Rare Disease Highlight: Acute Myeloid Leukemia

Acute Myeloid Leukemia (AML) is a blood cancer in which blast cells (immature myeloid precursor cells) multiply uncontrollably within the bone marrow and blood.1 The accumulation of blast cells disrupts normal blood cell production in the bone marrow as well as blood distribution to the tissues. This disruption leads to symptoms such as chronic fatigue, anemia, a compromised immune system, and high risk of hemorrhage.4,5 In addition, the current treatments for AML can cause many additional side effects due to drug toxicity.6-8 As these combined effects are severe, AML patients have low overall survival despite treatment and can still relapse after recovery is observed.2,3 According to the Surveillance, Epidemiology, and End Results (SEER) program, there were 19,520 new cases of AML in the US in 2018, with a reported 5-year survival rate of 27.4%.9  Risk factors associated with AML include age, history of blood disease, and/or genetic disorders.2,3 Thus, an unmet need exists for AML therapies which are non-invasive, non-toxic, and do not result in relapse.

References used
  1. Spivak JL. Acute Myelogenous Leukemia (AML). Merck Manual 2017; http://www.merckmanuals.com/professional/hematology-and-oncology/leukemias/acute-myelogenous-leukemia-aml. Accessed November 20, 2017, 2017.
  2. Pui C-H, Carroll WL, Meshinchi S, Arceci RJ. Biology, Risk Stratification, and Therapy of Pediatric Acute Leukemias: An Update. J Clin Oncol. 2011;29(5):551-565.
  3. PDQ PTEB. Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®). 2017.
  4. Cheng MJ, Hourigan CS, Smith TJ. Adult Acute Myeloid Leukemia Long-term Survivors. Journal of leukemia (Los Angeles, Calif). 2014;2(2):26855.
  5. Dohner H, Estey EH, Amadori S, et al. Diagnosis and management of acute myeloid leukemia in adults: recommendations from an international expert panel, on behalf of the European LeukemiaNet. Blood. 2010;115(3):453-474.
  6. Lipshultz SE, Sallan SE. Cardiovascular abnormalities in long-term survivors of childhood malignancy. J Clin Oncol. 1993;11(7):1199-1203.
  7. Volkova M, Russell R. Anthracycline Cardiotoxicity: Prevalence, Pathogenesis and Treatment. Curr Cardiol Rev. 2011;7(4):214-220.
  8. Von Hoff DD, Layard MW, Basa P, et al. Risk factors for doxorubicin-induced congestive heart failure. Ann Intern Med. 1979;91(5):710-717.
  9. SEER. Cancer Stat Facts: Leukemia – Acute Myeloid Leukemia (AML). 2018; https://seer.cancer.gov/statfacts/html/amyl.html.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutics areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Rare Disease Highlight: Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a genetic disease which affects the part of the nervous system controlling voluntary muscle movement.1 SMA is the most common cause of mortality in infants associated with a genetic mutation. It affects approximately 1 in 6,000 to 10,000 people.2 Patients with SMA develop progressive muscle weakness caused by the loss of specialized nerve cells called motor neurons in the spinal cord and the brain stem. The symptoms of SMA cover a broad spectrum of severity based on the age at disease onset and motor ability.3 The most common form of SMA (accounting for 95% of all cases) is associated with mutations in the survival motor neuron 1 (SMN1) gene which lead to SMN1 protein deficiency and eventual loss of motor neurons.4 Currently there is no cure for this serious and life-threatening disease. While there are treatments to help manage the condition, the development of new SMA therapies are necessary to address longstanding unmet needs.5

References used
  1. Faravelli, I., Nizzardo, M., Comi, G., & Corti, S. (2015). Spinal muscular atrophy—recent therapeutic advances for an old challenge. Nature Reviews Neurology, 11(6), 351-359. doi: 10.1038/nrneurol.2015.77
  2. Pearn, J. (1978). Incidence, prevalence, and gene frequency studies of chronic childhood spinal muscular atrophy. Journal Of Medical Genetics, 15(6), 409-413. doi: 10.1136/jmg.15.6.409
  3. Russman, B. (2007). Spinal Muscular Atrophy: Clinical Classification and Disease Heterogeneity. Journal Of Child Neurology, 22(8), 946-951. doi: 10.1177/0883073807305673
  4. Kolb, S. (2011). Spinal Muscular Atrophy. Archives Of Neurology, 68(8), 979. doi: 10.1001/archneurol.2011.74
  5. Spinal Muscular Atrophy Treatment – SMA News Today. (2019). Retrieved from https://smanewstoday.com/spinal-muscular-atrophy-treatment

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutics areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Rare Disease Highlight: Hepatocellular Carcinoma (Liver Cancer)

Hepatocellular Carcinoma is a form of liver cancer associated with various stages of malignant growth in the liver. It is the sixth most common cancer worldwide, but is rare in the United States, only affecting around 60,000 patients.1,2 Hepatocellular carcinoma is considered a deadly cancer, with a survival rate of only 12.2% for five years, and the third major leading cause of cancer-related deaths worldwide.3,4 While most liver cancers are preventable, the incidence of hepatocellular has been increasing in the United States. This is possibly due to the frequency of common risk factors such as chronic liver disease, viral liver infections such as hepatitis, and liver cirrhosis.3,5 Hepatocellular carcinoma can be treated with standard of care therapies. Unfortunately, many patients with the disease have a high risk of developing resistance creating an unmet need for new treatment options of this serious and life-threatening disease.6

References used
  1. SEER Cancer Stat Facts: Liver and Intrahepatic Bile Duct Cancer. NCI; 2018. https://seer.cancer.gov/statfacts/html/livibd.html. Accessed 03/15/2018.
  2. Zhu RX, Seto WK, Lai CL, Yuen MF. Epidemiology of Hepatocellular Carcinoma in the Asia-Pacific Region. Gut Liver. 2016;10(3):332-339.
  3. Momin BR, Pinheiro PS, Carreira H, Li C, Weir HK. Liver cancer survival in the United States by race and stage (2001-2009): Findings from the CONCORD-2 study. Cancer. 2017;123 Suppl 24:5059-5078.
  4. Zhu YJ, Zheng B, Wang HY, Chen L. New knowledge of the mechanisms of sorafenib resistance in liver cancer. Acta Pharmacol Sin. 2017;38(5):614-622.
  5. Kamarajah SK. Fibrosis score impacts survival following resection for hepatocellular carcinoma (HCC): A Surveillance, End Results and Epidemiology (SEER) database analysis. Asian Journal of Surgery. 2018;1(1)
  6. Pan S, Li Z, He Z, Qiu J, Zhou S. Molecular Mechanisms for Tumor Resistance to Chemotherapy. Clinical and Experimental Pharmacology and Physiology. 2016;2016(43):723-73

BioPharma Global is a not for profit-operating regulatory affairs firm specializing in FDA and EMA expedited designations for companies working to treat rare diseases. If you are a rare disease drug developer seeking Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, or other FDA/EMA designations, or if you are seeking pre-IND meeting assistance (type A, B, or C), the BioPharma Global regulatory team can help. Contact us today to arrange a 30-minute introductory call.

BioPharma Global is Proud to Reaffirm our Commitment to Asia

BioPharma Global, America’s premier regulatory specialists for FDA and EMA orphan drug designations and regulatory strategy, is honored to announce the enhancement of its Global Business Initiative and East Asian outreach with the addition of Yalin Zhang, who brings strong regulatory expertise and a unique understanding of the Asian market place. Yalin is a graduate of Georgetown University where she received a Master’s in Biotechnology. In addition to her other skills Yalin is Bilingual in English and Mandarin Chinese. With Ms. Zhang at our side we proudly extend our greetings and our services to the Chinese and other East Asian biopharmaceutical industries. We at BioPharma Global eagerly look to a future of connecting Asia’s brightest researchers with both the American  and EU markets by providing regulatory services specializing in FDA/EMA Orphan Drug, Breakthrough, and Fast Track Designations, among others. 

BioPharma Global荣幸的重申我们对亚洲的承诺

BioPharmaGlobal是美国FDAEMA孤儿药资格认定首要的专业药政咨询公司,我们荣幸的宣布扩大我们全球商业及亚洲业务拓展战略,并热烈欢迎新成员张雅林的加入。她拥有强大的药政专业知识和对亚洲市场的深入理解,毕业于Georgetown University并获得生物技术硕士学位,除此之外双语也是亮点之一。我们很荣幸的向中国和其他亚洲生物制药公司致以我们诚挚的问候。BioPharmaGlobal希望通过提供专业的FDA/EMA孤儿药资格认定,突破性疗法资格认定和快速审评等其他药政咨询,将亚洲最先进的药物研发项目与美国市场相对接。

Biopharma Champion Sen. Orrin Hatch is Retiring

Sen. Orrin Hatch (R-Utah), chairman of the Senate Finance Committee, announced Tuesday that he will not seek reelection in 2018. First elected in 1976, Hatch has done more to shape the legal and regulatory landscape for drug development than any other serving member of Congress. He has been the biopharma industry’s most reliable ally. The biopharma industry has supported Hatch, donating $999,000 over the last five years to his campaign committee and political action committees, according to Federal Election Commission data.

After initially opposing the Orphan Drug Act, Hatch co-sponsored the Senate version of the bill and urged President Ronald Reagan to sign it into law in 1983. Hatch has been a leading advocate in Congress for the creation of legislative incentives to stimulate R&D on rare diseases.

To Read the Complete Article at Biocentury, Click Here

Working on a cure for a rare disease? Need Breakthrough Therapy designation, Fast Track Designation, RMAT Designation, FDA or EMA Orphan Designations? The BioPharma Global Regulatory Team can help – We are the #1 US based not-for-profit Global regulatory affairs firm with cost-effective, time-efficient solutions to chaperone your path through the regulatory affairs process. Contact us at https://www.biopharmaglobal.com/contact/ or by phone +1(202)660-1826

BioPharma Global strongly advocates for retention of Orphan Drug Tax Credit

The Orphan Drug Tax Credit is at risk of being drastically reduced, if not repealed, in the new tax overhaul bill being revised by the Senate and House of Representatives. BioPharma Global believes the Orphan Drug Tax Credit should be preserved for the benefit of patients with rare diseases. Because of the tax incentive, many small pharmaceutical companies are able to pursue the research and development of drugs to treat crippling rare diseases for which development would otherwise be too costly.

The Orphan Drug Act was signed into law by President Reagan in 1983 to provide incentives for pharmaceutical companies to develop drugs for rare diseases. A rare, or orphan, disease is defined as one that affects less than 200,000 people. Companies able to obtain an Orphan Drug Designation from the FDA receive many incentives including seven years of market exclusivity and the aforementioned Orphan Drug Tax Credit, for which they get a 50% tax credit for qualified clinical trial costs.

The Orphan Drug Act creates opportunities for scientists and entrepreneurs to advance rare disease research. Since the Act was passed, more than 486 orphan products have been approved. Prior to the Orphan Drug Act, only 34 drugs were approved to treat rare diseases. A report from Ernst and Young estimates that eliminating the tax incentive would decrease orphan drug discovery by 33%.

Today, 96% of the 30 million Americans that suffer from a rare disease face a dearth of treatment options. Eliminating the Orphan Drug Tax Credit diminishes the hope of those still awaiting treatment. BioPharma Global strongly advocates that the Orphan Drug Tax Credit not be repealed.


Further reading:

Tax Overhaul Looks Set to Cut Credits for Drugs Targeting Rare Diseases (Wall Street Journal)

New White Paper from BIO, NORD, and EY Finds Orphan Drug Tax Credit is Crucial to Rare Disease R&D (Ernst & Young Whitepaper)

Perfecting Your Hiring Formula

Biotech is one of the most competitive spaces for employment so finding properly motivated employees to join your team is key to sustaining lower turnover and higher company performance.  The single most expensive line item is your human capital so creating a recruiting and hiring best practice that works for your business model is important.

Many times, growing biotech budgets are allocated first on R&D and secondly – or sometimes even thirdly – on H&R.  (That’s specifically hiring and recruiting – an important part of HR – but not the whole component).  Outsourcing your recruiting function is often more cost effective and efficient than having an internal recruiter, particularly for companies that have fluctuating needs.

Having a trusted provider who functions as a reliable resource to meet your company’s needs is paramount to success in creating an outsourced pipeline strategy.  At Biopharma Global, our process for filling positions falls in alignment with the data points made in a recent Fast Company article.  Knowing your hiring metrics from start to finish allows us to keep your pipeline full at all times for crucial, harder to fill positions.  We have many clients we serve all over the world and during the client onboarding process we work with them to evaluate their own past performance around hiring so we can create the necessary flow of candidates to them so they can hire key personnel within their established timelines.

It’s not common – but it does happen sometimes – that the first person you interview is perfect for the job.  It’s hard to hold back and not make an offer to hire them on the spot, but it’s always better to call back and follow up rather than acting impulsively.  Feeling stressed  to make an immediate choice is one of the main reasons candidates would not accept an offer on the spot. Interviewing is a lot like dating – you wouldn’t bring an engagement ring on a first date, so don’t feel compelled to make a job offer on the first interview.

Scientific people are analytical and can be scared off by an offer that’s immediate.  Using Biopharma Global to assist you in your recruiting allows you to engage an experienced partner in facilitating acceptance and onboarding employees for success.  Hiring the right employee or contractor is a delicate balance and while money may be a driver in people’s decisions, its not the only factor they use to evaluate an offer.

If you want to hire the right person for your company’s culture, take a little time to ensure you know what it is that motivates them individually, what they are looking for from an employer and determine if you can fulfill that for them.  Employees that are a great fit will stay longer and those decisions are not often made on the fly, therefore your offers shouldn’t be either.  Offers should be prepared with the proper sense of urgency, timing and due diligence to ensure a proper fit.

Want to discuss your current hiring needs?  No matter what your needs may be – from entry to executive level searches – direct hire, project or contract.  Our team has years of success with thousands of successful placements under their belts and is ready to assist you.  Email us to set a time to discuss at BioPharma Global Human Capital Solutions

Bio Pharma Market Data 2017

According to McKinsey & Company “The opportunity in biopharmaceuticals is big and growing too rapidly to ignore. Today, biopharmaceuticals generate global revenues of $163 billion, making up about 20 percent of the pharma market. It is by far the fastest-growing part of the industry: biopharma’s current annual growth rate of more than 8 percent is double that of conventional pharma, and growth is expected to continue at that rate for the foreseeable future. The current biologics-development pipeline supports an outlook of continued healthy growth. The number of biotech patents applied for every year has been growing at 25 percent annually since 1995. There are currently more than 1,500 biomolecules undergoing clinical trials, and the success rate for biologics has so far been over twice that of small-molecule products, with 13 percent of biopharma products that enter the Phase I trial stage going on to launch. If anything, the emerging long-term picture is even more exciting, with disruptive innovations such as immunotherapies, antibody drug conjugates, and gene and cell therapies all making progress toward commercial launch in the next few years.

Quality functions are struggling to keep up with the rising demands of regulators, primarily the US Food and Drug Administration. The industry has received an unprecedented number of warning letters and remediation programs in the last five years, and scrutiny is unlikely to decrease. Furthermore, the increasing relevance of global markets (beyond the United States, European Union, and Japan) is adding the complexity of multiple quality standards and regulatory regimes. Compliance, robustness of processes, and efficiency will need to be squared in one equation.”

Worldwide biotechnology market (therapeutics and diagnostics, R&D services, other) consists of 14,000+ companies, with ~4,000 of them located in the United States.

Companies per sector (Worldwide)

Sector Total companies
Biotechnology – Therapeutics and Diagnostics: 3,862
Biotechnology / R&D Services: 6,493
Biotechnology – other: 3,717
Pharma: 2,299
Medical Technology: 5,125
Investor: 735
Professional Services and Consulting: 5,013
Public / Non-Profit Organizations / Medical Facil: 5,202
Supplier & Engineering: 8,890
Media: 171
HealthTech: 844
Total: 42,351
Total Number of Biotech companies: 14,072

 

 

Companies per sector (USA)

Sector Total companies
Biotechnology – Therapeutics and Diagnostics 1,773
Biotechnology / R&D Services 1,672
Biotechnology – other 320
Pharma 219
Medical Technology 1,341
Investor 248
Professional Services and Consulting 1,260
Public / Non-Profit Organizations / Medical Facility 862
Supplier & Engineering 1,153
Media 55
HealthTech 135
Total 9038