Following the news that the National Institute for Health and Clinical Excellence has accepted a proposal for a new type of scheme to provide patients with access to Millennium Pharmaceutical and Janssen Cilag's previously rejected cancer drug Velcade, industry observers have been analysing its implications.
Earlier this year, Velcade (bortezomib) was rejected by NICE for use in patients with myeloma on grounds of cost, but a subsequent appeal by cancer charities Myeloma UK, Cancerbackup Leukaemia CARE agreed that the appraisal committee should take another look at its use on the National Health Service.
Now, under the proposed risk-sharing scheme, the NHS will fund treatment with Velcade as long as patients show a "full or partial response" to the drug but, if there is no response, the cost will be refunded by the product's manufacturer.
Commenting on the proposal, a spokeswoman for Myeloma UK told PharmaTimes that the group is delighted patients are closer to getting access to Velcade. "We are still looking at the finer details of the agreement, including the fact that patients could be taken off the drug after just four treatment cycles...but the overriding sense is that it is a positive step forward, no matter what the caveats are."
And a spokesman for the Association of the British Pharmaceutical Industry told PharmaTimes that the agency "supports the selective use of such agreements when there is no other way to get patients access to medicines." But, "given that the drugs bill is a stable part of the NHS budget and only one pence in every £1 spent is invested in cancer therapies," he says that the Health Technology Assessment should be more flexible especially with regard to funding expensive cancer medicines targeting small patient populations.
NICE has come under increasing scrutiny in recent times - culminating in a current investigation by the Office of Fair Trading into the way the Institute operates - and its decision on which medicines to reject or include on the NHS treatment menu are often fiercely criticised.
On the one hand, there are those that question the use of increasingly expensive medicines on a cash-strapped NHS, but others argue that patients have a right to access the latest advances available under the health system.This latest move by NICE will likely spark some heated debate and, if successful, may even signal a seismic shift in the way medicines are funded in the UK.
According to Ernst & Young analyst Andrew Jones: "The agreement offers the prospect of a win-win situation for patients, the NHS and industry: patients get access to a new medicine; the NHS ensures efficiency of expenditure and the manufacturer secures an opportunity to get a return on its investment."He added that "the news should come as a welcome development to the biopharmaceutical industry which is being hit hard by pricing and reimbursement pressure across Europe.
NICE's endorsement of the scheme signals willingness for flexibility, and if agreed and successfully implemented, the scheme could help guide future change to the UK system and support market access for other new therapeutics".
Addressing affordabilityBut he also warned that risk-sharing schemes "do little to address the issue of affordability - the NHS will still have to find the budget to meet the cost of new and effective drugs...the issue of affordability raises the question of how the healthcare systems such as the NHS will be able to support the introduction of new and important drugs in the future without compromising in other areas."
And management consultancy Hay Group has also voiced its concern. Stephen Welch, Head of Pharma Sector at the company, says that, at £25,000 per patient, Velcade is expensive, regardless of its clinical efficacy. "Could the money-back guarantee distort doctors' views on which drugs to prescribe for their patients? And what of patient pressure?" Furthermore, he says, "In offering these sorts of deals the pharmaceutical companies are effectively putting greater pressure on NICE and prescribers by undermining the health-economic argument against using high-cost drugs for lower-volume conditions with limited efficacy."
He also points out that the time invested by PCT managers in calculating what refund, if any, they might be due, will only increase if more companies follow suit in offering such deals. "All in all, the principle of partnership will likely make for better, more cost-effective drugs, while increasing accessibility of those on the borderline. However, until the organisational and process implications have been thought through in detail, the most likely clinical outcome is confusion," Mr Welch concludes.
By Selina McKee
Source: PharmaTimes
2 comments:
Innovation in Pharmacology in the form of Biopharmaceuticals
Beginning in the 1970, biopharmaceuticals were being researched for conceptual production in those places once called academic institutions, and conducted basic research to idendify new product candidates applying a great amount of research. The same protocol is applied with biopharmaceutical companies today.
The first biopharmaceutical ever was synthetic insulin called Humulin made by Genetech in 1982 utilizing what is called rDNA technology, which also is used to produce human growth hormones. Later the rights were sold to Eli Lilly for this insulin. Biopharmas are distant and covert relatives of big pharma, yet become intimate with them more often now than ever before due to dry pipelines of big pharma- GSK and Roche in particular, yet most big pharma examine acquisition of biopharma companies for assured profit more than any other reason, they may speculate. Big pharma has a clear need to diversify which has created alignments with biopharmaceutical companies to create a pharmatech of sorts with the resources from both big pharma and biopharmaceutical companies. Furthermore, big pharma likes the fact that there is no design to produce biogenerics along with another fact that biopharmaceutical companies historically are fast growing companies of a different ethnicity of their own.
Over 20 biopharma drugs were approved in 2005, I believe. They grew almost 3 times as much as big pharma recently. Last year, biopharmaceutical companies made close to 80 billion in sales as well. Over 20 biopharmaceutical products are blockbusters by definition. They are overall very effective treatments for very difficult diseases to manage, as they target specific aspects of certain diseases, which limits side effects experienced by the patient placed on therapy with a biopharma drug. Unlike traditional drug development, biopharmaceutical companies first seek disease targets by genetic analysis and then search for a way to manipulate this target in a very specific way. The biopharmaceutical companies typically have a high profit margin and their products. Also, these products are biologically synthesized and do not cross into the bloodstream.
There are about a dozen f different classes or mechanisms of action of biopharmaceuticals that have about a half of dozen different types of uses that is always increasing, as lablel alterations of biopharmas are requested soon after the approval of a particular medication by this method that is allowed for further exploration of potential additional uses of thier new drug which, like other biopharmaceuticals, work in novel ways. Some cause apoptosis, or cell death of specific tumor cells. Some cause angiogenesis to occur, which means they cut off the blood supply to tumors. Then some biopharmaceuticals have multiple modes of action that benefit certain patient types and their diseases greatly, as with most biopharma products, the safety and efficacy is evident and reinforced with clinical data and eventual experience with the biopharmaceutical that is chosen to be utilized. And this clinical data is of a different method as well. Patients in the clinical trial are profiled, which allows better interpretation of this clinical data on their products.
Some biopharmas are more noteworthy than others, such as Enbrel, which was originally created for the many forms of RA- arthritis caused by the patient’s immune system attacking their bodies. At one point, demand exceeded supply for this drug, as the efficacy and safety was evident and demand was unexpected by the manufacturer, so Enbrel was sought out by doctors and patients both due to the clinical data verifying the efficacy and benefits of the drug. Relief from the pain and decreased quality of life was the primary motivation for patients seeking this particular drug. Enbrel was approved in 1998 and produced from what are called monoclonal antibiodies, which is one of several ways in which biopharmaceuticals are produced. In fact, some call the 1990s the biopharmaceutical decade.
Partnering of biopharmaceutical companies and larger pharmaceutical companies began during this decade in large part. Or a big pharma company will acaquire a biopharmaceutical company for complete ownership and bypass what would otherwise be a symbiotic relationship in that big pharma can provide the resources and connections necessary to launch and grow a particular biopharmaceutical drug, with the return on this investment being speculated revenue generated from what should be eventually a blockbuster biopharmaceutical.
The country of Belgium provides the most biotech products to the biopharmaceutical companies in the United States, which leads the industry in the world, with more than 70 percent of both revenues and research and development expenditures. Canada is ranked number two in this area, I’ve been told.
Some biopharmaceutical drugs are more profitable than others as well. Biopharmas compose around 10 percent of the pharmaceutical market presently, I understand. And with the government health care programs being the largest U.S. payers for pharmaceuticals, Medicare pays 80 percent of the cost of biopharmaceuticals. One other controversial, yet profitable biopharma class is known as EPOs, with names like Procrit and Epogen, and are indicated for anemia that is experienced in patients on dialysis or who have cancer in particular. Doctors are monetarily incentivized to exceed dosing requirements of these agents for their anemic patients, but this excess causes premature deaths and accelerated cancer progression for the patients that are over-dosed in this way. Once this tactic was exposed, there are now limitations regarding the amounts authorized to be given to particular patients placed on these EPOs. They are in the class of hormone biopharmaceutical drugs, which is another type of several classes of biopharmaceuticals, and they reduce the need for blood transfusions as they increase RBC proliferation.
While biopharmaceuticals are very efficacious and safe, as well as having therapeutic and diagnostic benefits for the very sick, the cost of them is outrageous, some have said. Genzyme has one biopharma product that costs a half a billion dollars a year. Yet most biopharmaceuticals cost around 100 grand a year for therapy that provides with some only limited life extension or survival rate of these patents that is only a few months. Furthermore, with cancer drugs, they are used together with chemotherapy for their treatment regimens, so there is no real improvement in the quality of life of some patients on biopharmaceuticals, considering the devastating side effects of chemo treatment. In addition, Co-pays financially drain such patients and their families, yet there is no other choice for therapy because of the avoidance in the creation of generic biopharmaceuticals, for example. Oncology, by the way, is the primary commercial focus of biopharmaceutical companies now and in the immediate future, so maybe competition will be a cost advantage to such patients in time.
Several years ago, I saw Roy Vagelos, former CEO of Merck, speak to others at Washington University in St. Louis. And during his presentation, he stated something similar regarding the cost of biopharmaceuticals and asked as well about whether or not the value related to the cost of biopharmaceticals appropriate for such a brief life extension of cancer patients in particular, for the most part. An issue or issues are always associated with new paradigms and innovations. Yet there are only a few biopharmaceuticals out of many available with debatable benefits with the high price tag. It ends up being what the market will bear. Yet the real question is the clinical evidence behind biopharmaceuticals: If a biopharmaceutical stops tumor progression without remarkable survival benefits or improvement in quality of life, is the price tag worth it to the patient? Assuring that a logical construct is examined to conclude that such a drug will be of benefit to a very sick patient will or should be the determining factor on the treatment regimen selected for such a patient.
How do these drugs differ from typical drugs that have been made before this advent of biopharmaceuticals? Unlike the small molecule, synthetic, carbon based pharmaceuticals of yesterday, biopharmaceuticals, classified under what is called Red biotechnology due to this being a medical process in the biotechnology world, essentially are larger and very complex modified proteins derived from living biological materials that vary depending on what medication will be manufactured and for what disease state. In fact, it is difficult to identify the clinically active component of a biopharma drug, which is why there is no pathway for generic copies of such drugs, as it would require expensive and meticulous clinical trial processes. Yet recently, a company called Insmed demonstrated bioequivalence to Amgen’s Nupogen that increases whitle blood cells. While there still is no defined pathway for follow-on biologics, this study demonstrated that another biologic drug can show that it is therapeutically equivalent. Insmed’s drug in this study will not be available for marketing until next year or later, though.
Also, a transformed host cell is developed to synthesize this protein that is altered and then inserted into a selected cell line. The master cell banks, like fingerprints, are each unique and cannot be accurately duplicated, which is why there are no generic biopharmas, yet biosimilar drugs are again a possibility, yet it’s in more of a conceptual phase presently, so it seems. So after this insertion, the molecules are then cultured to produce the desired protein for the eventual biopharmaceutical product. These proteins are very complex and are manufactured from living organisms and material chosen for whatever biopharma may be desired to be created. It is difficult to identify the clinically active component of biopharma drugs. So manufacturing biopharmas is a difficult process, and a small manufacturing change could and has raised safety issues of a particular biopharma in development that manufacturing had a manufacturing flaw of some sort. It takes about 5 years to manufacture a biopharmaceutical. And each class has a different method of production and alteration of life forms to create what the company intends to develop. Yet overall, their development methods are rather effective.
Another difference is that biopharmas are regulated under what is called the Public Health Service Act, and unlike the FDA, they authorize the marketing of biopharmaceuticals. The Act’s role in monitoring the promotion of biopharmaceuticals is unknown. Safety protocols regarding biopharmaceuticals are a mystery to me as well. What is known is that biopharmaceuticals have the potential to discover therapies to treat the cause of a particular disease state instead of treating it symptomatically. They set out to solve unmet clinical needs by science that has yet to be proven. Biopharmaceutcals save, enhance, and extend the quality of life of patients with terrible diseases, and over 250 million people have benefited from their products.
Presently, few biopharmaceutical companies are actually profitable. Also, with biopharmaceuticals, some years are better than others from a revenue point of view. Yet like any business, some years are better than others, and biopharmaceuticals are anticipated to offer quite a bit to public health in the future, with a focus on cancer patients in particular.
The cost of developing a biopharmaceutical exceeds a billion dollars, with about a third actually making it to market. The market size of biopharmaceuticals is rapidly approaching 100 billion dollars a year, with average annual growth between 10 and 20 percent. Between70and 80 percent of cancer drugs are prescribed off-label, so it will be interesting on how these drugs will be used in such disease states now and in the future.
Regardless of the challenges that are and will be faced by biopharmaceuticals, I’m pleased to see the results and realization of true innovation in pharmacology by taking a different path of drug development. Furthermore, I believe others should behave in a similar manner and be inspired by the biopharmaceutical companies and what they have done and continue to do for the benefit of the patients.
“The progressive development of man is vitally dependent on invention.” --- N. Tesla
Dan Abshear (what has been written is based upon information and belief)
I'm not very knowledgeable about what is going on in the pharmaceutical world, however my mother suffers from multiple myeloma and is now on Velcade as it is her last avenue. I wish we were in the UK, we're in the U.S. and she pays out of pocket and with the aid of generous donations $500 a week for her treatment. It infuriates me that we live a world where you literally have to pay just to attempt to prolong your life when you are already plagued with an uncanny,proactive, deleterious disease such as Multiple Myeloma aka the Laced Disease.
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