe, and JapanTable: The FDAs definitions of similarityList of FiguresFigure: US FDA orphan approvals, 2001–11 (part 1)Figure: US FDA orphan approvals, 2001–11 (part 2)Figure: Rare diseases, medical need, and disease prevalenceFigure: Distribution of US orphan drug approvals by disease prevalence (to 2010)Figure: European orphan drug approvals by disease prevalence (to 2010)Figure: Exploiting knowledge resources for rare disease researchFigure: The organizational relationships within the EMA pertaining to orphan drugsFigure: Schematic relationship between rare disease prevalence and commercial returnsFigure: Identifying suitable patients for clinical studiesFigure: The influence of prevalence on research strategyFigure: Current exploitation of rare disease spaceFigure: "Similar" approved BCR-ABL inhibitors with orphan drug statusFigure: Similar approved endothelin receptor antagonists with orphan drug statusFigure: Selecting a rare disease to targetFigure: Alternative strategies to identifying new treatments for rare diseasesFigure: Strategic pathways for identifying development candidatesFigure: Comparison of duration and size of clinical studies in developing drugs to treat majordiseases (a), and rare diseases (b)Figure: Number of participants in European orphan drug clinical trialsFigure: Comparing commercial opportunitiesFigure: Strategic options for developing drugs for already targeted rare diseasesFigure: Different approaches to treating cystic fibrosisFigure: GlaxoSmithKline and Pfizer; exploiting internal and external resources in rare diseaseresearchContact: for more information.