Pharmaceutical innovations

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Pharmaceutical innovations are currently guided by a patent system, [1] the patent system protects the innovator of medicines for a period of time. The patent system does not currently stimulate innovation or pricing that provides access to medicine for those who need it the most, It provides for profitable innovation. [2] As of 2014 about $140 Billion is spent on research and development of pharmaceuticals which produces 25–35 new drugs annually. Technology, which is transforming science, medicine, and research tools has increased the speed at which we can analyze data but we currently still must test the products which is a lengthy process. [3] Differences in the performance of medical care may be due to variation in the introduction and circulation of pharmaceutical innovations. [4]

Contents

The pharmaceutical industry does not apply the same definition of "innovative" as other industries because while a new product might offer a new mechanism of action, in and of itself that holds very little value. While a pharmaceutical company may view a product as innovative if it is patentable, in reality for a pharmaceutical product to be truly innovative it must address an unmet or inadequately met need and offer health outcomes that were not previously achievable. [5]

A decline in research and development has been coined as Eroom's Law. [6] [7]

Current model

The FDA has implemented the Breakthrough Therapy Designation which should help bring new needed products to the market faster. [3]  One of the key concerns in the current patent system is that an innovation patent is overly generous given that it has a very low inventiveness threshold. [8]

The Medicines Patent Pool (MPP), is a United Nations-backed organisation founded in July 2010 [9] and based in Geneva, Switzerland. The MPP aims to improve access to appropriate, affordable HIV medicines and technologies for people living with HIV in developing countries. Working in partnership with a range of stakeholders, the MPP opens the door to generic low-cost production of key HIV therapies as well as fixed-dose combinations and paediatric formulations by creating a pool of relevant patents for sub-licensing and product development. [10]

Measurement

Kristopher J. Hult has proposed a new measurement based on novel and incremental innovation. The model predicts a decline of 40% for the 2010s. [11]

One current way to measure is the Internal rate of return, which is used to measure and compare the profitability of investments. Reuters used the method to measure 2012 and found that the rate fell from 7.7% to 7.2%. [12]

Barriers

Regulatory uncertainty is one area that stifles innovation. The approval processes are becoming increasingly complex. [13]

Proposed models

The Health Impact Fund (HIF) is a proposed pay-for-performance mechanism that would provide a market-based solution to problems concerning the development and distribution of medicines globally. It would incentivize the research and development of new pharmaceutical products that make substantial reductions in the global burden of disease. The HIF is the creation of a team of researchers led by the Yale philosopher Thomas Pogge and the University of Calgary economist Aidan Hollis, and is promoted by the non-profit organization Incentives for Global Health (IGH). [14]

Low investment into unpatentable options

An unintended but real consequence of the influence of a patent-heavy industry is that money is not put into unpatentable elements with the same fervor. A patent gives its owner the legal right to exclude others from making, using, or selling an invention for a a limited period of time. This can provide a competitive advantage for the patent holder. In health-care pharmaceuticals this advantage has proven to be highly profitable, driving over $83 billion dollars investment into Research and Development by the pharmaceutical industry in 2019. [15] This focus on the profits of patentability leaves a downside where investment money is not going, that is, into unpatentable research.

The process of having something be deemed usable as a medical product involves going through a clinical trial. These can be quite expensive, sometimes costing into the billions of dollars. [16] That elevated cost makes it a bad investment to pay for a medicine to be approved that does not guarantee years of profits to those that put up the money.

Biological patents are subject for debate and can vary from jurisdiction to jurisdiction. [17] As of 2013 the US Supreme Court ruled that isolation by itself is not sufficient for something to be deemed inventive subject matter, and thus patentable. [18] As a rule, raw natural material is generally rejected for patent approval by the United States Patent and Trademark Office, but genetically modified organisms have been patented several times.

The current patent structure lays incentives in a pattern that has been observed several times:

1) Some interesting characteristic is discovered through observation on something that is naturally occurring.
2) Experiments are made to verify the observed characteristic.
3) If the verified characteristic is deemed interesting, further experiments are made that attempt to isolate the compound that causes the interesting behavior.
4) Once isolated, the compound is again subject to experimentation to verify that it is, indeed, the one that causes the interesting behavior.
5) In order to be able to patent, efforts are then made to try and modify the original substance in a way that differentiates it.
6) Once the original substance has been successfully modified, a patent application is presented for the new substance.
7) If the patent is granted the new pharmaceutical candidate can then be taken to clinical trials.

An example of such an occurrence can be seen in the story of Human Breast Milk's cancer killing properties [19] and HAMLET (Human Alpha-lactalbumin Made LEthal to Tumor cells), the modified and patented [20] variation.

1) In 1995 Human Breast milk was observed that Human Breast Milk killed cancerous human cells while leaving healthy cells intact. [19]
2) Experiments were made and the observed characteristic was verified. [19]
3) Further experiments were made that isolated the compound multimeric alpha-lactalbumin (MAL) that caused the interesting behavior.
4) In 2000 an active compound for tumericidal activity was found to be a complex that involved oleic acid. [21] as well as alpha-lactalbumin.
5) Alpha-lactalbumin was partially unfolded to allow for release of the calcium ion and replacement with an oleic acid molecule, this enabled cytotoxicity and stabilization of the molecule. [22] [23]
6) A patent for making HAMLET through combining alpha-lactalbumin with oleic acid was granted in 2008 (expired 2018). A patent for recombinant HAMLET was presented in 2010 (expires 2030). This can be viewed as HAMLET 2.0. Peptides for second generation use were patented in 2011. Use of HAMLET as prophylaxis (preventive was) patented in 2013 (expires 2033). The use of HAMLET for Papilloma was patented in 2013 (expires 2023). [20]
7) HAMLET derived drug candidate, Alpha1H is in clinical trials for use with patients that have bladder cancer. [24]

As much money as has been put into HAMLET... it is remarkable that no money has been put into the use of actual Human Breast Milk as a medical product that could fight cancer. Again, Human Breast milk is not patentable, thus investing into clinical trials for its use as a medical product means that profit cannot be had.

There are many naturally occurring plants and compounds that have medicinal properties. An example of a plant that holds multiple properties, but that has not had money put into clinical trials, is Aloe vera .

"Research on the use of aloe for specific conditions shows:

Burns and wounds. Application of aloe gel appears to shorten the duration of wound healing for first- and second-degree burns. Aloe gel might also promote wound healing.

Acne. Research suggests that aloe gel, applied in the morning and evening in addition to the use of the topical prescription acne medicine tretinoin (Retin-A, Atralin, others), might be more effective in reducing acne than using a topical prescription alone.

Psoriasis. Aloe extract cream might reduce redness, scaling, itching and inflammation caused by mild to moderate psoriasis. You might need to use the cream several times a day for a month or more to see improvements in your skin.

Herpes simplex virus. Applying a cream containing aloe extract might help lesions heal sooner.

Oral lichen planus. Research suggests that twice-daily application of aloe gel for eight weeks might help reduce symptoms of this inflammatory condition that affects the inside of the mouth.

Constipation. Whether oral use of aloe latex is effective at treating constipation is unclear. While it acts as a laxative, aloe latex can also cause abdominal cramps and diarrhea." [25]

It is impossible to quantify something that does not happen. We can count how much money got put into research by companies that rely on patents and we can estimate how much a process for a clinical trial costs, but it is impossible to define how much money SHOULD have been spent just like it is impossible to estimate the effect of a process that has not been realized.

How much money could have been saved world-wide if doctors were allowed to prescribe medicine that many people can grow at home at a very low cost, like Aloe vera? How many people could have been spared death by skin cancer if human breast milk could be used topically to eliminate cancerous cells? We cannot know. We just know that research and clinical trial money was not put into unpatentable targets at the same rate and volume that it was for patentable ones.

See also

Related Research Articles

Experimental cancer treatments are mainstream medical therapies intended to treat cancer by improving on, supplementing or replacing conventional methods. However, researchers are still trying to determine whether these treatments are safe and effective treatments. Experimental cancer treatments are normally available only to people who participate in formal research programs, which are called clinical trials. Occasionally, a seriously ill person may be able to access an experimental drug through an expanded access program. Some of the treatments have regulatory approval for treating other conditions. Health insurance and publicly funded health care programs normally refuse to pay for experimental cancer treatments.

<span class="mw-page-title-main">Chemical patent</span> Patent for an invention in the chemical or pharmaceuticals industry

A chemical patent, pharmaceutical patent or drug patent is a patent for an invention in the chemical or pharmaceuticals industry. Strictly speaking, in most jurisdictions, there are essentially no differences between the legal requirements to obtain a patent for an invention in the chemical or pharmaceutical fields, in comparison to obtaining a patent in the other fields, such as in the mechanical field. A chemical patent or a pharmaceutical patent is therefore not a sui generis right, i.e. a special legal type of patent.

<span class="mw-page-title-main">Medication</span> Substance used to diagnose, cure, treat, or prevent disease

A medication is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and relies on the science of pharmacology for continual advancement and on pharmacy for appropriate management.

<span class="mw-page-title-main">Breast milk</span> Milk produced by the mammary glands in the breast of a female human

Breast milk or mother's milk is milk produced by the mammary glands in the breast of female humans. Breast milk is the primary source of nutrition for newborn infants, comprising fats, proteins, carbohydrates, and a varying composition of minerals and vitamins. Breast milk also contains substances that help protect an infant against infection and inflammation, such as symbiotic bacteria and other microorganisms and immunoglobulin A, whilst also contributing to the healthy development of the infant's immune system and gut microbiome.

Pharming, a portmanteau of farming and pharmaceutical, refers to the use of genetic engineering to insert genes that code for useful pharmaceuticals into host animals or plants that would otherwise not express those genes, thus creating a genetically modified organism (GMO). Pharming is also known as molecular farming, molecular pharming, or biopharming.

<span class="mw-page-title-main">Medical research</span> Wide array of research

Medical research, also known as health research, refers to the process of using scientific methods with the aim to produce knowledge about human diseases, the prevention and treatment of illness, and the promotion of health.

A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, they include vaccines, whole blood, blood components, allergenics, somatic cells, gene therapies, tissues, recombinant therapeutic protein, and living medicines used in cell therapy. Biologics can be composed of sugars, proteins, nucleic acids, or complex combinations of these substances, or may be living cells or tissues. They are isolated from living sources—human, animal, plant, fungal, or microbial. They can be used in both human and animal medicine.

Test data exclusivity refers to protection of clinical trial data required to be submitted to a regulatory agency to prove safety and efficacy of a new drug, and prevention of generic drug manufacturers from relying on this data in their own applications. It provides a form of market exclusivity outside that provided by patent rights.

<span class="mw-page-title-main">Drug development</span> Process of bringing a new pharmaceutical drug to the market

Drug development is the process of bringing a new pharmaceutical drug to the market once a lead compound has been identified through the process of drug discovery. It includes preclinical research on microorganisms and animals, filing for regulatory status, such as via the United States Food and Drug Administration for an investigational new drug to initiate clinical trials on humans, and may include the step of obtaining regulatory approval with a new drug application to market the drug. The entire process—from concept through preclinical testing in the laboratory to clinical trial development, including Phase I–III trials—to approved vaccine or drug typically takes more than a decade.

<span class="mw-page-title-main">Lapatinib</span> Cancer medication

Lapatinib (INN), used in the form of lapatinib ditosylate (USAN) is an orally active drug for breast cancer and other solid tumours. It is a dual tyrosine kinase inhibitor which interrupts the HER2/neu and epidermal growth factor receptor (EGFR) pathways. It is used in combination therapy for HER2-positive breast cancer. It is used for the treatment of patients with advanced or metastatic breast cancer whose tumors overexpress HER2 (ErbB2).

<span class="mw-page-title-main">Albumin</span> Family of globular proteins

Albumin is a family of globular proteins, the most common of which are the serum albumins. All of the proteins of the albumin family are water-soluble, moderately soluble in concentrated salt solutions, and experience heat denaturation. Albumins are commonly found in blood plasma and differ from other blood proteins in that they are not glycosylated. Substances containing albumins are called albuminoids.

α-Lactalbumin Protein-coding gene in the species Homo sapiens

α-Lactalbumin, also known as LALBA, is a protein that in humans is encoded by the LALBA gene.

<span class="mw-page-title-main">Myriad Genetics</span> American biotechnology company

Myriad Genetics, Inc. is an American genetic testing and precision medicine company based in Salt Lake City, Utah, United States. Myriad employs a number of proprietary technologies that permit doctors and patients to understand the genetic basis of human disease and the role that genes play in the onset, progression and treatment of disease. This information is used to guide the development of new products that assess an individual's risk for developing disease later in life, identify a patient's likelihood of responding to a particular drug therapy, assess a patient's risk of disease progression and disease recurrence, and measure disease activity.

<span class="mw-page-title-main">Gadobutrol</span> Chemical compound

Gadobutrol (INN) (Gd-DO3A-butrol) is a gadolinium-based MRI contrast agent (GBCA).

Medication costs, also known as drug costs are a common health care cost for many people and health care systems. Prescription costs are the costs to the end consumer. Medication costs are influenced by multiple factors such as patents, stakeholder influence, and marketing expenses. A number of countries including Canada, parts of Europe, and Brazil use external reference pricing as a means to compare drug prices and to determine a base price for a particular medication. Other countries use pharmacoeconomics, which looks at the cost/benefit of a product in terms of quality of life, alternative treatments, and cost reduction or avoidance in other parts of the health care system. Structures like the UK's National Institute for Health and Clinical Excellence and to a lesser extent Canada's Common Drug Review evaluate products in this way.

The Health Impact Fund is a proposed pay-for-performance mechanism that would provide a market-based solution to problems concerning the development and distribution of medicines globally. It would incentivize the research and development of new pharmaceutical products that make substantial reductions in the global burden of disease. The Health Impact Fund is the creation of a team of researchers led by the Yale philosopher Thomas Pogge and the University of Calgary economist Aidan Hollis, and is promoted by the non-profit organization Incentives for Global Health (IGH).

HAMLET is a complex between alpha-lactalbumin and oleic acid that has been shown in cell culture experiments to induce cell death in tumor cells, but not in healthy cells.

Anticancer genes have a special ability to target and kill cancer cells without harming healthy ones. They do this through processes like programmed cell death, known as apoptosis, and other mechanisms like necrosis and autophagy. In the late 1990s, researchers discovered these genes while studying cancer cells. Sometimes, mutations or changes in these genes can occur, which might lead to cancer. These changes can include small alterations in the DNA sequence or larger rearrangements that affect the gene's function. When these anticancer genes are lost or altered, it can disrupt their ability to control cell growth, potentially leading to the development of cancer.

GcMAF is a protein produced by modification of vitamin D-binding protein. It has been falsely promoted as a treatment for various medical conditions, but claims of its benefits are not supported by evidence.

Breastmilk medicine refers to the non-nutritional usage of human breast milk (HBM) as a medicine or therapy to cure diseases. Breastmilk is perceived as an important food that provides essential nutrition to infants. It also provides protection in terms of immunity by direct transfer of antibodies from mothers to infants. The immunity developed via this mean protects infants from diseases such as respiratory diseases, middle ear infections, and gastrointestinal diseases. HBM can also produce lifelong positive therapeutic effects on a number of chronic diseases, including diabetes mellitus, obesity, hyperlipidemia, hypertension, cardiovascular diseases, autoimmunity, and asthma.

References

  1. ↑ Review of the Innovation Patent System (PDF). 2014. p. 2. ISBN   978-0-9804542-7-7. Archived from the original (PDF) on 2015-02-27.
  2. ↑ Pogge, Thomas (18 December 2011), Medicine for the 99 percent , retrieved 2015-04-15
  3. 1 2 Milet, Sylvain. "MedidataVoice: The Next Wave Of Pharmaceutical Innovation: An Interview With Bernard Munos". Forbes . Retrieved 2015-04-15.
  4. ↑ Westerling, Ragnar; Westin, Marcus; McKee, Martin; Hoffmann, Rasmus; Plug, Iris; Rey, Grégoire; Jougla, Eric; Lang, Katrin; Pärna, Kersti; Alfonso, José L.; MacKenbach, Johan P. (2014). "The timing of introduction of pharmaceutical innovations in seven European countries". Journal of Evaluation in Clinical Practice. 20 (4): 301–310. doi:10.1111/jep.12122. PMC   4282430 . PMID   24750393.
  5. ↑ Morgan, Steven; Lopert, Ruth; Greyson, Devon (2008). "Toward a definition of pharmaceutical innovation". Open Med. 2 (1): e4–7. PMC   3091590 . PMID   21602949.
  6. ↑ Feyman, Yevgeniy (13 April 2015). "Is Pharmaceutical Productivity In Decline? Maybe Not". Forbes. Retrieved 16 April 2015.
  7. ↑ Scannell, JW; Blanckley, A; Boldon, H; Warrington, B (2012). "Eroom's Law in pharmaceutical R&D". Nature Reviews Drug Discovery. 11 (3): 191–200. doi:10.1038/nrd3681. PMID   22378269. S2CID   3344476.
  8. ↑ "Review of the Innovation Patent System" (PDF). www.acip.gov.au. Archived from the original (PDF) on 2015-02-27. Retrieved 2015-04-15.
  9. ↑ "Archived copy" (PDF). www.unitaid.eu. Archived from the original (PDF) on 23 April 2010. Retrieved 25 January 2022.{{cite web}}: CS1 maint: archived copy as title (link)
  10. ↑ RUNYOWA, TAVENGWA (10 March 2011). "Medicines Patent Pool Aims To Increase Access To HIV Drugs In Developing Countries". IP Watch. Retrieved 2 June 2015.
  11. ↑ Hult, Kristopher. "Kristopher J. Hult: Research". University of Chicago - Department of Economics. Archived from the original on 2015-04-16. Retrieved 2015-04-16.
  12. ↑ "Measuring the return from pharmaceutical innovation 2012". Reuters. Archived from the original on 16 April 2015. Retrieved 16 April 2015.
  13. ↑ "measuring the return from pharmaceutical innovation 2014" (PDF). www2.deloitte.com. Retrieved 2015-04-15.
  14. ↑ Hollis, A; Grootendorst, P; Levine, DK; Pogge, T; Edwards, AM (2011). "New approaches to rewarding pharmaceutical innovation". Canadian Medical Association Journal. 183 (6): 681–5. doi:10.1503/cmaj.100375. PMC   3071389 . PMID   21149519.
  15. ↑ "Research and Development in the Pharmaceutical Industry". www.cbo.gov. 8 April 2021. Retrieved 2022-01-13.
  16. ↑ Dimasi, Joseph A; Grabowski, Henry G; Hansen, Ronald W (2016). "Innovation in the pharmaceutical industry: New estimates of R&D costs". Journal of Health Economics. 47: 20–33. doi:10.1016/j.jhealeco.2016.01.012. hdl: 10161/12742 . PMID   26928437.
  17. ↑ Sharples, Andrew (2011-03-23). "Gene Patents in Europe Relatively Stable Despite Uncertainty in the U.S." Genetic Engineering and Biotechnology News. Retrieved 2013-06-13.
  18. ↑ Sheehan, Teige. "The Supreme Court Holds Genes Are Patent-Ineligible Products of Nature" (PDF). Retrieved 20 June 2020.
  19. 1 2 3 Håkansson A, Zhivotovsky B, Orrenius S, Sabharwal H, Svanborg C (August 1995). "Apoptosis induced by a human milk protein". Proc. Natl. Acad. Sci. U.S.A. 92 (17): 8064–8. Bibcode:1995PNAS...92.8064H. doi: 10.1073/pnas.92.17.8064 . PMC   41287 . PMID   7644538.
  20. 1 2 HAMLET PHARMA. "Patents". www.hamletpharma.com. Retrieved 13 Jan 2022.
  21. ↑ Svensson M, Håkansson A, Mossberg AK, Linse S, Svanborg C (April 2000). "Conversion of alpha-lactalbumin to a protein inducing apoptosis". Proc. Natl. Acad. Sci. U.S.A. 97 (8): 4221–6. Bibcode:2000PNAS...97.4221S. doi: 10.1073/pnas.97.8.4221 . PMC   18203 . PMID   10760289.
  22. ↑ Gustafsson L, Hallgren O, Mossberg AK, Pettersson J, Fischer W, Aronsson A, Svanborg C (May 2005). "HAMLET kills tumor cells by apoptosis: structure, cellular mechanisms, and therapy". J. Nutr. 135 (5): 1299–303. doi: 10.1093/jn/135.5.1299 . PMID   15867328.
  23. ↑ Pettersson-Kastberg J, Aits S, Gustafsson L, Mossberg A, Storm P, Trulsson M, Persson F, Mok KH, Svanborg C (2009). "Can misfolded proteins be beneficial? The HAMLET case". Ann. Med. 41 (3): 162–76. doi:10.1080/07853890802502614. PMID   18985467. S2CID   31198109.
  24. ↑ HAMLET PHARMA. "About Clinical Trials". www.hamletpharma.com. Retrieved 13 Jan 2022.
  25. ↑ "Aloe (Aloe vera)". Mayo Clinic. 17 September 2017. Retrieved 21 January 2020.
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