Tufts Center for Drug Development

In an effort to extend product life cycles and generate larger and longer revenue streams per product, pharmaceutical and biotechnology companies have stepped up their efforts to seek approval for new or modified indications of already approved drugs. 

A recent study conducted by the Tufts Center for the Study of Drug Development finds that the number of new or modified indication approvals following the original new drug approval (NDA) increased 17% between the periods 1998 to 2003 and 2004 to 2009. Original NDAs as a proportion of total drug approvals continues to decline gradually. The number of new drug approvals for pediatric indications following original NDAs rose sharply, increasing 107% between 1998 and 2009. The number of NDA new indications increased 15%, and the number of supplemental NDA new indications declined 5% during this period. Anti-infective and CNS drugs each accounted for one-fifth of all new or modified indication approvals between 1998 and 2009. Cardiovascular and oncology drugs accounted for 15% and 13% of all new or modified indications respectively. Moving forward, with finite resources, sponsors must strike the right balance in maximizing portfolio value by allocating R&D resources between novel, original NDAs, and new or modified indication approvals. 

—Tufts Center for the Study of Drug Development, 

Articles

Rising Proportion of New and Modified Indication Approvals

A recent study conducted by the Tufts Center for the Study of Drug Development (Tufts CSDD) found that more than half of all protocols require one or more amendments. Based on data from 3,410 protocols provided by 17 large and midsize pharmaceutical and biotechnology companies, Tufts CSDD found that completed protocols across all phases had an average of 2.5 amendments requiring 6.9 changes to the protocol per amendment. Later-stage Phase II and III protocols had a higher average number of amendments, 2.7 and 3.5 respectively. The most commonly cited causes of amendments included the availability of new safety information, requests from regulatory agencies to amend the study, and changes in the study strategy. Protocol design flaws and difficulties recruiting study volunteers were also cited as top causes. The study found that the incidence of amendments is associated with protocol complexity and duration. The mean number of amendments per protocol, for example, was positively and significantly correlated (p<.001) with the number of procedures per protocol, study length, and the number of investigative sites conducting that protocol. 

 Mean number of amendments per protocol. (Source: Tufts Center for the Study of Drug Development) 

A recent study conducted by the Tufts Center for the Study of Drug Development (Tufts CSDD) found that more than half of all protocols require one or more amendments.

Most Protocols Require Multiple Amendments

Biotechnology is delivering substantially higher numbers of new product approvals. During the period 2000 to 2009, 65 biopharmaceutical products received US marketing approval, nearly double the number of products approved during the 1990 to 1999 period and five times the 1980 to 1989 level.  

Biopharmaceutical approvals represented one-third of all new therapeutics approved by the FDA between 2000 and 2009. This is up from 7% and 14% of all new therapeutics approved by the FDA between 1980 to 1989 and 1990 to 1999 respectively. Recombinant proteins comprise the majority of new biopharmaceutical approvals at 57% of the total. Monoclonal antibody product approvals now account for 28% of all new biopharmaceutical approvals, up from only 8% of new product approvals between 1980 and 1989. Half of new biopharmaceuticals approved by the FDA during the 2000s received a priority-review rating.  

Biotechnology is delivering substantially higher numbers of new product approvals. During the period 2000 to 2009, 65 biopharmaceutical products received US marketing approval, nearly double the number of products approved during the 1990 to 1999 period and five times the 1980 to 1989 level.

Strong Growth in Biopharmaceutical Approvals

A 2010 survey conducted by the Tufts Center for the Study of Drug Development (Tufts CSDD) finds that sponsor companies are allocating resources, modifying organizational structures, and increasing investment in the development of personalized medicines (i.e., tailoring of medical treatment and healthcare delivery based on individual patient characteristics including genetic, molecular, and imaging). 

 The average percentage of company pipelines relying on biomarker data.			

Responses from 25 companies show that all sponsors are using biomarkers and/or targeted therapies to evaluate compounds in discovery. The use of biomarkers and/or targeted therapies in clinical are less common at this time, with the highest incidence in early stages of development. Nearly 60 percent and 50 percent—on average—of sponsor company preclinical and Phase I/IIA clinical pipelines respectively rely on biomarker data. Only a third of late stage Phase IIb/IV clinical pipelines—on average—rely on biomarker data. The Tufts CSDD found that oncology is the therapeutic area the farthest along in integrating biomarkers and diagnostics. Development programs in cardiovascular, CNS, and immunologics are less advanced than oncology but they are making headway in integrating personalized medicine practices.

— Tufts Center for the Study of Drug Development, 

A 2010 survey conducted by the Tufts Center for the Study of Drug Development (Tufts CSDD) finds that sponsor companies are allocating resources, modifying organizational structures, and increasing investment in the development of personalized medicines (i.e., tailoring of medical treatment and healthcare delivery based on individual patient characteristics including genetic, molecular, and imaging).

Author | Tufts Center for Drug Development