biologics

Barriers to Biologic Treatment - Is Injecting Such a Pain?

Mary Assimakopoulos — Thu, 10 Oct 2013 14:46:01 +0000

Précis:

Mary Assimakopoulos examines a common yet often unaddressed patient concern – the very human fear of needles!

Insights from our market research into autoimmune diseases clearly demonstrate that patients who are treated with a biologic are more satisfied with their treatment, report significant improvements in their symptoms, are more likely to be able to live a “normal” life and have more positive feelings about coping with their disease. Biologic treatments halt the progression of the illness, rather than just deal with the symptoms.

Time to switch?

Despite this, it often takes a long time for a doctor to discuss biologic treatment options with their patient, during which time their symptoms may have been getting progressively worse. Factor in the time to presentation (often more than a year) and time to diagnose (another 18 months or more) and our findings reveal that RA patients, for example, are looking at around 7 years before they are switched to a biologic. In a previous column, I talked about the RA patient journey and the fact that only around half of patients reported having discussed biologic treatment options with their doctor. Similarly, around half of patients with psoriatic arthritis report not being given a full understanding of treatment options and 62% said they had not discussed the possibility of a biologic treatment. Over half (59%) of patients with AS and SpA have not discussed this option either.

What are the barriers?

So what is preventing some patients being treated with biologics? There are a number of factors we can identify, not least amongst them the cost of this form of treatment. Some patients are not eligible, perhaps because their symptoms aren’t yet severe enough. Some are responding well with their current DMARD treatment. For some who are too frail it wouldn’t be a sensible option. However, where the choice for treatment is in the hands of the patient, there are a proportion who have taken the decision themselves not to switch.

...of these patients around one third to one half state that this is because they dislike the idea of injection.

Biologics are strong drugs which come with side effects and contraindications. They need to be given by IV or injection, something which is scary for some patients, particularly the idea of administering the injection themselves. In our ‘Living With’ surveys, 15% of RA patients, 10% patients of SpA patients and 11% of PsA patients have refused a biologic treatment offered to them by their doctor and of these patients around one third to one half state that this is because they dislike the idea of injection. Online RA forums are full of people saying they just can’t inject themselves as they are too frightened and their partner can’t do it for them either. This means regular trips to the hospital, which means a higher risk of non-adherence, which is not good for long-term outcomes.

Fear of injection

The findings from our research reveal that despite this, just under half of patients who aren’t on a biologic are willing to accept an injection-based treatment, most likely because there are no other better options. Two thirds of patients from our RA study said if they were offered a pill which gave them the same efficacy as a biologic, they would prefer this. But for today, many patients say they are willing to accept injection as a mode of administration because they want greater health benefits. One patient said “I would be willing to try almost anything to relieve my condition and I trust my doctor's opinion and advice”.

Communication and Support

Patients who are on a biologic seem to cope well with injections overall. In our study, patients across all disease areas report feeling ‘comfortable’ and ‘confident’ around their injection. We found that those who can self-inject found the training they were given to be a positive experience. Many also reported being very happy with the support they were being given by their healthcare professional during the process.

So could the patients who are refusing a better treatment be persuaded with the help and support of their doctor?

An analysis of our patient and physician data seems to suggest so. We sought to understand if there is a causal link between the type of relationship an RA patient has with their Rheumatologist and their willingness to take their biologic treatment. We also sought to understand if there are “typical” demographic elements to an RA patient who is more likely to actively seek ownership of their biologic treatment.

We found that RA patients who felt their treatment decision was a result of “joint decision-making” between themselves and their rheumatologist felt more positively about their RA disease and the impact the treatment was having on their lives. They were also less likely to skip doses, whereas patients who felt they were allowed full control over the decision, were more likely to skip doses. It seems that a collaborative approach, where both the doctor and RA patient make the decision to start a biologic treatment, results in a more positive outcome for the patient.

So the barriers to being given a biologic treatment can be overcome with the right education, communication and support from the healthcare network.

It will be interesting to see what effect the arrival of new oral drugs and biosimilars will have on the treatment landscape for autoimmune in the not-too-distant future. The race is on among manufacturers to come up with the new gold standard improved delivery system for injectables – spanning autoimmune, diabetes, MS, growth hormones etc. A new solution will revolutionise the market. Who is going to claim this ‘holy grail’?’

Questions or comments? You can share you thoughts with our audience in the comments section below, alternatively you can email the author directly at marya@researchpartnership.com.

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EMA Releases Final Guidance on Biosimilars, Seeks Comment

Ben Steele — Mon, 17 Jun 2013 09:41:50 +0000

Précis:

The EMA has released the last of a series of three proposed guidelines on biosimilar medicines for public six-month consultation.

The latest document sets out the EMA’s current position regarding both clinical and non-clinical development requirements for biosimilar copies of medicines which have lost patent protection, providing an update to earlier guidance released in 2006.

Since 2006, the EMA says that “14 biosimilar medicines have received marketing authorisation in the EU”; the agency also notes that the number of applications being processed by its Committee for Medicinal Products for Human Use (CHMP) has risen substantially in this time.

The efforts towards defining regulatory pathways for biosimilars have been necessitated by a change in the market, as many of the early generation of biologic drugs are coming off patent. Biosimilars demand different, more stringent assessment than generic versions of chemical medicines as biologic drugs cannot be exactly replicated; because biosimilars are not exact copies of the original biologic, regulators need to ensure safety and efficacy are not compromised. The EMA’s attempts to refine the process of approving biosimilar drugs will be seen as imperative for many governments struggling to afford the high prices of patented biologic medicines.

A report by market research firm Visiongain predicts that the market for biosimilars is set to grow rapidly in the next decade, with this year alone seeing a 20% gain from last year, taking the total value of the market up to $2.44 billion. Currently the leading segments in this market are monoclonal antibodies, insulins, interferons, erythropoietin, growth hormone and follicle stimulating hormone (FSH). The loss of patent protection for many biologics will be the main driver for growth in this area.

The latest guidelines to be released deal with factors emerging since 2006, including the move towards risk-based approaches for non-clinical studies, and the practice of using pharmacodynamic markers to demonstrate clinical comparability. It also covers issues surrounding patient selection and surrogate endpoints, extrapolating efficiency and safety from one therapeutic indication to another, and designing immunogenicity studies. The two other guidelines included a document on the general principles regarding the demonstration of biosimilarity, which was released last month for comment, and a document discussing quality issues for biosimilars published in May 2012.

The US is currently also working on the details of the regulatory pathway for biosimilars. The Biologics Price Competition and Innovation Act (BPCI) mandated the development of such a pathway, and recently the FDA released its fourth guidance document intended to set out what the regulatory body requires from the developers of biosimilars.

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New Research Holds Potential For Production Of Purer And Cheaper DNA Molecule Drug Versions

Lucy Brake — Wed, 05 Jun 2013 09:19:55 +0000

Précis:

A novel way to mass produce specific DNA molecules is believed to hold the key for many of the challenges that come with current production methods. This ultimately means that high quality DNA molecules can be manufactured in large quantities at a cost effective price.

A collaborative effort between researchers based at Karolinska Institute in Sweden and Harvard University has seen the development of an innovative technique to manufacture short, single-stranded DNA molecules, which are also known as oligonucleotides. These DNA molecules are an essential investigatory tool and are an important component in a wide range of pharmaceutical research projects.

Reported this week in the scientific publication Nature Methods, this new technique has the potential to be used for the development of drugs consisting of DNA fragments as well as in DNA nanotechnology.

Without the use of oligonucleotides, many of the recent progress made in genetic and molecular biological research and development would have been impossible, such as the ability to quickly scan an organism's genome.

Co-developer Björn Högberg, from the Swedish Medical Nanoscience Center explains: "We've used enzymatic production methods to create a system that not only improves the quality of the manufactured oligonucleotides but that also makes it possible to scale up production using bacteria in order to produce large amounts of DNA copies cheaply”.

During bioproduction DNA sequences are copied using bacteria. From this processes manufacturers can then produce vast numbers of DNA copies at a low cost. However, current processes for synthesising oligonucleotides result in the number of errors increasing with the length of the sequence. In contrast, the developers report that this new technique works well for long oligonucleotides of several hundred nitrogenous bases.

The paper published outlines that because the DNA molecules are formed using long strands folded back on itself which have been cut up by enzymes, and then several different oligonucleotides can be produced at the same time in a balanced combination. This is important if the DNA molecules are to be crystallised or used therapeutically.

Analyses of the formation of a DNA crystal and folding of DNA nanostructures by the researchers enabled them to confirm the scalability, purity and stoichiometry of the produced oligonucleotides. As a result of this work the scientists have opened up the possibilities to reduce the costs of creating higher quality DNA molecule-based pharmaceuticals: "Oligonucleotide-based drugs are already available, and it's very possible that our method could be used to produce purer and cheaper versions of these drugs", points out Högberg.

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Bio Boy! Developing Markets to See Surge in Biosimilar Drug Growth

Louise Kelly — Tue, 14 May 2013 13:27:44 +0000

Précis:

The global biosimilar drug market for 2013 will see a 20% growth on last year’s figures to a whopping $2.44 billion, and a new survey indicates that this trend will continue to flourish for the next decade.

Last year, the majority of global revenues for biosimilars came from emerging markets where such products were first launched over twenty years ago. However, according to a new report by Visiongain, growth of these ‘follow-on’ drugs in developed markets is set to surpass previous global standards over the next decade as patent expirations on many blockbuster biologics during 2013-17 are anticipated.

The global biosimilars market’s current leading segments are: monoclonal antibody biosimilars (mAbs); insulins; interferons; erythropoetins; filgrastim; somatropin; and follicle stimulating hormone (FSH). According to the report, the approval and release of similar products will drive impressive growth in the biosimilars space up to 2023, particularly in the US and the European Union (EU).

To date, two companies (CellTrion and Hospira) have already requested approval from the European Medicines Agency (EMA) for biosimilar antibody applications, with product launches across the EU expected in 2014. By which time - predicts the study - many companies worldwide will be attempting to tap into the trend, with a special focus on mAbs. Fast forward to 2023, insulin products and mAbs are expected to account for 57% of the global biosimilars market.

Many drug manufacturers, including the larger firms, such as Pfizer, Merck and Boehringer are being tempted by the biosimilars market as these drugs offer a simpler way to launch biopharmaceuticals compared with developing novel products from scratch, according to Richard Lang, a pharmaceutical market analyst for Visiongain. “Many of these companies are focusing on biosimilar monoclonal antibody development – those three companies have clinical-stage candidates in that sector.”

Complexities and unclear regulation

The positive outlook for growth does come with a warning, as the development of the large complex molecules of biologics, regardless of cloning the original product, can often translate to a long and costly development cycle. Unlike the more common small-molecule drugs, biologics can be quite sensitive to changes in manufacturing processes – and the companies attempting to create a biosimilar drug will not have access to the original engineering.

As a result, very slight variations from the blockbuster drug can have serious health implications for the intended market, a concern that has fed through to reluctant policy makers and given scope for drug manufacturers to contest a copy of their innovative product. For example, a lobby group of biotech drug companies in India has recently raised concerns about the approval of Cipla's first biosimilar drug Etanercept, used for the treatment of rheumatic disorders.

Despite the positive forecast, and in contrast to the long solidified guidelines for follow-on drugs by the EMA, launches of biosimilars in the US have faced some difficulty as the FDA has been slow to clarify certain queries around how the agency will evaluate these drugs. Released last year, the draft guidelines left numerous questions unanswered as to drug classes and definitions, safety standards, labelling etc. More concrete, albeit strict, standards were released by the FDA earlier this year and biopharma companies won’t want to be left behind in the race against the patent cliff clock.

According to the Generics and Biosimilars Initiative, patents for 12 blockbuster biologic compounds generating a combined $67 billion in sales will expire by 2020, so these opportunistic firms will surely be fulfilling the predictions of the new survey and bombarding the agency with applications.

Alas, the obstacles don’t end even when approval is achieved, according to Lang, “The recent release of development guidelines by regulatory agencies in markets worldwide will increase the success rate of biosimilar development. However, commercializing biosimilars is as challenging as developing them. Companies will need to brand and market their biosimilars in a similar way to novel drugs, engaging key stakeholders such as doctors, health care payers and patients, to achieve high market penetration.”

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Florida Joins Growing Number of States Passing Biosimilar Substitution Bills

Lucy Brake — Tue, 07 May 2013 13:03:10 +0000

Précis:

Last week, the state of Florida passed legislation allowing for the substitution of biologics with biosimilars alternatives, joining a growing list of states in America to do so.

The legislation in Florida sets out the requirements for pharmacists to substitute biologics when they are determined to be biosimilar and interchangeable with the brand name product. It also outlines the requirements for the state board of pharmacy to maintain a current list of interchangeable products.

Through this bill, biotech companies Amgen and Genentech sought to limit future access to biosimilars once they reach the market. However, they were unsuccessful and the law is due to take effect from 1 July 2013.

A number of states, including Virginia, Indiana and Utah have passed legislation to allow physicians to side-step pharmacists who would otherwise substitute a biosimilar drug, while requiring the pharmacist to notify the physician and patient when substitution had taken place. Some of the bills, such as that in Virginia, included a sunset provision where it would expire after five years.

Of the eleven states that have considered the measures to restrict biosimilar access, North Dakota is the only state that has passed a bill to restrict the substitution. Legislation currently is under consideration in California, Illinois, Massachusetts, Oregon, Pennsylvania and Texas.

The US Patient Protection and Affordable Care Act of 2010 included an amendment creating an abbreviated regulatory approval pathway for biosimilars, but the US Food and Drug Administration still has to create regulations for them before it can approve biosimilars under such a pathway.

From the generic drug industry’s perspective this case was an effort to limited access to biosimilars, which are generally believed to be much cheaper than brand named drugs, particularly the block buster pharmaceuticals. The concern from many biotechnology companies is that biosimilars may in fact be slightly different from the brand named drugs, and as such there are health and safety risks for the patient from substituting them.

The US trade group for the generic drug industry, the Generic Pharmaceutical Association (GPhA), applauded the passing of Florida’s bill, noting that the approved legislation facilitates future patient access to interchangeably biosimilars. According to the GPhA generic pharmaceuticals fill 80 per cent of the prescriptions dispensed in the US but consume just 27 per cent of the total drug spending.

"Florida spent more than $104 million on biologic medicines in 2011 alone for its state Medicaid program, a number that only will grow," said GPhA president and CEO Ralph Neas. "Once the FDA approves full guidance on biosimilar approval and interchangeability, we are confident that states like Florida will benefit enormously from these lower-cost, life-saving medicines.

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$13.6 Billion Buyout of Life Tech by Thermo Fisher Creates Personalized Medicine Heavyweight

Lucy Brake — Tue, 16 Apr 2013 13:26:01 +0000

Précis:

In one of the America’s biggest corporate takeovers this year, science service provider Thermo Fisher Scientific has brought out the scientific and laboratory equipment maker Life Technologies Corporation for US$13.6 billion.

There has been much discussion recently about the proposed buyout of Life Technologies by Thermo Fisher, which was finally confirmed yesterday by both companies.

Today Online reported that the acquisition would propel Thermo Fisher into the “hot field of genetic sequencing” where scientists, pharmaceutical manufacturers and physicians are exploring the genetic factors of diseases to allow improved treatment that can be specifically tailored to individual patients.

Life Technologies has been gaining a reputation as an expert in DNA-sequencing tests, a critical technology needed in the emerging field of genetic-based medicine. It is believed that this corporate takeover will establish Thermo Fisher as a major force in the emerging personalized medicine industry.

In a prepared press release, Thermo Fisher stated that the joining together of the companies will create an “unrivalled leader in serving research, specialty diagnostics and applied markets”. In particular, they noted that organizations with an interest in proteomics, genomics and cell biology would benefit from the strengthened technological expertise and offerings.

Marc N. Casper, president and chief executive officer of Thermo Fisher Scientific said “The acquisition of Life Technologies enhances all three elements of our growth strategy: technological innovation, a unique customer value proposition and expansion in emerging markets”.

“We’re especially excited about the new opportunities we will have to leverage our complementary offerings, fuelled by a shared commitment to continuous innovation”, he continued.

Life Technologies believes that the buyout will offer companies working in the highly competitive pharma sector significant benefits. Gregory T. Lucier, chairman and chief executive officer of Life Technologies, said, “This transaction brings together two companies intent on accelerating innovation for our customers and achieving greater success in a highly competitive global industry”.

Thermo Fisher has revenues of $13 billion, approximately 39,000 employees and serve customers within pharmaceutical and biotech companies, hospitals and clinical diagnostic labs, universities, research institutions and government agencies, as well as in environmental and process control industries.

Life Technologieshas revenues of $3.8 billion and around 10,000 employees. They provide an extensive range of scientific and laboratory instruments, including genetic analysis, molecular biology, bioproduction, genomic medicine and molecular diagnostics.

The deal still requires regulatory approval from US and foreign governments and is expected to be finalized early in 2014.

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Biosimilars: The new iPad or Sony Betamax?

Dr Andree Bates — Wed, 06 Mar 2013 14:35:37 +0000

Précis:

Globally, while the total sales of biological agents continue to grow, branded biologic manufacturers are facing increasing competition from biosimilars, and while some biosimilars are having runaway success, others entering the market are having a tough time succeeding.

IMS predict that biosimilars will be a market worth around US $1.9 – 2.6 billion by 2015. For some time now there has been speculation as to what the impact of biosimilars will be on the biologics that they are ‘similar’ to.

Will biosimilars become the next big thing, much like the iPad? Or will they wither and die on the vine like the infamous Sony Betamax during the format wars?

What are the factors for branded biologics facing biosimilar competitors, and biosimilars competing with originator biologics that result in success, or lack thereof? Let’s examine some of these.

Cost to Develop

Biosimilars are essentially a generic alternative to a biological – you could say they’re biogenerics.

A typical chemical small-molecule generic costs between $1-4 million to get to market, whereas to get a biosimilar to market can cost between $100-400 million as achieving similarity is a lot more complex in biologics than in small-molecule chemical entities. This means that companies that decide to step into the biosimilar space really need to ensure that that are going to get a decent return.

“Many generics companies are out of options so they need to consider this space to survive”

Of course, $100-400 million may not sound like much when compared with the biologic originator costs of between $1-12 billion but they are not small, and therefore, preclude some of the smaller players from entering the market alone. However, many generics companies are out of options so they need to consider this space to survive, and therefore are attempting to get into bed with some of the bigger players to ease their passage. The early birds in this market were largely generics companies (e.g. Sandoz, Stada, Hospira, Teva, etc), then there was the wave of collaborations as the costs started to add up (Merck and Hanwha, Pfizer and Biocon, Merck Serono and Dr Reddy, Samsung and Biogen Idec). Now, in 2013, we are seeing many collaborations breaking down and a reduced number of players, and some surprising new entries in this space, e.g. Fuji Film. This seems about right when you consider the marketplace is not as easy a walk in the park - as was expected; this is a larger barrier to entry than a normal generics marketplace is.

Price

On first blush one would think ‘cheaper to get to market, therefore cheaper price’…and this is somewhat true, although in reality the Biosimilars are not coming onto the market significantly cheaper than the biologics. In many cases the list price is not too different from the originator biologic price, so the only way the biosimilar can gain a price advantage is by discounting to institutions and gaining access via contracts. Overall, so far, the biosimilars come in at best around 10-30% cheaper than originators. If we look at generics in Japan that come in 30% cheaper than the chemical entity, the cost advantage is not enough to create sweeping changes, although they do slowly chip away. This is potentially the same case with some biosimilars if cost is the key issue being used to sell them. In the US, one of the payer issues will be level of co-pay as, unless they are seriously lower, the incentive to use will be less. However, even in emerging markets, their success based on this is not assured.

Experience

In some therapeutic areas there is already a degree of acknowledgement of similarity between originators and biosimilars, and in these areas it is likely that uptake will be faster. If the area has a limited number of treatment centers that can be targeted this makes it even more attractive for biosimilar uptake, and this is certainly seen in categories like G-CSF and EPOs. However, many therapy areas have not experienced ‘similarity’ and, for these, it is likely that the only reason physicians would switch is when patients fail on the originator treatment (e.g. in autoimmune diseases).

Physician Acceptance

Physician acceptance will be one of the key barriers for biosimilars. Of course, it will depend on their experience. In a survey done by Decision Resources in 2010, it was found that the acceptance varied by therapy area and unlike physicians in other specialties, Oncologists were the most likely to adopt biosimilars into existing treatment algorithms. They also reported they would be satisfied with less clinical trials and would be less likely to require head to head clinical trials with the originator biologic, immunogenicity data, and safety data, before prescribing. This suggests that Oncologists may turn to biosimilars before other types of physicians.

“Their success depends on convincing the physicians of the ‘similarity’ with the originator and providing added benefits”

However, how companies marketing biosimilars leverage this opportunity will depend on sales and marketing skills. When a biologic comes to market, physicians have reams of clinical data to look at in journals and at conferences before it even gets to market. They are prepared; they participate in advisory boards and key opinion leader meetings and so on. In addition, the key aspect pharmaceutical marketers promote on is utilizing clinical trials showing efficacy, manageable side effect profiles and safety. Biosimilars will not have the advantage of this.

So when a biosimilar comes to market, what do they have? Not a lot in terms of what physicians are used to, given there are no clinical trials per se for these to gain approval. Their success depends on convincing the physicians of the ‘similarity’ with the originator and providing added benefits (be it price or service bundles). In fact, it really comes down to sales and marketing - and that is something that many generics companies are not used to doing. This offers an edge to biologics who have strong analytics and can quickly see all the drivers and non-drivers of their markets (not just price), and what they need to do better. Biosimilar manufacturers may also start to do analytics and ramp up their sales and marketing skills as this will become the key differentiator of these products. If they do so, this will become a much fiercer battleground.

Patient Acceptance

Patients are increasingly involved in treatment decisions and in markets where there is a co-pay obstacle, this becomes even greater. If the physician has not been fully convinced of the efficacy, side effects and safety profiles of the biosimilar, it will be hard to convince a patient. Companies may investigate implementing patient assistance programs to assist and boost their markets. Interestingly, in many therapy areas, patients are involved in social media networking and discussions, and therapeutic areas involving biologics facing biosimilar competitors will probably be a fertile and productive ground for social media marketing activities.

How well biologics do to defend against biosimilars, and how well biosimilars do in each market, will depend on pricing and reimbursement, influence and perceptions of stakeholders as well as attitudes towards their adoption and use, and of course, their use of sales / marketing analytics and smooth execution of their strategy.

Conclusion

It seems clear to me that Apple is great at marketing. Their products are wonderful but also have an inherent ‘cool’ factor about them due to their marketing. Other companies make similar products but don’t get the same traction Apple do with theirs.

Sony unveiled the Betamax video recorder in the 70s, quickly followed by JVC’s VHS system. In terms of quality of performance, Sony’s Betamax was far superior to its rival. However the battle for hearts and minds is not always won by the superior force, but rather by the best pitch – or in this case the best marketing strategy.

The battle between biologics and Biosimilars won’t solely come down to price, but that as a marketing component along with a strong analytics-driven sales and marketing strategy and execution, will almost certainly win the day.

For information on analytics in the biologics and biosimilars space, please contact the author for more information at any of the Eularis offices.

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Forecasting the future of biologics

Peter Mansell — Thu, 15 Sep 2011 11:40:52 +0000

Précis:

Peter Mansell reports on how to balance complexity and predictability when forecasting for biologics

The growing presence of biologics in R&D pipelines and on the pharmaceutical market has brought with it issues of complexity and controversy around product composition, variability and reproducibility, safety, modes of administration, and costs to healthcare systems.

All of which suggests that forecasting for these therapies is a much trickier proposition than for small-molecule drugs.

Not necessarily so, says Florian Turk, executive director and head of Global Health Economics & Outcomes Research at Novartis Pharma AG.

Turk’s focus is on market access and health technology assessment scenarios that feed into estimates of product uptake.

The overarching challenge in forecasting MA potential is accuracy, particularly given the proliferation of customers and stakeholders acting according to their particular business rationale and budget, he points out.

The complexity of this process is “certainly increasing,” Turk comments.

But it is not automatically more of a problem for biologics than for small-molecule drugs.

For one thing, biologics tend to be better characterized than their small-molecule equivalents.

“Sometimes, forecasting for specialties is now easier for us than in primary care,” Turk says.

“As part of your business preparation, it is easier to see what the likely restrictions are and what the likely offerings are in terms of the patient populations to be targeted.”

Nor, he says, does the relative complexity of biologics necessarily muddy patient pathways, at least from a forecasting perspective.

A better-defined patient population will make these pathways easier to plot. (For more on biologics, see ‘Patent expiration: Innovate or die’ and ‘Dr. Bates Talkback: How to mount an effective defense against generics’.)

Moreover, primary care products sold at premium prices are at the same risk of factors that complicate the treatment regime, such as prior authorization criteria, protocols, or demand for extra data to underpin duration of therapy, Turk notes.

So the basic rules of engagement are already in place.

Tipping toward complexity

What may tip biologics toward complexity is treatment response, particularly when products are bundled with companion diagnostics and the treatment regime depends on response analyses that may thin out the available patient population. (For more on companion diagnostics, see ‘New challenges and techniques in forecasting’ and ‘Personalized medicine: Regulating companion diagnostics’.)

“First of all, you need a good forecast of response, of patient population, of genotype,” Turk explains.

“Secondly, companion diagnostics, and these different starting and stopping rules, are usually developed alongside the drug.”

So the uncertainty resides not so much in the nature of biologics as in the business models developed for them.

These may involve factors where “we don’t have many market analogues”, Turk notes.

Potential variables include how accurate a companion diagnostic may be; whether a suitable diagnostic will be available/accepted and, if so, in which markets; and the greater likelihood that biologics will be subject to risk-sharing or pay-for-performance schemes. (For more on risk sharing, see ‘Managing market uncertainty: Is risk sharing the answer?’ and ‘Market access: Risk sharing and alternative pricing schemes’.)

In general, and despite the ongoing debate around pricing, HTA conditions for biologics are not fundamentally different from those for small-molecule drugs, Turk says.

All the same, actual product uptake will depend on ‘real-life’ treatment effectiveness.

“When you do the forecast, you have some idea about the product’s efficacy and safety,” Turk comments. “But you don’t know what the effectiveness will be.”

Generic entry

With the regulatory framework for biosimilars starting to clarify, biologics forecasts also need to factor in the potential impact of generic entry down the line.

Turk is not convinced, though, that the competitive threat is any less measurable than for mainstream generics.

There may be more uncertainty from a technological viewpoint, depending on the methods of synthesis or production used, he points out.

But the same factors can lead to marked variability between biologics themselves.

“In the end, when you forecast you have the same assumptions: How many players do we expect to enter the market? How many of them do we expect to enter at risk?” (For more on generics, see ‘Forecasting for generic erosion rates’.)

Another consideration is whether biologics, particularly those sold at high prices, have a place in the emerging markets that will increasingly drive pharmaceutical industry revenue and profits.

Here also, Turk sees no black-and-white distinction between cutting-edge therapies and small-molecule drugs.

The superior targeting available with many biologics means “there is a much stronger case for a limited number of patients in a number of emerging growth markets,” Turk says.

It really depends on the stage of development. There are emerging markets that have evolved to the point where they can afford additional treatment solutions for small patient populations in real need.

Then there are less developed countries where even conventional branded drugs have only a very small market share.

In South Africa, for example, the issue is not so much the type of molecule as whether it is branded or generic, Turk observes.

For a mainstream drug with a large patient population or for a biologic with a more targeted population, “In the end, what matters for the market is overall budget impact,” he says.

Familiarity is also easing the challenges of forecasting for biologics.

With the first generation of products, such as the monoclonal antibody Remicade, it was “really unknown territory,” Turk comments.

Now that a number of analogies are available in different countries, there is a much better understanding of how the market functions.

Accordingly, much of the uncertainty has been removed from biologics forecasting, Turk believes.

Rather, the scales have tilted so that “a lot of uncertainty has been added to the conventional products as well.”

On this more level playing field, he adds, “It’s all about predicting the changes in the external environment.”

For all the latest on forecasting, join the sector’s other key players at the 5th Annual Forecasting Excellence conference on October 4-6 in Boston.

For an overview of eyeforpharma’s forecasting coverage, see ‘Highlights from eyeforpharma’s Forecasting coverage’.

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