The biopharmaceutical industry is pretty well known for some…creative naming skills and vocabulary, and the term ‘orphan diseases’ definitely fits in there. Despite how it might sound, it’s obviously not referring to diseases contracted by or found primarily in orphans; the American Heritage Medical Dictionary defines an orphan disease as “a disease that is so rare that it is not considered commercially viable to develop drugs to treat it.”

Lately, however, companies seem to be redefining their considerations of what is and isn’t ‘commercially viable,’ as both orphan drug collaborations and the market as a whole are on the rise.

GBI Research released a new report this month on orphan disease therapeutics in genetic disorders, covering major orphan genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, Fabry disease and Pompe disease. The report estimates that the global orphan disease therapeutics in genetic disorders market had a price tag of $1.5 billion in 2010, and is expected to grow at a CAGR of 13.8 percent to reach $6.1 billion by 2018.

GBI’s report notes that the “high rate of growth in the historic period” can be attributed to the entry of novel, high-priced therapies such as Myozyme for Pompe disease and enzyme replacement therapies for Fabry disease (which clock in with costs of over $200,000 annually per patient). That growth can be expected to continue with the approval of Kalydeco for cystic fibrosis and new collaborations and developments for Duchenne muscular dystrophy. “Competitive activity is also increasing as larger companies realize the rewards of investing in orphan therapeutics, and the high unmet need and low competition in many markets is expected to attract more into the space in the future,” the report adds.

More and more companies are establishing collaborations in the field of orphan diseases, drawn by the opportunities they represent; even though diseases with the ‘orphan’ designation usually affect only single-digit percentages of the population, those numbers still add up quickly, resulting in high unmet need and nearly open markets. Synageva BioPharma Corp. and Mitsubishi Tanabe Pharma, for example, began a collaboration in October for an undisclosed orphan disease, and Biogen Idec and Isis Pharmaceuticals Inc. initiated an option and collaboration agreement early this year to develop an investigational antisense drug for the treatment of spinal muscular atrophy. Most recently, ProMetic Life Sciences Inc. and Hematech Biotherapeutics Inc. announced earlier this week the signing of definitive agreements to co-develop and exclusively co-commercialize a plasma-derived orphan drug, a deal with a $10 million price tag.

And drugs with indications in that field are getting some regulatory help as well, as orphan drug designations often allow drugs to wade through the approval process more quickly if they’re designated for orphan diseases with high unmet needs.

It’s not the only attention orphan disease are getting, either, as a conference is being held this June focused on “Orphan Diseases and the Future of BioPharma.” The conference, the 2012 Orphan Disease Forum, is part of the 2012 BIO International Convention in Boston. Co-organized by the National Organization for Rare Disorders (NORD) and Centric Health Resources, and hosted by BIO, the forum will cover trends and developments in policy, technology, biotech and pharmaceuticals with relation to orphan diseases.

The forum will look at and discuss novel pathway approvals for orphan drugs, new developments in supply chain economics for the orphan markets and whether the pharmaceutical/biotechnology market is moving toward “orphan and narrow” indications as a whole. Alnylam Pharmaceuticals, Genzyme and Millennium: The Takeda Oncology Company will be co-sponsoring the event.

Despite the progress, there are still likely to be diseases that will have to wait to see any attention, let alone benefits, but it’s encouraging that some companies are stepping out into new disease fields regardless of the potential market size.

Generics play a big role in the industry today, both a catalyst and benefactor of change as the pharmaceutical market grows and adjusts to economic pressures. As companies and consumers look to drive down costs, the use of generics has increased, with companies looking to grab a piece of the pie by slipping their versions of blockbuster drugs into the available spaces left after patents expire. With lower prices than those of brand-name drugs, generics generally enjoy good levels of sales when they hit the market, but these days even they are feeling the squeeze as consumers look for even lower prices.

A new report from Espicom, The World Generic Market Report, notes that the global markets are going to continue to see growth in generic drugs, though distribution will vary. The United States’ generics market is expected to grow to $104.1 billion by 2016 as more blockbuster drugs lose their patents and pressure increases for generics to play a bigger role in Medicare drug plans. The biosimilar trend will also have an effect, as more companies look to biosimilars as an option to get drugs to market sooner than beginning compounds “from scratch,” as it were. (For its part, the biosimilar market is expected to grow to $3.7 billion by 2015, according to Datamonitor.)

Efforts regarding generics are a global trend, with governments in countries such as France and Japan taking steps to promote and reward increased use of generics. Other countries, such as Germany and the United Kingdom, are facing difficulties due to price cuts and the continuing economic issues (both globally and in Europe in particular), even though the United Kingdom represents one of the most competitive generic markets worldwide.

The FDA has an entire section dedicated to generic drugs on its website, and notes that these days, “nearly eight in 10 prescriptions filled in the United States are for generic drugs,” with the expectation that generics are going to see increased use as more drugs come off patent through 2015. And there’s good cause for that popularity, as the site notes that “on average, the cost of a generic drug is 80 to 85 percent lower than the brand name product,” and it’s estimated that the use of generics saved $158 billion in 2010 alone. A study of Medicaid drug spending in 2000 revealed that there could have been $229 million worth of potential savings if there had been greater use of generic drugs, and “if the best available prices from each state had been used nationally, savings would have increased to $450 million.”

Companies facing that generic pressure aren’t the only ones that are hyperaware of the market, as seen in the increasing number of acquisitions of generics-focused companies of late. On April 25, Watson Pharmaceuticals announced that it would be acquiring Actavis Group for approximately EUR4.25 billion (roughly $5.56 billion). The deal follows Watson’s acquisitions of Specifar and Ascent Pharmahealth, and will make Watson the third-largest global generics company; it’s expected that the company will see 2012 pro forma revenue of about $8 billion. In another large acquisition, Teva snapped up Cephalon in 2011, a move that bolstered its generics presence as well. Novartis acquired Fougera Pharmaceuticals just this week in a move that will make Sandoz, its generic pharmaceuticals division, the number-one global company in generic dermatology medicines. Acquiring generics companies allows firms to claim parts of the markets left empty when competitors’ drugs lose patent protection, and allows them to see revenues both from branded and generic products.

And some are working to improve on the generics idea even further, with supergenerics seeing increased interest and presence. Supergenerics, as noted in a new report from GBI Research, are small molecule drugs “which offer a therapeutic advantage of differ from me-too generic products.” Generic drugs are close twins of branded products, with few and subtle differences, but supergenerics represent new therapeutic entities. They, like generics, benefit from preexisting and late-phase clinical data, allowing for less complex development processes and shorter timeframes, but supergenerics offer improved delivery or design over standard generics. According to the report, this is another trend that can be expected to continue.

Like everything else related to the healthcare/pharmaceutical industry, it’s a give and take with generics. They offer opportunities for significant savings (and possibly revenues) compared to branded drugs, but as customers get used to generics prices, they start to call for lower ones. Generics allow new companies to step into the market as patents expire, but also open up issues related to pay-for-delay deals and expiration waiting periods. It’ll be interesting to see how branded products will continue to compete with generics and what will come of the recent insistence on increased generics usage in programs such as Medicaid, as consumer thriftiness and company revenue concerns continue to play tug-of-war with the market.

Few mental disorders get as much time in the entertainment limelight as obsessive compulsive disorder (OCD)—think “As Good As It Gets” and “Monk”—but it seems to be a give and take situation, because according to a new report from GlobalData, that’s pretty much the only attention the disorder is getting lately—and is likely to get for a while.

Public awareness of the disorder is on the rise, the report says, but candidates in the industry pipeline for the disorder are few and far between. According to GlobalData’s research, the presence of established, accepted treatment approaches—regardless of their indifferent success rates and high relapse rates—might be the reason pharmaceutical companies aren’t displaying much interest in pursuing OCD treatments, despite the market potential and significant unmet need. (The OCD therapeutics market grew at a low CAGR of 1.1 percent from 2006 to 2011 (largely due to Zoloft and Paxil losing their patent exclusivity), reaching $317 million, but that is expected to pick up; GlobalData reports that the market is expected to grow at a CAGR of 4.8 percent and reach $461 million by 2019.) Still, despite the unmet need and the numbers—the NIMH estimates the prevalence of OCD at 1 to 2 percent of the adult population in the United States, and the disorder is appearing in more children each year—there aren’t a lot of options, and according to GlobalData, that’s not going to change any time soon.

For the most part, the report notes, antidepressants are the current standard for treating OCD, a trend that is expected to hold steady for the next seven to eight years. Selective serotonin reuptake inhibitors (SSRI), most commonly known as antidepressants, are used as the first line of treatment, but high relapse rates tend to occur in OCD after patients discontinue treatment. Another option, often used in concert with SSRIs, is cognitive behavioral therapy (CBT), in which patients are helped to confront their fixations both through talking about them and exposure to them—patients are encouraged to pick things up that fell on the floor, if they’re afraid of germs, or to break a repetition-based habit. However, this is usually an extremely slow and, for those with OCD, often agonizing process.

The report notes that most OCD-focused trials currently underway are small, university- or institute-sponsored undertakings looking at finding a secondary line of therapy for those who don’t respond to SSRIs. Only nine molecules are currently in development.

The University of South Florida has a few studies in place, one “to examine if d-cycloserine enhances cognitive-behavioral therapy for pediatric OCD,” another examining neurocognitive factors that could predict how well someone will do in CBT and one to see if Palperidone is effective in treating OCD in patients who aren’t responding to SRIs.

Yale University completed a study recently that looked at the potential of ketamine infusions as a treatment for OCD, and has another underway studying “Riluzole Augmentation in Treatment-refractory Obsessive-compulsive Disorder.”

Specialty pharmaceutical company Transcept Pharmaceuticals, Inc. represents one of the few companies with a potential OCD treatment in the pipeline. The company is working on a drug candidate—TO-2061, a low dose of ondansetron—as an adjunctive therapy for patients who haven’t responded to first-line therapy. The drug candidate is a serotonin subtype 3 receptor antagonist that has previously been used as a treatment for nausea and vomiting as a result of chemotherapy, radiation therapy and/or surgery, and is currently in a phase II proof-of-concept study.

It’s likely that the lack of attention OCD is getting is due to public opinion and how society sees the disorder. There’s a general bias, just like with lung cancer: in the same way people generally consider that those with lung cancer “brought it on themselves” by smoking (which is not always the case), OCD is generally portrayed as comic gimmick on TV or in movies. The typical OCD sufferer is that person who has to wash their hands three times every time, or quadruple-check all the locks, or has to straighten the silverware at a restaurant—odd but harmless.

People with OCD are often considered to be weird (or comic relief) rather than stricken with something serious, but few people realize how crippling bad cases of OCD can be. For those with the worst cases, it is quite literally impossible for them not to engage in their obsessive behaviors, because the condition leads to an overwhelming, visceral fear that something terrible will happen if the behaviors aren’t carried out. It can make it impossible for people to leave their homes, let alone interact with others.

Public opinion is obviously not the only reason OCD is hurting for attention, since a large part of it is also due to pharmaceutical companies’ focus on markets that, due to their larger incidence rates (such as conditions like kidney disease or obesity), offer the potential for greater returns. Regardless, something needs to give. If a company can get an effective new treatment into the market, they’d have significant unmet need and little to no competition; hopefully that vacancy will appeal to someone soon, and OCD will finally get some attention other than on TV.