PharmaProjects Rare Disease Case Study Flyer - Informa
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Attaining Insights on the Rare Disease Drug Development Landscape Drug development is a complex endeavor, particularly within rare diseases. Given the additional challenges impacting rare disease R&D, the ability to easily generate critical insights and hone your development strategy is key. Through Pharmaprojects, effectively assess the rare disease landscape, including identification of: Top 20 Companies Developing Orphan Drugs Source: Pharmaprojects ® , September 2020 Key Players With Orphan Drug Status Through the Orphan Drug Designation Program, various development incentives are available to qualifying drugs targeting rare diseases, including exclusivity rights and cost benefits, protocol assistance from FDA, and potential decreased wait-time for drug approval. A client interested in identifying Orphan Drug companies reached out to our analyst team via Ask The Analyst, which is included within Pharmaprojects and provides direct access to our industry experts. The analyst provided an overall strategy as well as suggestions to further refine the search. Searching for drugs in Pipeline development (Global Statuses spanning Preclinical to Pre-registration) that have been granted Orphan Drug Status provides insights into these players possessing a potential advantage within the challenging rare disease space. Overall, 1,292 companies are developing 1,387 drugs that have received Orphan Drug Status. Takeda Pfizer Bristol-Myers Squibb Roche Novartis Sanofi Ligand Pharmaceuticals Johnson & Johnson Amgen GlaxoSmithKline AbbVie Eli Lilly BeiGene Regenxbio Astellas Pharma AstraZeneca Biogen Merck & Co. Bayer Gilead Sciences 0 Number of drugs 5 10 15 20 25 30 35 40 38 35 34 32 27 25 19 18 17 16 15 15 14 14 13 13 13 13 11 11 Drugs with Orphan Drug Status are easily identifiable in Pharmaprojects by using the Event Type field.
Transcript of PharmaProjects Rare Disease Case Study Flyer - Informa
Attaining Insights on the Rare Disease Drug
Development LandscapeDrug development is a complex endeavor, particularly within rare diseases. Given the additional challenges impacting rare disease R&D, the ability to easily generate critical insights and hone your development strategy is key. Through Pharmaprojects, effectively assess the rare disease landscape, including identification of:
Top 20 Companies Developing Orphan Drugs
Source: Pharmaprojects®, September 2020
Key Players With Orphan Drug Status
Through the Orphan Drug Designation Program, various development incentives are available to qualifying drugs targeting rare diseases, including exclusivity rights and cost benefits, protocol assistance from FDA, and potential decreased wait-time for drug approval.
A client interested in identifying Orphan Drug companies reached out to our analyst team via Ask The Analyst, which is included within Pharmaprojects and provides direct access to our
industry experts. The analyst provided an overall strategy as well as suggestions to further refine the search.
Searching for drugs in Pipeline development (Global Statuses spanning Preclinical to Pre-registration) that have been granted Orphan Drug Status provides insights into these players possessing a potential advantage within the challenging rare disease space.
Overall, 1,292 companies are developing 1,387 drugs that have received Orphan Drug Status.
TakedaPfizer
Bristol-Myers SquibbRoche
NovartisSanofi
Ligand PharmaceuticalsJohnson & Johnson
AmgenGlaxoSmithKline
AbbVieEli Lilly
BeiGeneRegenxbio
Astellas PharmaAstraZeneca
BiogenMerck & Co.
BayerGilead Sciences
0
Number of drugs
5 10 15 20 25 30 35 40
383534
3227
2519
1817
1615151414
13131313
1111
Drugs with Orphan Drug Status are easily identifiable in Pharmaprojects by using the Event Type field.
To learn more about Pharmaprojects and the advantages we can deliver to your company, please visit: pharmaintelligence.informa.com or email: [email protected]
US Toll-Free: +1 888-670-8900 US Toll: +1 908-547-2200 UK & Europe: +44 (0) 203 377 3737 Australia: +61 2 8705 6907
*Search results reflects landscape as of September 2020
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“Fallen Angel” Drugs for Rare DiseasesQuickly search for drugs targeting rare diseases using the Drug Disease Group field
Less than 10% of potential new drugs actually make it to market, resulting in a vast pool of “fallen angel” drugs that have already undergone early stages of research and potential candidates for repurposing.
The search can be refined, such as limiting by• Therapeutic group to focus on anticancer drugs, reducing the pool of candidates to 2,629 drugs • Highest development status reached by the drug before discontinuation, such as Phase III, which returns
241 drugs• When the drug dropped out of development to identify recent activity, including discontinuations between
2015-2017, which includes 1,652 drugs
Further inform your research by reviewing reasons behind discontinuation. When publicly disclosed, Pharmaprojects captures these details within the Drug Event Details for the Event Type Discontinuation Confirmed, including:
Identify “fallen angel” drugs for rare diseases by searching for Global Statuses of Discontinued or No Development Reported in the Rare Diseases Drug Disease Group – a total of 7,397 drugs.
Top 5 Diseases with “Fallen Angels”
Cancer, ovarian
Cancer, pancreatic
Cancer, liver
Cancer, leukaemia, acute myelogenous
Cancer, renal
0 100 200 300 400 500 600 700
Number of drugs
604
577
328
359
313
Adverse events
Efficacy
Financial
Strategic
Miscellaneous
Unspecified
0 50 100 150 200 250 300
Number of drugs
177
44
81
38
222
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