Two Labs Blog

Two Labs Blog

Jessica Krauser

Director of Marketing who leverages Two Labs unique culture and exceptional pharma expertise to create client-centric marketing. To do this, Jessica believes you must have a clear understanding as to who the client is, what their needs and problems are, and engage them in a way that’s meaningful to them.
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Recent Posts

Two Labs Joins Global Celebration of Rare Disease Day

Posted by Jessica Krauser on February 28, 2019

DSC_0423Since 2008, Rare Disease Day has been on the last day of February – which every four years is a rare 29th of the month. The observance was created to drive awareness of the 6,000 existing rare diseases that affect 1 in every 20 of us.

As a way of demonstrating our support, we joined in on Rare Disease Day’s face-painting event at our offices, along with donations to Rare Disease Day and the National Organization for Rare Disorders to help further the amazing work.DSC_0416

Check out some photos from the event and join in to #ShowYourRare on social media! We’d also encourage you to check out some of the inspiring testimonials on Rare Disease Day’s website.

We're also supporting Mediaplant in the campaign for Rare Diseases by bringing awareness to the challenges manufacturers, regulators and payers face with orphan drug market access. 

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This year, the theme is “Bridging health and social care,” which is something that is extremely meaningful to us at Two Labs. In recent years, we’ve become increasingly involved in rare disease drug launches across the globe and we’ve seen how much support and assistance is needed by those suffering from rare diseases.  While the innovation and progress with rare diseases are very impressive, we can’t forget what our work is really about – improving people’s lives.

Topics: MKO, Pennside Partners, Rare Disease Day

Current and Future Oncology Management in the United States

Posted by Jessica Krauser on February 1, 2019

The cost of treating cancer patients is high and rising in the United States. Payers are exposed to cost through doctor visits, laboratory tests, imaging tests, radiation treatment, drugs, hospital stays, surgery, home care, transportation and travel, and caregiving. This study focuses on the cost of medication from the viewpoint of U.S. payers. Although new tools for managing these costs have been gaining attention, prices continue to rise, and challenges to managing costs remain high. Innovative tools are necessary for controlling the cost of care in oncology, but their effectiveness is still unclear.

Read full article published at the JMCP (Journal of Managed Care & Specialty Pharmacy).

Innovative Access Agreements

Posted by Jessica Krauser on January 1, 2019

A potential payer solution for Cell & Gene Therapies

A US and European perspective

Payer concerns over increasing cost of therapies and uncertainty of clinical data have led to a growing interest in market access tools. These tools have many names and definitions, often referred to as innovative (IAA) or alternative access agreements (AAA). For the purpose of this review we will refer to them collectively as IAA. The agreements can be split into two key categories: finance-based, designed to reduce uncertainties over budget impact and cost; and outcome-based, designed to reduce clinician uncertainties over drug performance.

Download White Paper

Failure to Launch: Five key reasons that biosimilars have failed to gain traction in the US market

Posted by Jessica Krauser on November 1, 2018

Although biosimilars have taken off in Europe, they have yet to gain significant traction in the United States. More than thirty biosimilars have been approved and twenty-six are marketed in Europe. In the US, there are only eleven biosimilars approved with only five marketed: Zarxio, Fulphila, Retacrit, Renflexis, and Inflectra.

Read the full report by downloading this paper.

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Two Labs Marketing is Changing Its Name

Posted by Jessica Krauser on September 5, 2017

Two Labs Pharma Services Name Change

Two Labs Marketing is Now Two Labs

Two Labs Marketing, LLC is announcing our name change to Two Labs, LLC, a pharma services consulting company. To some it might sound like a small change, but to those who have been around Two Labs Marketing know that for over 14 years we have evolved into much more and we want our name to reflect that. Our company was established in 2003 with the idea to educate and train pharma product managers about the commercialization process. 

Since then, the Two Labs team consistently created new servic

es based on client needs:

  • PharmaLicense, LLC in 2007
  • Compendia expansion in 2009
  • eRx Solutions, LLC in 2013
  • Post-Launch Trade Management Services in 2013
  • Specialty Commercialization expansion in 2014
  • Patient (HUB) Services in 2016

Our name, Two Labs, allows us to brand all our services and affiliates under one umbrella in a more integrated, meaningful way.  For starters, every employee (PharmaLicense and eRx Solutions team included) will have a Two Labs email address. The second biggest change with any company rebranding is the website: www.twolabs.com.

Learn More About Our Services

Our goal is to become a full pharma services consulting organization. The re-branding is just the beginning.  

Topics: Two Labs

7 Top Pharma Trends for 2017

Posted by Jessica Krauser on February 7, 2017

From the effects of Donald Trump’s election as US president to pioneering therapies for Alzheimer’s, some trends are set to continue and others to emerge as the global pharmaceutical industry pushes into 2017.

Analyzing the top seven pharma trends forecast for the new year, Pennside Partners offers some key insights into the essentials.

 

Pharma Trend #1: High-tech pharma

With inhalers that track doses and products that monitor patient compliance, high-tech in medication is becoming increasingly important. “The big thing that strikes me is how pharma is becoming more and more dependent on medtech,” commented Dr. Munna Choudhury of AlacraMed in an interview with Life Science Investing News. These technological additions give a product its competitive edge, according to Choudhury. The article highlights that as a result, we could see more collaborations between pharmaceutical companies and unlikely players, such as telecoms companies and mobile device manufacturers. With support from these tech firms, pharmaceutical manufacturers can develop holistic products to support applications including diagnostics, monitoring, and compliance.

Similarly, PharmaPro expects companies to unlock the potential of AI, Big Data, and Cloud technologies to support their supply chains, predict outcomes, and prescribe actions autonomously – offering significant promise for return on investment. PharmaPro said: “In 2017 these advancements will continue to evolve to make the entire supply chain autonomous.”

 

Pharma Trend #2: New treatments for Alzheimer’s

As the baby boomer generation grows older, the Alzheimer’s Association predicts Alzheimer’s Disease (AD) to account for almost 25% of Medicare spending by 2040 – a huge increase over the predicted 2.1% of Medicare spending in 2020. So far, the pharmaceutical industry has not discovered an optimal response to the disease. According to Forbes.com, although billions of dollars have been invested in targeted antibody drugs, the trend is likely to shift towards alternative scientific approaches in 2017.

One theory is that Alzheimer’s can be fought by preventing the immune system from removing synapses required for neuronal functioning. The article also highlights that an anti-inflammatory drug reportedly improved memory in a small set of patients with mild cases of AD.

 

Pharma Trend #3: Political uncertainty in the UK

A significant carry-over trend from 2016 is the political uncertainty following Brexit. According to Pharmafocus, significant consequences of the UK leaving the EU on the pharmaceutical industry could be regulatory. The article explains that for a drug to be placed on the EU market it must have a marketing authorization (MA), which may be granted by the European Medicines Agency’s recommendation to the European Commission.

Alternatively, applications may be considered by a ‘reference member state’ (RMS) residing in the EU. Once approved by that country, the other EU RMS should grant national approvals. It is not yet clear whether the UK could continue to be the RMS for authorized products after it leaves the EU. For more analyses of the possible consequences of Brexit, click here.

 

Pharma Trend #4: The Trump Effect

In addition to UK and EU market uncertainties, the US is anticipating its own economic policy uncertainty with the election of Donald Trump as president. Trump’s plans appear to be a mixed bag for consumers and Big Pharma alike because it is nearly impossible to gauge their potential impact. Trump’s health plan states: “Reform the Food and Drug Administration, to put greater focus on the need of patients for new and innovative medical products.” According to The Washington Post, this represents a hint that the drug and medical device industries may soon have an easier time getting products to market. Robert Weissman, president of Public Citizen, countered, “…the general deregulatory rhetoric…is a worry for us, but as applied to FDA, it would be very troubling,” meaning that deregulation may compromise safety standards.

Trump has also pledged to cut corporation tax for American companies, with The Telegraph reporting that this move could trigger significant pharma mega-mergers. Furthermore, Trump has suggested he would allow businesses to repatriate money earned overseas into the US without facing hefty fines.

Yet, Trump has intimated that he would allow the importation of high-price medications from abroad, where they are cheaper, notes The Telegraph. This would result in parallel markets entering into direct competition with the US market. While it is impossible to foresee where these proposed reforms will take the economic and regulatory aspects of the pharmaceutical industry, it will be vital to have a finger on the pulse.

 

Pharma Trend #5: Drug prices

The end of 2016 saw several major pharma companies sued for allegedly fixing drug prices, and public outcry in the face of pricing is likely to continue into 2017. Pharmaceutical consultant Carole Bruckler told Pharmaceutical Investment News: “In the US, there is clear political will forming against annual or quarterly drug price increases to fuel sales growth.”

Although the election of Trump is likely to prove more favorable to drug prices than a Clinton victory would have – under Trump, we could see reduced regulation on drug price inflation – pharma companies may have to reconsider their business models, warns the article. Bruckler added, “For pharma revenues to be sustainable in the future there needs to be an increased focus on volume growth … so investors need to really understand the substance behind their investments.”

 

Pharma Trend #6: Biosimilar profitability

Biosimilar drugs present a more affordable option for the consumer and are also potentially lucrative for manufacturing companies. Importantly in 2017, these biological products are set to continue to increase in popularity. Since the first biosimilar drug was approved for use in America in 2015, there are now more than 700 biosimilars approved or in the pipeline. Sales could be boosted by 11 key biologics that face loss of exclusivity over the next seven years, with biosimilar sales expected to generate 27 per cent of the total pharma market by 2020.  

 

Pharma Trend #7: The microbiome

DNA sequencing tools that have boosted understanding of how we exist with the bacteria in our guts has brought the microbiome to significant prominence in biology over recent years. Research suggesting that the bacterial composition of the gut affects mood opens up new avenues for the treatment of depression and other mental illness or disorders of the central nervous system. However, according to Forbes.com, how biotech and pharma companies can develop drugs based on this science is an open question – despite development in that direction.

Topics: Pharma Trends, Pharma Consulting

Medical 3D Printing Breakthroughs Keep on Coming

Posted by Jessica Krauser on August 4, 2016

Medical 3D printing is making quick leaps and bounds and could soon be such a key integrated part of the medical world that we will wonder how we ever lived without it.

Doctors are now able to print 3D “living” body parts – which will be a significant advancement for regenerative medicine. A positive breakthrough has already been made, with the successful implantation of bone, muscle and cartilage into animals.

Previously, the field had hit a wall: keeping the cells alive was difficult, as they became starved of oxygen and nutrients. This problem led to a very promising solution by the team at Wake Forest Baptist Medical Centre.


The Sponge

It is exactly what it sounds like. The Wake Forest team developed the idea of printing the tissue with channels interwoven throughout to allow key ntrients to pass through it.

How can a “living” body part be printed with these channels? Through the Integrated Tissue and Organ Printing System (ITOP), which utilizes a bio-degradable plastic to give the tissue its structure, as well as a water-based gel that contains the cells and encourages growth.

These channels allow blood vessels and nerves to grow into the printed tissue. Before the ITOP 3D printer, the largest structure to be kept alive without blood cells was 0.007 inches thick. With the ITOP size isn’t an issue, as the microscopic pores are interwoven into the printed structure allowing the i
plant to be kept alive indefinitely.

Are 3D Organ Transplants Next?

This development gives 3D organ printing a promising outlook. Within the next few years we could be expecting success stories on artificial heart, liver, kidney, and other major organ transplants, giving many on the transplant list hope. Pennside will continue to follow the development of 3D printing in the healthcare industry.

If you aren’t familiar with how 3D printing is impacting the pharma and biotech industry check out Pennside’s earlier blog postings to learn more.

The above post is a condensed and edited summary of NBC News: Wake Forest University Scientists Print Living Body Parts, BBC News: Doctors 3D-print 'living' body parts and Popular Mechanics: Incredible 3D Printer Can Make Bone, Cartilage, and Muscle

Topics: Medical 3D Printing

Prescription Drugs: To Import or Not to Import?

Posted by Jessica Krauser on July 12, 2016

Do cheaper, imported drugs really save money – or just compromise safety?

50 million Americans did not fill a prescription in 2012 because of its cost, a Commonwealth Fund report revealed.[1] Now, prescription costs are under the political spotlight again, with US presidential candidates Donald Trump, Hillary Clinton and Bernie Sanders all commenting on the issue.  

Speaking out against current Food and Drug Administration (FDA) laws that state it is illegal to import drugs into the USA except in rare cases, the presidential candidates all voiced support for the enhanced importation of prescription drugs to address the issue of cost.

Clinton believes America needs “to promote competition and leverage our nation’s bargaining power to lower drug costs on behalf of Americans”.[2] Sanders wants to allow individuals, pharmacists, and wholesalers to import prescription drugs from licensed Canadian pharmacies.[3] And Trump has proposed removing “barriers to entry into free markets for drug providers that offer safe, reliable and cheaper products”.[4]

But do cheaper, imported drugs really save money – or just compromise safety?

The cost of the FDA

Among the reasons drugs sold in America cost more than in places like Canada or Europe are the approval processes laid out by the FDA. The agency states on its website that it is “illegal for individuals to import drugs into the United States for personal use, because the FDA cannot ensure the safety and effectiveness of these drugs.”[5]

The FDA’s Center for Drug Evaluation and Research (CDER) has the job of evaluating new drugs before they are sold to ensure that brand name and generic drugs work correctly and that the health benefits of the drug outweigh the known risks.

It states on its website: “A team of CDER physicians, statisticians, chemists, pharmacologists, and other scientists reviews the company's data and proposed labeling. If this independent and unbiased review establishes that a drug's health benefits outweigh its known risks, the drug is approved for sale. The center doesn't actually test drugs itself, although it does conduct limited research…”[6]

However, the FDA’s regulatory processes have driven up the cost of prescription drugs to sometimes unaffordable levels – for both consumers and pharmaceutical companies. According to the Tufts Center for the Study of Drug Development, the cost of developing drugs has increased phenomenally since 1975.[7]

Its research shows that in 1975, the pharmaceutical industry spent the equivalent of $100 million researching and developing the average drug approved by the FDA. By 2005, that figure had risen to $1.3 billion. At the same time, the high rate of drug failure (only one in 12 drugs that enter human clinical trials secure FDA approval) means that pharmaceutical companies spend about $5 billion per new drug.

Driving this increase has been the FDA’s regulatory process for Phase III clinical trials. These trials typically represent 90% or more of the cost of developing an individual drug from laboratory to pharmacy, highlights commentator Avik Roy in an article on Forbes.com.

The consequence is higher health spending, he explains, as pharmaceutical companies have to charge more to recoup their costly investments. [8]

The dangers of importation

Some believe that the cost of these regulatory processes is a necessary one. The Philadelphia Inquirer’s Robert Blancato argues that any money-saving notions are too good to be true. In his article he comments: “Drug importation is a terrible idea that would put Americans at risk of harm from impure, unsafe, and counterfeit copies of prescription drugs[9].”

Existing cases support his argument. During the drug importation debate in 2003, the FDA examined mail shipments and found that 88% of the imported drugs they tested did not meet FDA standards, with some packages containing unapproved or counterfeit drugs.[10]

In a speech at that time, former FDA Commissioner Mark McClellan commented: “In addition to allowing in some drugs that might be safe, [drug importation] would also create wide and poorly-regulated channels through which a lot of potentially unsafe drugs will also be able to enter the country more easily.”[11] The FDA website explains that these drugs may be contaminated or contain the wrong or no active ingredient. On the other hand, they could have the right active ingredient, but at the wrong dose. [12] 

Campaign rhetoric or a real solution to pricing problems?

Could loosening the reins on drug importation provide American’s some financial relief? If Americans were able to import from select pharmacies in other countries drug costs would decrease and the FDA could still keep a hand in regulating.

Are the presidential candidates right? Does the question of price outweigh these issues of safety?

In 2013, a group from Maine, consisting of state employees, Portland employees, and one large company, claimed to have saved approximately $10 million over several years by purchasing their drugs through a Canadian mail-order pharmacy. [13]

The same year, the National Health Interview Survey revealed worrying statistics about the affordability of prescription drugs: to save money, almost 8% of U.S. adults did not take their medication as prescribed, and just over 15% asked a doctor for a lower-cost medication.[14]

Based on these statistics, if lack of affordability forces people to avoid taking medication as prescribed, is importation really a dangerous idea? After all, in Canada there are drug checks similar to those carried out by the FDA: Health Canada (HC) regulates drug approvals, claiming that “the safety and wellbeing of Canadians is the paramount concern.”

Cost vs. speed

In Europe, where drugs are also cheaper than in America, there is the European Medicines Agency (EMA), which is responsible for the “protection and promotion of public and animal health, through the evaluation and supervision of medicines.”[15]

There are differences between the bodies, though, providing possible reasons for the discrepancies in cost. Let’s take the question of time: the FDA trumps both the Canadian and European agencies for speed. Research conducted by Yale University’s Joseph Ross revealed that the FDA approved drugs almost two months faster than its counterparts, getting products to market first in the majority of cases.[16]

It took the FDA an average of 322 days to approve a drug product, compared to 366 days for the EMA and 409 days for Health Canada (HC). Perhaps then, America is paying the price of efficiency.

Perhaps it also paying the price of a more concise approvals process. According to an article published in Oncology Journal in 2013, the FDA both scientifically evaluates new agents and issues marketing approval decisions for therapeutics. Meanwhile, the scope of the EMA’s Committee for Medicinal Products for Human Use is limited to the scientific evaluation of therapeutics. The EMA then makes recommendations to the European Commission for marketing approval of the drug.

The article adds that additional differences between the agencies arise because each EU member state has a different system for therapeutics approvals. For example, in Britain, licences can be acquired through the Medicines and Healthcare Products Regulatory Agency (MRHA) or the EMA. The National Institute for Health and Care Excellence (NICE) must then evaluate efficacy and any cost concerns.

In the EU, national authorities are free to set the prices of medicinal products and to designate the treatments they wish to reimburse under their social security systems. At the same time, pricing and reimbursement systems are closely linked to the realization of European policy objectives such as the internal market, pharmaceutical competitiveness, sustainable research and development, and the protection of human health. The variety of healthcare and social security systems in the EU has an impact on the pharmaceutical industry, wholesalers, pharmacists, doctors, health insurers, and patients.[17]

Looking past the election 

For now, cost concerns are still a part of life for many American patients. But as the election campaign heats up, we’ll have to wait and see what lies in store post-November for the future of prescriptions.

 

[1] Collins, Sara R., Ruth Robertson, Tracy Garber, and Michelle Doty. "Insuring the Future: Current Trends in Health Coverage and the Effects of Implementing the Affordable Care Act." The Commonwealth Fund, April 2013. Accessed April 25, 2016.

[2] "Hillary Clinton's Plan for Lowering Prescription Drug Costs." Hillary Clinton's Plan for Lowering Prescription Drug Costs. Accessed April 25, 2016. https://www.hillaryclinton.com/briefing/factsheets/2015/09/21/hillary-clinton-plan-for-lowering-prescription-drug-costs/.

[3] "On the Issues: Fighting to Lower Prescription Drug Prices." Bernie Sanders RSS. Accessed April 25, 2016. https://berniesanders.com/issues/fighting-to-lower-prescription-drug-prices/.

[4] "Healthcare Reform." Make America Great Again! Accessed April 25, 2016. https://www.donaldjtrump.com/positions/healthcare-reform.

[5] "U.S. Food and Drug Administration." Personal Importation. Accessed April 25, 2016. http://www.fda.gov/ForIndustry/ImportProgram/ImportBasics/ucm432661.htm.

[6] "Development & Approval Process (Drugs)." U.S. Food and Drug Administration. Accessed June 09, 2016. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/.

[7] Roy, Avik. "How the FDA Stifles New Cures, Part I: The Rising Cost of Clinical Trials." Forbes. April 24, 2012. Accessed April 29, 2016. http://www.forbes.com/sites/aroy/2012/04/24/how-the-fda-stifles-new-cures-part-i-the-rising-cost-of-clinical-trials/#2d2a18bf5bc6.

[8] Roy, Avik. "How the FDA Stifles New Cures, Part I: The Rising Cost of Clinical Trials." Forbes. April 24, 2012. Accessed May 02, 2016. http://www.forbes.com/sites/aroy/2012/04/24/how-the-fda-stifles-new-cures-part-i-the-rising-cost-of-clinical-trials/#2d2a18bf5bc6.

[9] Blancato, Robert. "Commentary: Drug-import Proposal Would Compromise Safety." Philly-archives. April 07, 2016. Accessed April 25, 2016. http://articles.philly.com/2016-04-07/news/72105577_1_drugs-online-prescription-drugs-fda-associate-commissioner.

[10] Turner, Grace-Marie. "Drug Importation Equals Unsafe Drugs, Mr. Trump." Forbes. March 16, 2016. Accessed April 25, 2016. http://www.forbes.com/sites/gracemarieturner/2016/03/16/drug-importation-equals-unsafe-drugs-mr-trump/#799fd87bd3d3.

[11] McClellan, Mark B. "Speech before Fifth Annual David A. Winston Lecture." FDA U.S. Food and Drug Administration. October 20, 2003. Accessed April 25, 2016. http://www.fda.gov/NewsEvents/Speeches/ucm053609.htm.

[12] "U.S. Food and Drug Administration." Counterfeit Medicine. Accessed April 26, 2016. http://www.fda.gov/Drugs/ResourcesForYou/Consumers/BuyingUsingMedicineSafely/CounterfeitMedicine/.

[13] Space, Judi. "Is Maine Right About Importing Prescription Drugs?" Is Maine Right About Importing Prescription Drugs? November 23, 2013. Accessed May 02, 2016. http://www.pennside.com/blog-pharma-biotech/bid/326207/Is-Maine-Right-About-Importing-Prescription-Drugs.

[14] Cohen, Robin A., and Maria A. Villarroel. "Strategies Used by Adults to Reduce Their Prescription Drug Costs: United States, 2013." Centers for Disease Control and Prevention. January 29, 2015. Accessed May 02, 2016. http://www.cdc.gov/nchs/products/databriefs/db184.htm

[15] "European Medicines Agency." EUROPA. Accessed June 13, 2016. http://europa.eu/about-eu/agencies/regulatory_agencies_bodies/policy_agencies/ema/index_en.htm.

[16] Gaffney, Alexander. "Researchers: FDA Beats EMA, Health Canada in Drug Approval Times | RAPS." RAPS: Regulatory Affairs Professional Society. June 20, 2012. Accessed May 09, 2016. http://www.raps.org/focus-online/news/news-article-view/article/1798/.

[17] "Pricing and Reimbursement of Medicinal Products." European Commission. Accessed May 19, 2016. http://ec.europa.eu/growth/sectors/healthcare/competitiveness/products-pricing-reimbursement/index_en.htm.

NHS England Overhauls Access to Cancer Treatments

Posted by Jessica Krauser on June 30, 2016

Patients in England could have faster access to effective cancer treatments through the reincarnated Cancer Drugs Fund (CDF), the National Institute for Health and Care Excellence (NICE) has announced.

The new fund will replace the existing – and greatly overspent – service, which provides access to cancer drugs that are not routinely available at the National Health Service (NHS) in England. Its costs rose to £340 million between 2015 and 2016, following an annual budget of £200 million when it was set up in 2011.

Devised to counter this overspending, the new system will launch in July, fixing the fund's annual budget at £340 million.

Old CDF Versus New CDF

While the existing CDF can choose to pay for innovative drugs that NICE has rejected for widespread use on the NHS, under the new system the health watchdog will make all the decisions.

In a European first, NICE will issue draft guidance on new cancer drugs or significant new license indications before they have received marketing approval in the UK.

Any drug that receives a positive draft recommendation will be funded from the point of license, with NICE normally issuing final guidance within 90 days of the license. Drugs given a “maybe” rating will be considered for the CDF.

The aim is to bring clarity to decisions about which new cancer drugs will receive NHS funding, as well as to allow the NHS to offer conditional funding for those with uncertain evidence for use.

Sir Andrew Dillon, NICE chief executive, commented in a press release: “Patients in this country will now have access to clinically and cost effective, innovative new cancer drugs faster than ever before.”

He added: “In a first of its kind approach, NICE will issue draft recommendations on the use of cancer medicines before they receive their license, with funding from NHS England available if approved. No other country in Europe does this.”

Impact on the pharma industry

If the case for routine use of a drug is not clear cut and more evidence is needed to prove its cost effectiveness, NICE can recommend the drug for temporary, conditional use in the new CDF.

The drug will remain available in the CDF for up to two years while the manufacturer gathers evidence to show that the medicine works and is fairly priced.

After two years, NICE will conduct a review to consider the drug for routine commissioning on NHS England, with the medicine moving out of the CDF and into routine budgets, or being made available on an exception only basis. 

Dillon argued: “The new CDF will be fair. Companies will be given the opportunity to demonstrate their drugs will benefit patients and bring value to the NHS.”

However, concerns about the new fund have been raised, with claims that NICE’s appraisal process could block access to drugs. The Pharma Times reported that the Association of the British Pharmaceutical Industry was disappointed by NHS England’s decision to push ahead with proposals that carry a “very real risk of significantly setting back patient access to cancer medicines, now and for the foreseeable future”.

Paul Catchpole, value and access director of the Association, told the journal: “If cancer medicines go through more or less exactly the same NICE appraisal process that was in place five years ago - which necessitated the setting up of the CDF in the first place - we will largely get the same answers as before - the majority of medicines will be turned down”.

He warned that under the proposals, two thirds of existing CDF medicines would be likely to no longer be available to NHS patients by the end of the year.

Cancer charities agree. In a letter to the British prime minister published in newspaper The Daily Telegraph, 15 charities wrote that many drugs available the western world would "now struggle to gain approval" in England due to NICE’s “last-century methodology”.

The letter stated: "Unfortunately the new system does not update the methodology used by NICE, introduced back in 1999, and many clinically-effective treatments will now struggle to gain approval.”

The charities highlighted that since 2011, when it was launched, the CDF has paid for 84,000 cancer treatments that NICE turned down. Speaking to the BBC, Baroness Delyth Morgan, chief executive of charity Breast Cancer Now, commented: "Not a single breast cancer drug has been considered cost-effective by NICE in the last seven years and this simply cannot continue." Among the breast cancer drugs NICE has rejected is Kadcyla, a treatment which can prolong a dying patient’s life by six months on average.

Cancer Research UK explains on its website that NICE’s approval process is based on whether a drug works, whether it is cost effective and contributions from patient organizations, health professionals and experts.

Important Dates

The current CDF closed on 1 April 2016. Drugs transferring from the old CDF will be appraised by NICE over the next 18 months. Entirely new drugs will be able to enter the CDF from July 2016.

All drugs on the existing CDF list will continue to receive funding until the point that NICE has completed a new appraisal of them, and any patients currently receiving them will finish their treatment.

The CDF was established in 2011 and covers England.

This article is a synopsis of: Faster access to effective cancer treatments through new Cancer Drugs Fund, Cancer Drugs Fund changes deeply concerning, say charities, and What is NICE and how does it work?

Pennside Partners Expands European Presence through new UK office located South of Manchester

The Pennside team is excited to announce their new office in Alderley Edge in the UK, 25 KM south of Manchester. This ideal location will help us better serve our European client base due to its proximity to local pharma and biotech companies. Thomas Penrice, Vice President and Partner will be the EU Managing Director. Learn more

T-Cell Immunotherapy Market Predicted To Grow

Posted by Jessica Krauser on April 21, 2016

Nearly one million people are diagnosed with cancer in the United States per year, according to The American Cancer Society, and although cancer therapeutics are among the most active areas in drug development, treatment still falls short of meeting patients’ needs.

However, experts believe that immunotherapeutics – in particular T-cell immunotherapy – could help fill a vital gap, with Roots Analysis’ "T-Cell Immunotherapy Market, 2015-2030" report predicting the market will be worth $30 billion USD by 2030. 

Robust products in T-cell immunotherapy

Shannon Flynn, a consultant at Pennside with 3 years’ experience in gene therapy, explains that T-cell immunotherapy is an emerging therapeutic modality in which tumor-targeting T-cells are administered to the patient as a sort of “living drug.”

The therapy involves collecting T-cells, either from the patient (autologous) or a donor (allogeneic), as the active ingredient for treatment.  The cells are then modified to enhance their anti-tumor potential, before being expanded to increase total cell numbers for an optimized final product which can be administered to the patient.

Separated into three segments – CAR-T, TCR, and TIL therapies, the market is characterized by a robust pipeline of products targeting hematological cancers and solid tumours.

Researchers identified more than 180 T-cell therapies across various phases of development: at 56%, CAR-T therapies are the most common – and likely to gain the most attention – followed by TCR (25%) and TIL (19%) therapies. Engineered CAR-Ts with switch technologies are among the latest additions to the T-cell immunotherapy pipeline.

Pennside’s analysts note that some of the most recent developments in the field involve engineering CAR-T cells with sophisticated switch technologies which serve to modulate the activity of the cells in vivo. These technologies are primarily aimed at improving safety of the cells by migrating on-target/off-tumor toxicities.

For example, cells can be designated to turn “on” or “off” in the presence of a small molecule drug, while others might be engineered to activate only in response to stimuli from the tumor itself. Novel technologies such as these afford the CAR-T cells with mechanisms to enhance their tumor targeting potential and avoid damaging healthy tissues.

Driving factors behind T-cell developments

As for progress, the numbers speak for themselves: the report identified investments in T-cell research nearing USD 3 billion across start-ups and small firms. Big industry players are also driving research in the field.

Further helping push the T-cell market forward are lucrative rounds of VC funding, the discovery of novel targets, encouraging clinical trial results, and developments in immunotherapy products.

Meanwhile, various providers have pioneered improved efficacy and safety in developing platforms for engineering T-cells. The market has also attracted interest from technology providers with capabilities in genome editing, and viral and non-viral gene transfer. Other providers are developing innovative safety switches to manage side effects, such as cytokine release syndrome and B-cell aplasia.

The report, published by Roots Analysis, provides a comprehensive view of the market, which is still in its infancy.

This blog is based on the press release: http://www.prnewswire.com/news-releases/t-cell-immunotherapy-market-2015-2030-300234571.html and report http://www.rootsanalysis.com/reports/view_document/t-cell-immunotherapy-market-2015-2030/108.html