Personalized medicine

With the rise of combination therapy – the use drugs with different mechanisms of action to combat a specific disease state – comes the need to address medical costs and reimbursement issues.  Joint negotiation of package deals with government and health insurers may prove useful, particularly for companion diagnostics and treatment of chronic conditions.  Companies that share drug development risks and costs (preclinical, clinical trials, sales and marketing, etc.) with each other are not only better positioned to negotiate for reimbursement but are also better poised to defend against competition.   Multiple collaborations, however, increase the risk of legal complexity for all concerned.

Source: Reuters

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For pharmaceutical companies, is personalized medicine more of a threat than an opportunity?  In addition to the development of new drugs, genetic information can also help target the use of current medications (e.g., Plavix).  The use of genetic (or other) information to target patient population subsets is expected to increase drug safety and render cost savings to both insurer and patient, but can it also be expected to limit the potential market and lower pharmaceutical sales?  By potentially enhancing drug safety, personalized medicine is expected to elicit fewer adverse drug reactions, thereby leading to fewer liability claims against drug companies.  Drug development costs rise, however, if preclinical scientists also must isolate a genetic trigger and develop a companion test for a treatment, even if the size of clinical trials can potentially be reduced and additional income can be expected through purchase of both medication and companion diagnostic.  Even when a drug is utilized in target populations, how much risk will be deemed acceptable?   Whether personalized medicine stimulates or inhibits pharmaceutical drug development remains to be determined.

Source: Wall Street Journal

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Ideally, every new drug would represent an unprecedented breakthrough and lead to the creation of a
completely novel treatment. This, however, is not the reality of the pharmaceutical industry, or of any other
development-based industry. Creating drugs based on incremental innovations provides pharmaceutical
companies with a secure stream of revenue, which can be directed to higher-risk, potential blockbuster-yielding
research. Policies aimed at reducing the industry’s ability to obtain revenues from incremental innovations
could be self-defeating, as those industries will then have less revenue to reinvest in R&D for new drugs. Put
simply, limiting incremental drug innovation is analogous to limiting competition. The ultimate result could
have devastating consequences for the future of the pharmaceutical industry and for the millions of patients
who depend on it.

Ideally, every new drug would represent an unprecedented breakthrough and lead to the creation of a completely novel treatment.  This, however, is not the reality of the pharmaceutical industry, or of any other development-based industry.  Most new drugs represent the combined weight of seemingly small improvements achieved over time.  Creating drugs based on incremental innovations provides pharmaceutical companies with a secure stream of revenue, which can then be directed to higher-risk, more innovative research.  Many critics contend that “Me-too” drugs — drugs within the same chemical class as one or more already on the market — add little or no therapeutic value to existing formularies.   Conversely, advocates claim that new drugs based on incremental improvements generally represent advances in safety, efficacy, selectivity, and ultimately increase the utility of drugs within a specific therapeutic class.  Innovations may also include new formulations and dosing options.  Changes in one or more of these parameters generally increase patient compliance and improve health outcomes.  Furthermore, patients can respond differentially to drugs within a single class, thus having multiple drug options within a therapeutic class enables optimization of medical treatment to best fit a patient’s needs.  From an economic standpoint, while it is unrealistic to presume that every incremental innovation leads to cost savings, the sum of all drug innovations can reduce overall treatment costs, shorten or eliminate hospitalization, increase worker productivity and reduce absenteeism, and eventually lower drug costs through increased competition among manufacturers.

In conclusion, policies aimed at reducing an industry’s ability to obtain revenue from incremental innovations could be self-defeating, as less revenue will be available to reinvest in research and development.  In pharmaceutical terms, limiting incremental drug innovation is analogous to limiting competition.  The result could have devastating consequences for the future of the pharmaceutical industry and ultimately for patients.

Source:  Competitve Enterprise Institute

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Rare diseases can be described in terms of incidence, etiology, morbidity, and survival.  Incidence can vary, however, from rare (e.g., Gaucher disease, cystic fibrosis) to epidemic proportions (e.g., HIV, malaria, cholera).  The incidence of less frequently occurring cancers (e.g., pancreatic and renal cell carcinoma, myeloma, and glioma), although more common in relation to other rare diseases, meet most regulatory requirements for orphan designation.

In a report of the top 200 brand drugs by retail dollars in 2007, 17 top brands (retailing from $144.7 million to $1.837 billion) had an approved orphan use.  Six of these 17 top drugs, in terms of retail dollars, were indicated solely (USA) for an orphan use: opioid dependence, organ transplant rejection, relapsing multiple sclerosis, and cystic fibrosis.  Eleven of the top 17 brands had an additional 2-9 approved uses, which likely factored favorably into their retail sales volume.

Blockbusters can get their start in rare diseases.  Botox (botulinum toxin type A) was first approved in 1989 as an orphan drug for a rare eye movement disorder (blepharospasm) associated with dystonia before seeking approval for cosmetic use.   ClinicalTrials.gov and the Pharmaceutical Research and Manufacturers of America (PhRMA) reported that over 80% of clinical research trials for rare diseases were not industry sponsored.

In regard to the misuse of the Orphan Drug Act, attempts to further subdivide diseases to achieve questionable subsets small enough to qualify for orphan designation seem unlikely to succeed given the requirement to provide support that a condition is a recognized disease with documented incidence.  There are valid and appropriate ways to target subset populations (e.g., pediatrics, refractory patients, severe forms of a more common disease, specific genotype).

The 2007 FDA Amendments Act (FDAAA) priority review (transferable) voucher incentive program and the 2008 common application form agreement between the USA and EU regulatory bodies demonstrate further global  support for rare diseases.  The outlook going forward is that orphan indication exclusivity can be one aspect of a profitable drug’s overall product life cycle, including non-orphan uses, and can contribute to its profitability.

Source:  Drug Information Journal

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FDA Encourages Applications for Orphan-Drug Status

Posted by cdavenport on Friday Mar 12, 2010 Under FDA, Personalized medicine

Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including 7 years’ marketing exclusivity and tax breaks.  Last year, just 250 requests for orphan-drug designation were filed, and 160 were successful.  There are roughly 350 orphan drugs approved, covering about 150 rare diseases.  Timothy Coté, director of the FDA’s Office of Orphan Products Development, said that his office is offering 2 workshops with on-the-spot regulatory advice to try to elicit more orphan drug applications.  Over the course of the 2-day workshop, each company met 4 times with FDA staffers who offered advice on 9 critical issues in filling out an application.  Orphan drug applications are 6-7 pages.  At the conclusion of the first workshop, only 14 of the 29 companies submitted applications, although they can still submit at any time.  It usually takes the FDA 60 days to determine whether the designation will be given.  Up to 50 more organizations can attend a second workshop to be held at the University of Minnesota in August 2010.  Dr. Coté said he is considering a workshop in Europe.  Next time, he wants only applicants who can file, without the delay of company approval, at the end of the 2 days to attend.

Source: Wall Street Journal (digital)

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In general, drugs often work in only half of the people who take them.  In addition, billions are spent to treat adverse drug reactions and other complications.  The personalized medicine approach is to elucidate the differences between patients who respond to a drug and those who don’t, and to treat only those who will benefit, thus enhancing drug safety.  Fear of lost market share has been given as 1 reason for the slow acceptance of this approach.  Pharmacy benefit managers (PBMs; e.g., Medco and CVS Caremark) may be tweaking the business model.  PBMs anticipate making money by selling personalized medical services to employers, who are willing to pay them higher fees for improved health outcomes and lower prescription costs.   Both employers and PBMs expect genetic testing will increase the percentage of patients using cheaper generic drugs, thus increasing profits.

The business case for the personalized medicine approach is expected to strengthen as more genes and drug responses are linked.  Warfarin and Plavix (blood thinners) and tamoxifen (a breast cancer drug) were suggested as current candidates for this approach.  Additionally, results from any given genetic test are generally applicable to many drugs.  For example, the same variation that determines the response to Plavix can help determine how the anti-anxiety drug Valium, heartburn drugs like Nexium, and the antidepressant Celexa should be used.

Although “the blockbuster mentality is still in place, drugmakers are coming around” asserts Dr. Bruce Littman of Translational Medicine Associates.  Furthermore, regulatory agencies may refuse to approve and payers may decline to reimburse future drugs that can’t be directed to the right patients.  According to Richard Schatzberg of Generation Health, drugmakers predict a future business model where they can secure all of the smaller market of appropriate patients.  In theory, this approach should reduce adverse events and help to differentiate drugs based primarily on efficacy.

Source:  Business Week

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Personalized Medicine – why the sudden popularity?

Posted by cdavenport on Thursday Nov 12, 2009 Under Blog, Personalized medicine

The dream of tailor-made medication is not a new one, but the prospective cost of such treatments has always been a barrier to growth.  Simply put, the return on compounds that only treat small populations cannot justify the investment.  With life expectancy increasing and the cost of health care rising, however, an economic imperative is driving the change from managing sickness to managing wellness.   A wellness-management approach detects conditions early, and hence the level of reversibility is high;  the current reactive model has lower reversibility.  With drugs targeted toward specific populations, the importance  and acceptance of in silico modeling would increase.  The author presumes that drug development programs will run more quickly, and that regulatory bodies would be able to approve drugs much earlier once a response in the target population has been proven.  This is anticipated to reduce the clinical development time line and lead to cheaper drugs and prolonged exclusivity.

With the trend towards a lower acceptance of risk and thus bigger and more expensive clinical trials, do you agree with the author’s assumptions, especially given a smaller target population?

Source:  Drug Discovery and Development

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